{ "generated": "2026-10-12", "snapshot": "UNII 4Aug2026 \u00b7 Drugs@FDA 12 Oct 2026 \u00b7 Orange Book 12 Oct 2026 \u00b7 EMA 12 Oct 2026 \u00b7 Health Canada 12 Oct 2026 \u00b7 Swissmedic 12 Oct 2026 \u00b7 PMDA 12 Oct 2026 \u00b7 GSRS relationship graph", "funnel": { "foreign_rows": 18107, "resolved": 6493, "unmatched": 11614, "biologic_or_mixture": 420, "in_us": 2027, "in_us_string": 1878, "in_us_gsrs": 149, "review": 9, "candidate_small_mol": 637, "needs_translit_unmatched": 232, "named_drug": 338, "unnamed": 267, "no_authorisation_in_loaded_registers": 32, "unresolved_by_source": { "EMA": 375, "EU national (Art57)": 7914, "Health Canada": 221, "PMDA-JP": 232, "Swissmedic": 2872 }, "rows_by_source": { "EMA": 2304, "EU national (Art57)": 11892, "Health Canada": 702, "PMDA-JP": 234, "Swissmedic": 2975 } }, "us_ingredient_rows": 188948, "counts": { "named": 338, "unnamed": 267, "review": 9, "dossier_only": 32, "orphan": 2, "inn": 334, "multi_regulator": 22 }, "candidates": [ { "unii": "HB6PN45W4J", "name": "IDEBENONE", "moiety": "IDEBENONE", "inn": true, "usan": true, "type": "chemical", "sources": [ "EMA", "EU national (Art57)" ], "orphan": true, "tier": "exact-moiety", "examples": [ "Idebenone", "idebenone" ], "area": "Optic Atrophy, Hereditary, Leber", "atc": "N06BX13", "indication": "Raxone is indicated for the treatment of visual impairment in adolescent and adult patients with Leber\u2019s Hereditary Optic Neuropathy (LHON).", "approved": "08/09/2015", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EMA": "authorised", "EU national (Art57)": "authorised" }, "n_countries": 4, "countries": [ "Iceland", "Liechtenstein", "Norway", "Portugal" ], "pubmed": 36, "trials": 35, "us_orphan": "unknown", "chembl": [ { "disease": "Attention Deficit Disorder with Hyperactivity", "phase": 4.0 }, { "disease": "Optic Atrophy, Hereditary, Leber", "phase": 4.0 }, { "disease": "Muscular Dystrophy, Duchenne", "phase": 3.0 }, { "disease": "Friedreich Ataxia", "phase": 3.0 }, { "disease": "MELAS Syndrome", "phase": 2.0 }, { "disease": "Multiple Sclerosis, Chronic Progressive", "phase": 1.0 }, { "disease": "Non-alcoholic Fatty Liver Disease", "phase": 1.0 }, { "disease": "Huntington Disease", "phase": 1.0 } ], "orpha": null, "eu_orphan_designation": { "use": "Treatment of Leber's hereditary optic neuropathy", "status": "Expired", "number": "EU/3/07/434" }, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] }, "eu_orphan_designations": [ { "use": "Treatment of Leber's hereditary optic neuropathy", "status": "Expired", "number": "EU/3/07/434" }, { "use": "Treatment of Friedreich's ataxia", "status": "Positive", "number": "EU/3/04/189" }, { "use": "Treatment of Duchenne muscular dystrophy", "status": "Positive", "number": "EU/3/07/437" }, { "use": "Treatment of Friedreich's ataxia", "status": "Positive", "number": "EU/3/01/062" } ], "us_crl": { "application": "NDA (Chiesi, LHON)", "date": "02/27/2026", "kind": "COMPLETE RESPONSE", "company": "Chiesi Farmaceutici S.p.A", "source": "https://mitopatients.org/wp-content/uploads/2026/03/Chiesi-statement-FDA.pdf" } }, { "unii": "K824X25AYA", "name": "LERIGLITAZONE", "moiety": "LERIGLITAZONE", "inn": true, "usan": true, "type": "chemical", "sources": [ "EMA" ], "orphan": true, "tier": "exact-moiety", "examples": [ "leriglitazone", "leriglitazone hydrochloride" ], "area": "Adrenoleukodystrophy", "atc": "A16AX23", "indication": "Nezglyal is indicated for the treatment of Cerebral Adrenoleukodystrophy (cALD) in males with Adrenoleukodystrophy (ALD) aged 2 to 12 years with non Gadolinium (Gd) enhancing lesions (i.e. Gd negative) in brain Magnetic Resonance Imaging (MRI), with a Neurological Functional Score (NFS) of 0 or 1.", "approved": "21/09/2026", "flags": [ "US_RPD_DESIGNATION_HELD: Minoryx holds FDA Rare Pediatric Disease + Fast Track + orphan designations for X-ALD (sponsor-stated); CALYX pivotal fully enrolled 2026-09, FDA-agreed NDA-enabling, topline early 2028 \u2014 a US NDA before the 2029-09-30 sunset is plausible but the originator's to file" ], "foreign_status": "authorised", "status_by_source": { "EMA": "authorised" }, "n_countries": 0, "countries": [], "pubmed": 2, "trials": 2, "us_orphan": "unknown", "chembl": [ { "disease": "Adrenoleukodystrophy", "phase": 2.0 }, { "disease": "Friedreich Ataxia", "phase": 2.0 } ], "orpha": null, "eu_orphan_designation": { "use": "Treatment of Friedreich\u2019s ataxia", "status": "Positive", "number": "EU/3/19/2212" }, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] }, "eu_orphan_designations": [ { "use": "Treatment of Friedreich\u2019s ataxia", "status": "Positive", "number": "EU/3/19/2212" } ] }, { "unii": "I6B4B2U96P", "name": "VILDAGLIPTIN", "moiety": "VILDAGLIPTIN", "inn": true, "usan": true, "type": "chemical", "sources": [ "EMA", "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Vildagliptin", "vildagliptin" ], "area": "Diabetes Mellitus, Type 2", "atc": "A10BH02", "indication": "Vildagliptin is indicated as an adjunct to diet and exercise to improve glycaemic control in adults with type 2 diabetes mellitus: as monotherapy in patients in whom metformin is inappropriate due to contraindications or intolerance. in combination with other medicinal products for the treatment of diabetes, including insulin, when these do not provide adequate glycaemic control (see sections 4.4, 4.5 and 5.1 for available data on different combinations).", "approved": "19/11/2008", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EMA": "authorised", "EU national (Art57)": "authorised" }, "n_countries": 30, "countries": [ "Austria", "Belgium", "Bulgaria", "Croatia", "Cyprus", "Czech Republic", "Denmark", "Estonia", "Finland", "France", "Germany", "Greece", "Hungary", "Iceland", "Ireland", "Italy", "Latvia", "Liechtenstein", "Lithuania", "Malta", "Netherlands", "Norway", "Poland", "Portugal", "Romania", "Slovakia", "Slovenia", "Spain", "Sweden", "United Kingdom (Northern Ireland)" ], "pubmed": 0, "trials": 209, "us_orphan": "unknown", "chembl": [ { "disease": "Diabetes Mellitus", "phase": 4.0 }, { "disease": "Diabetes Mellitus, Type 2", "phase": 4.0 }, { "disease": "Prediabetic State", "phase": 3.0 }, { "disease": "Diabetes Mellitus, Type 1", "phase": 3.0 }, { "disease": "Glucose Intolerance", "phase": 2.0 }, { "disease": "Diabetes, Gestational", "phase": 2.0 }, { "disease": "Breast Neoplasms", "phase": 2.0 }, { "disease": "Depressive Disorder, Major", "phase": 1.0 } ], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] }, "us_crl": { "application": "NDA (Novartis, type 2 diabetes)", "date": "02/2007", "kind": "APPROVABLE LETTER", "company": "Novartis", "source": "Novartis approvable letters Feb 2007 and 2008; never resubmitted" } }, { "unii": "URQ2517572", "name": "ALFACALCIDOL", "moiety": "ALFACALCIDOL", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)", "Health Canada" ], "orphan": false, "tier": "exact-moiety", "examples": [ "ALFACALCIDOL", "Alfacalcidol" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised", "Health Canada": "authorised" }, "n_countries": 29, "countries": [ "Austria", "Belgium", "Bulgaria", "Cyprus", "Czech Republic", "Denmark", "Estonia", "Finland", "France", "Germany", "Greece", "Hungary", "Iceland", "Ireland", "Italy", "Latvia", "Lithuania", "Luxembourg", "Malta", "Netherlands", "Norway", "Poland", "Portugal", "Romania", "Slovakia", "Slovenia", "Spain", "Sweden", "United Kingdom (Northern Ireland)" ], "pubmed": 31, "trials": 52, "us_orphan": "unknown", "chembl": [ { "disease": "Osteoporosis", "phase": 3.0 }, { "disease": "Muscular Diseases", "phase": 3.0 }, { "disease": "Osteoporosis, Postmenopausal", "phase": 3.0 }, { "disease": "Pelvic Organ Prolapse", "phase": 3.0 }, { "disease": "Calcinosis", "phase": 2.0 }, { "disease": "Death, Sudden, Cardiac", "phase": 2.0 }, { "disease": "Scoliosis", "phase": 2.0 }, { "disease": "Renal Insufficiency", "phase": 2.0 } ], "disease_terms": "Osteoporosis; Muscular Diseases; Osteoporosis, Postmenopausal; Pelvic Organ Prolapse", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "PA1123N395", "name": "BILASTINE", "moiety": "BILASTINE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)", "Health Canada" ], "orphan": false, "tier": "exact-moiety", "examples": [ "BILASTINE", "Bilastine", "Bilastine Monohydrate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised", "Health Canada": "authorised" }, "n_countries": 29, "countries": [ "Austria", "Belgium", "Bulgaria", "Croatia", "Cyprus", "Czech Republic", "Denmark", "Estonia", "Finland", "France", "Germany", "Greece", "Hungary", "Iceland", "Ireland", "Italy", "Latvia", "Lithuania", "Luxembourg", "Malta", "Norway", "Poland", "Portugal", "Romania", "Slovakia", "Slovenia", "Spain", "Sweden", "United Kingdom (Northern Ireland)" ], "pubmed": 12, "trials": 28, "us_orphan": "unknown", "chembl": [ { "disease": "Eye Manifestations", "phase": 4.0 }, { "disease": "Hypersensitivity", "phase": 4.0 }, { "disease": "Rhinitis, Allergic, Seasonal", "phase": 3.0 }, { "disease": "Urticaria", "phase": 3.0 }, { "disease": "Rhinitis, Allergic, Perennial", "phase": 3.0 }, { "disease": "Conjunctivitis, Allergic", "phase": 3.0 }, { "disease": "Cold Urticaria", "phase": 2.0 } ], "disease_terms": "Eye Manifestations; Hypersensitivity", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "47ISU063SG", "name": "ZUCLOPENTHIXOL", "moiety": "ZUCLOPENTHIXOL", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)", "Health Canada" ], "orphan": false, "tier": "moiety-base", "examples": [ "ZUCLOPENTHIXOL DECANOATE", "Zuclopenthixol Acetate", "Zuclopenthixol Decanoate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised", "Health Canada": "authorised" }, "n_countries": 29, "countries": [ "Austria", "Belgium", "Bulgaria", "Cyprus", "Czech Republic", "Denmark", "Estonia", "Finland", "France", "Germany", "Greece", "Hungary", "Iceland", "Ireland", "Italy", "Latvia", "Lithuania", "Luxembourg", "Malta", "Netherlands", "Norway", "Poland", "Portugal", "Romania", "Slovakia", "Slovenia", "Spain", "Sweden", "United Kingdom (Northern Ireland)" ], "pubmed": 3, "trials": 10, "us_orphan": "unknown", "chembl": [ { "disease": "Psychotic Disorders", "phase": 3.0 }, { "disease": "Anxiety", "phase": 3.0 }, { "disease": "Dementia", "phase": 3.0 }, { "disease": "Depressive Disorder", "phase": 3.0 }, { "disease": "Schizophrenia", "phase": 3.0 } ], "disease_terms": "Psychotic Disorders; Anxiety; Dementia; Depressive Disorder; Schizophrenia", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "G4PX8C4HKV", "name": "GLICLAZIDE", "moiety": "GLICLAZIDE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)", "Health Canada" ], "orphan": false, "tier": "exact-moiety", "examples": [ "GLICLAZIDE", "Gliclazide" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised", "Health Canada": "authorised" }, "n_countries": 27, "countries": [ "Austria", "Belgium", "Bulgaria", "Croatia", "Cyprus", "Czech Republic", "Denmark", "Estonia", "France", "Germany", "Greece", "Hungary", "Iceland", "Ireland", "Italy", "Latvia", "Lithuania", "Luxembourg", "Malta", "Netherlands", "Poland", "Portugal", "Romania", "Slovakia", "Slovenia", "Spain", "United Kingdom (Northern Ireland)" ], "pubmed": 8, "trials": 85, "us_orphan": "unknown", "chembl": [ { "disease": "Diabetes Mellitus", "phase": 4.0 }, { "disease": "Diabetes Mellitus, Type 2", "phase": 3.0 }, { "disease": "Epilepsy", "phase": 1.0 } ], "disease_terms": "Diabetes Mellitus", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "081D12SI0Z", "name": "ATOSIBAN", "moiety": "ATOSIBAN", "inn": true, "usan": true, "type": "chemical", "sources": [ "EMA", "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Atosiban", "Atosiban Acetate", "atosiban (as acetate)" ], "area": "Obstetric Labor, Premature", "atc": "G02CX01", "indication": "Atosiban is indicated to delay imminent pre-term birth in pregnant adult women with: regular uterine contractions of at least 30 seconds\u2019 duration at a rate of ? 4 per 30 minutes; a cervical dilation of 1 to 3 cm (0-3 for nulliparas) and effacement of ? 50%; a gestational age from 24 until 33 completed weeks; a normal foetal heart rate.", "approved": "31/07/2013", "flags": [], "foreign_status": "authorised", "status_by_source": { "EMA": "authorised", "EU national (Art57)": "authorised" }, "n_countries": 24, "countries": [ "Austria", "Belgium", "Bulgaria", "Croatia", "Czech Republic", "Denmark", "Finland", "France", "Germany", "Greece", "Iceland", "Italy", "Latvia", "Liechtenstein", "Luxembourg", "Netherlands", "Norway", "Poland", "Portugal", "Slovakia", "Slovenia", "Spain", "Sweden", "United Kingdom (Northern Ireland)" ], "pubmed": 7, "trials": 23, "us_orphan": "unknown", "chembl": [ { "disease": "Premature Birth", "phase": 3.0 }, { "disease": "Endometriosis", "phase": 2.0 } ], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "X015L14V0O", "name": "BROMAZEPAM", "moiety": "BROMAZEPAM", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)", "Health Canada" ], "orphan": false, "tier": "exact-moiety", "examples": [ "BROMAZEPAM", "Bromazepam" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised", "Health Canada": "authorised" }, "n_countries": 24, "countries": [ "Austria", "Belgium", "Bulgaria", "Croatia", "Cyprus", "Czech Republic", "Denmark", "Estonia", "France", "Germany", "Greece", "Ireland", "Italy", "Latvia", "Lithuania", "Luxembourg", "Malta", "Netherlands", "Poland", "Portugal", "Romania", "Slovakia", "Slovenia", "Spain" ], "pubmed": 6, "trials": 5, "us_orphan": "unknown", "chembl": [ { "disease": "Anxiety", "phase": 4.0 }, { "disease": "Dementia", "phase": 3.0 }, { "disease": "Depressive Disorder", "phase": 3.0 }, { "disease": "Schizophrenia", "phase": 3.0 } ], "disease_terms": "Anxiety", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "137R1N49AD", "name": "AGOMELATINE", "moiety": "AGOMELATINE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EMA", "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Agomelatine", "agomelatine" ], "area": "Depressive Disorder, Major", "atc": "N06AX22", "indication": "Treatment of major depressive episodes in adults.", "approved": "19/02/2009", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EMA": "authorised", "EU national (Art57)": "authorised" }, "n_countries": 22, "countries": [ "Austria", "Bulgaria", "Czech Republic", "Denmark", "Estonia", "Finland", "France", "Germany", "Hungary", "Iceland", "Ireland", "Liechtenstein", "Lithuania", "Malta", "Netherlands", "Norway", "Poland", "Portugal", "Slovakia", "Spain", "Sweden", "United Kingdom (Northern Ireland)" ], "pubmed": 9, "trials": 27, "us_orphan": "unknown", "chembl": [ { "disease": "Depressive Disorder, Major", "phase": 4.0 }, { "disease": "Depressive Disorder", "phase": 4.0 }, { "disease": "Anxiety", "phase": 3.0 }, { "disease": "Dementia", "phase": 3.0 }, { "disease": "Schizophrenia", "phase": 3.0 }, { "disease": "Obsessive-Compulsive Disorder", "phase": 2.0 } ], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "468GE2241L", "name": "PROPIVERINE", "moiety": "PROPIVERINE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)", "Health Canada" ], "orphan": false, "tier": "moiety-base", "examples": [ "PROPIVERINE HYDROCHLORIDE", "Propiverine Hydrochloride" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised", "Health Canada": "authorised" }, "n_countries": 14, "countries": [ "Austria", "Belgium", "Croatia", "Czech Republic", "Germany", "Greece", "Ireland", "Italy", "Luxembourg", "Portugal", "Slovakia", "Slovenia", "Spain", "United Kingdom (Northern Ireland)" ], "pubmed": 15, "trials": 17, "us_orphan": "unknown", "chembl": [ { "disease": "Urinary Incontinence", "phase": 4.0 }, { "disease": "Polyuria", "phase": 4.0 }, { "disease": "Urinary Bladder, Overactive", "phase": 3.0 } ], "disease_terms": "Urinary Incontinence; Polyuria", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "Z1R9N0A399", "name": "TENOXICAM", "moiety": "TENOXICAM", "inn": true, "usan": true, "type": "chemical", "sources": [ "EU national (Art57)", "Health Canada" ], "orphan": false, "tier": "exact-moiety", "examples": [ "TENOXICAM", "Tenoxicam" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised", "Health Canada": "authorised" }, "n_countries": 11, "countries": [ "Belgium", "Cyprus", "France", "Greece", "Italy", "Luxembourg", "Portugal", "Romania", "Spain", "Sweden", "United Kingdom (Northern Ireland)" ], "pubmed": 4, "trials": 41, "us_orphan": "unknown", "chembl": [ { "disease": "Rheumatic Diseases", "phase": 4.0 }, { "disease": "Pain", "phase": 2.0 } ], "disease_terms": "Rheumatic Diseases", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "8OOT6M1PC7", "name": "DELAMANID", "moiety": "DELAMANID", "inn": true, "usan": true, "type": "chemical", "sources": [ "EMA", "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Delamanid" ], "area": "Tuberculosis, Multidrug-Resistant", "atc": "J04AK06", "indication": "Deltyba is indicated for use as part of an appropriate combination regimen for pulmonary multi-drug resistant tuberculosis (MDR-TB) in adults, adolescents, children and infants with a body weight of at least 10 kg when an effective treatment regimen cannot otherwise be composed for reasons of resistance or tolerability (see sections 4.2, 4.4 and 5.1). Consideration should be given to official guidance on the appropriate use of antibacterial agents.", "approved": "27/04/2014", "flags": [], "foreign_status": "authorised", "status_by_source": { "EMA": "authorised", "EU national (Art57)": "authorised" }, "n_countries": 3, "countries": [ "Iceland", "Liechtenstein", "Norway" ], "pubmed": 40, "trials": 44, "us_orphan": "unknown", "chembl": [ { "disease": "Tuberculosis, Multidrug-Resistant", "phase": 4.0 }, { "disease": "Tuberculosis", "phase": 4.0 }, { "disease": "Tuberculosis, Pulmonary", "phase": 3.0 } ], "orpha": { "code": "3389", "name": "Tuberculosis", "rarity": "rare", "rarity_evidence": "Orphanet 1-5 / 10 000 (Europe) \u2248 100,499 in the US, under 200,000", "us_prevalence_est": 100499, "onset": "mixed", "onset_evidence": "Orphanet age of onset: All ages \u2014 spans pediatric and adult" }, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "PASS", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "Orphanet 1-5 / 10 000 (Europe) \u2248 100,499 in the US, under 200,000" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "Orphanet age of onset: All ages \u2014 spans pediatric and adult" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "3XVL385Q0M", "name": "FILGOTINIB", "moiety": "FILGOTINIB", "inn": true, "usan": true, "type": "chemical", "sources": [ "EMA", "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Filgotinib", "filgotinib maleate" ], "area": "Arthritis, Rheumatoid", "atc": "L04AF04", "indication": "Rheumatoid arthritis Jyseleca is indicated for the treatment of moderate to severe active rheumatoid arthritis in adult patients who have responded inadequately to, or who are intolerant to one or more disease modifying anti rheumatic drugs (DMARDs). Jyseleca may be used as monotherapy or in combination with methotrexate (MTX). Ulcerative colitis Jyseleca is indicated for the treatment of adult patients with moderately to severely active ulcerative colitis who have had an inadequate response with, lost response to, or were intolerant to either conventional therapy or a biologic agent.", "approved": "24/09/2020", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EMA": "authorised", "EU national (Art57)": "authorised" }, "n_countries": 3, "countries": [ "Iceland", "Liechtenstein", "Norway" ], "pubmed": 11, "trials": 66, "us_orphan": "unknown", "chembl": [ { "disease": "Arthritis, Rheumatoid", "phase": 3.0 }, { "disease": "Crohn Disease", "phase": 3.0 }, { "disease": "Colitis, Ulcerative", "phase": 3.0 }, { "disease": "Arthritis, Psoriatic", "phase": 3.0 }, { "disease": "Spondylitis, Ankylosing", "phase": 3.0 }, { "disease": "Spondylarthropathies", "phase": 3.0 }, { "disease": "Sjogren's Syndrome", "phase": 2.0 }, { "disease": "Lupus Erythematosus, Cutaneous", "phase": 2.0 }, { "disease": "Uveitis", "phase": 2.0 }, { "disease": "Inflammatory Bowel Diseases", "phase": 2.0 }, { "disease": "Lupus Erythematosus, Systemic", "phase": 2.0 }, { "disease": "Kidney Diseases", "phase": 1.0 }, { "disease": "Arthritis, Juvenile", "phase": 1.0 } ], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] }, "us_crl": { "application": "NDA (Gilead, rheumatoid arthritis)", "date": "08/18/2020", "kind": "COMPLETE RESPONSE", "company": "Gilead", "source": "Gilead press release 2020-08-18; US pursuit ended 2020-12" } }, { "unii": "6K6L7E3F1L", "name": "GEFAPIXANT", "moiety": "GEFAPIXANT", "inn": true, "usan": true, "type": "chemical", "sources": [ "EMA", "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Gefapixant", "gefapixant" ], "area": "Cough", "atc": "R05DB29", "indication": "Lyfnua is indicated in adults for the treatment of refractory or unexplained chronic cough.", "approved": "15/09/2023", "flags": [], "foreign_status": "authorised", "status_by_source": { "EMA": "authorised", "EU national (Art57)": "authorised" }, "n_countries": 3, "countries": [ "Iceland", "Liechtenstein", "Norway" ], "pubmed": 0, "trials": 27, "us_orphan": "unknown", "chembl": [ { "disease": "Cough", "phase": 3.0 }, { "disease": "Idiopathic Pulmonary Fibrosis", "phase": 2.0 }, { "disease": "Osteoarthritis, Knee", "phase": 2.0 }, { "disease": "Cystitis, Interstitial", "phase": 2.0 }, { "disease": "Asthma", "phase": 2.0 }, { "disease": "Sleep Apnea, Obstructive", "phase": 1.0 }, { "disease": "Renal Insufficiency", "phase": 1.0 } ], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] }, "us_crl": { "application": "NDA (Merck, chronic cough)", "date": "12/20/2023", "kind": "COMPLETE RESPONSE", "company": "Merck", "source": "Merck press releases 2022-01-24 (first CRL) and 2023-12-20 (second CRL)" } }, { "unii": "7CDW97HUEX", "name": "LINZAGOLIX", "moiety": "LINZAGOLIX", "inn": true, "usan": true, "type": "chemical", "sources": [ "EMA", "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Linzagolix", "linzagolix choline" ], "area": "Leiomyoma", "atc": "H01CC04", "indication": "Yselty is indicated in adult women of reproductive age for: -           treatment of moderate to severe symptoms of uterine fibroids,-           symptomatic treatment of endometriosis in women with a history of previous medical or surgical treatment for their endometriosis (see section 5.1).", "approved": "14/06/2022", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['EXPORT ONLY']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EMA": "authorised", "EU national (Art57)": "authorised" }, "n_countries": 3, "countries": [ "Iceland", "Liechtenstein", "Norway" ], "pubmed": 0, "trials": 18, "us_orphan": "unknown", "chembl": [ { "disease": "Endometriosis", "phase": 3.0 }, { "disease": "Leiomyoma", "phase": 3.0 }, { "disease": "Liver Diseases", "phase": 1.0 }, { "disease": "Kidney Diseases", "phase": 1.0 } ], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] }, "us_crl": { "application": "NDA (ObsEva/Kissei, uterine fibroids)", "date": "08/22/2022", "kind": "NDA WITHDRAWN BEFORE ACTION", "company": "Kissei", "source": "Kissei announcement 2022-08-22; FDA had identified deficiencies" } }, { "unii": "1LM890Q4FY", "name": "MIFAMURTIDE", "moiety": "MIFAMURTIDE", "inn": false, "usan": true, "type": "chemical", "sources": [ "EMA", "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Mifamurtide", "mifamurtide" ], "area": "Osteosarcoma", "atc": "L03AX15", "indication": "Mepact is indicated in children, adolescents and young adults for the treatment of high-grade resectable non-metastatic osteosarcoma after macroscopically complete surgical resection. It is used in combination with postoperative multi-agent chemotherapy. Safety and efficacy have been assessed in studies of patients two to 30 years of age at initial diagnosis.", "approved": "06/03/2009", "flags": [], "foreign_status": "authorised", "status_by_source": { "EMA": "authorised", "EU national (Art57)": "authorised" }, "n_countries": 3, "countries": [ "Iceland", "Liechtenstein", "Norway" ], "pubmed": 16, "trials": 8, "us_orphan": "unknown", "chembl": [ { "disease": "Osteosarcoma", "phase": 4.0 }, { "disease": "Neoplasms", "phase": 4.0 } ], "orpha": { "code": "668", "name": "Osteosarcoma", "rarity": "rare", "rarity_evidence": "Orphanet 1-9 / 100 000 (Europe) \u2248 16,750 in the US, under 200,000", "us_prevalence_est": 16750, "onset": "pediatric", "onset_evidence": "Orphanet age of onset: Childhood \u2014 within birth-18" }, "eu_orphan_designation": { "use": "Treatment of echinococcosis", "status": "Positive", "number": "EU/3/16/1699" }, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "PASS", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "Orphanet 1-9 / 100 000 (Europe) \u2248 16,750 in the US, under 200,000" }, { "key": "primarily_pediatric", "status": "PASS", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "Orphanet age of onset: Childhood \u2014 within birth-18" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] }, "eu_orphan_designations": [ { "use": "Treatment of echinococcosis", "status": "Positive", "number": "EU/3/16/1699" }, { "use": "Treatment of hepatocellular carcinoma", "status": "Positive", "number": "EU/3/16/1700" } ], "us_crl": { "application": "NDA (IDM Pharma, osteosarcoma)", "date": "08/27/2007", "kind": "NOT APPROVABLE", "company": "IDM Pharma", "source": "IDM Pharma press release 2007-08-27 (Junovan)" } }, { "unii": "SE4TWR0K2C", "name": "PERFLUBUTANE", "moiety": "PERFLUBUTANE", "inn": true, "usan": true, "type": "chemical", "sources": [ "EMA", "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Perflubutane", "perflubutane" ], "area": "Ultrasonography", "atc": "V08D", "indication": "ultrasound imaging agent indicated for the detection of coronary artery disease (CAD)", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EMA": "never_authorised", "EU national (Art57)": "authorised" }, "n_countries": 3, "countries": [ "Denmark", "Norway", "Sweden" ], "pubmed": 2, "trials": 20, "us_orphan": "unknown", "chembl": [ { "disease": "Hypertension, Portal", "phase": 2.0 }, { "disease": "Carcinoma, Pancreatic Ductal", "phase": 1.0 } ], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "X3O30D9YMX", "name": "ROXADUSTAT", "moiety": "ROXADUSTAT", "inn": true, "usan": true, "type": "chemical", "sources": [ "EMA", "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Roxadustat", "roxadustat" ], "area": "Anemia;Kidney Failure, Chronic", "atc": "B03XA05", "indication": "Evrenzo is indicated for treatment of adult patients with symptomatic anaemia associated with chronic kidney disease (CKD).", "approved": "18/08/2021", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT', 'EXPORT ONLY']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs.", "US_ORPHAN_DESIGNATION_HELD: Kyntra Bio (fka FibroGen) granted FDA orphan designation for myelodysplastic syndromes 2025-12 (8-K 2026-01-07); Phase 3 in lower-risk MDS planned Q4 2026; CKD-anaemia NDA CRL 2021-08-11 never resubmitted" ], "foreign_status": "authorised", "status_by_source": { "EMA": "authorised", "EU national (Art57)": "authorised" }, "n_countries": 3, "countries": [ "Iceland", "Liechtenstein", "Norway" ], "pubmed": 5, "trials": 66, "us_orphan": "unknown", "chembl": [ { "disease": "Anemia", "phase": 4.0 }, { "disease": "Kidney Failure, Chronic", "phase": 4.0 }, { "disease": "Myelodysplastic Syndromes", "phase": 3.0 }, { "disease": "ST Elevation Myocardial Infarction", "phase": 2.0 }, { "disease": "Kidney Diseases", "phase": 1.0 } ], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] }, "us_crl": { "application": "NDA (FibroGen/AstraZeneca, CKD anaemia)", "date": "08/11/2021", "kind": "COMPLETE RESPONSE", "company": "FibroGen", "source": "FibroGen press release 2021-08-11; AdCom 13-1 against 2021-07-15" } }, { "unii": "8CZ82ZYY9X", "name": "TOLEBRUTINIB", "moiety": "TOLEBRUTINIB", "inn": true, "usan": true, "type": "chemical", "sources": [ "EMA", "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Tolebrutinib", "tolebrutinib" ], "area": "Multiple Sclerosis, Chronic Progressive", "atc": "L04AA62", "indication": "Cenrifki is indicated for the treatment of adult patients with secondary progressive multiple sclerosis (SPMS) without relapses in the last 2 years.", "approved": "19/06/2026", "flags": [], "foreign_status": "authorised", "status_by_source": { "EMA": "authorised", "EU national (Art57)": "authorised" }, "n_countries": 3, "countries": [ "Iceland", "Liechtenstein", "Norway" ], "pubmed": 0, "trials": 12, "us_orphan": "unknown", "chembl": [ { "disease": "Multiple Sclerosis, Chronic Progressive", "phase": 3.0 }, { "disease": "Multiple Sclerosis", "phase": 3.0 }, { "disease": "Kidney Diseases", "phase": 1.0 } ], "orpha": null, "us_crl": { "application": "NDA 219624", "date": "12/23/2025", "kind": "COMPLETE RESPONSE", "approval_status": "Unapproved", "company": "Genzyme Corporation", "letters": 1, "source": "openFDA CRL dataset" }, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "9G468C8B13", "name": "VERNAKALANT", "moiety": "VERNAKALANT", "inn": true, "usan": false, "type": "chemical", "sources": [ "EMA", "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Vernakalant Hydrochloride", "vernakalant hydrochloride" ], "area": "Atrial Fibrillation", "atc": "C01BG11", "indication": "Rapid conversion of recent onset atrial fibrillation to sinus rhythm in adults: for non-surgery patients: atrial fibrillation </= 7 days duration; for post-cardiac surgery patients: atrial fibrillation </= 3 days duration.", "approved": "01/09/2010", "flags": [], "foreign_status": "authorised", "status_by_source": { "EMA": "authorised", "EU national (Art57)": "authorised" }, "n_countries": 3, "countries": [ "Iceland", "Liechtenstein", "Norway" ], "pubmed": 1, "trials": 17, "us_orphan": "unknown", "chembl": [ { "disease": "Arrhythmias, Cardiac", "phase": 4.0 }, { "disease": "Atrial Fibrillation", "phase": 3.0 }, { "disease": "Atrial Flutter", "phase": 2.0 } ], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] }, "us_crl": { "application": "NDA (Correvio, atrial fibrillation)", "date": "12/24/2019", "kind": "COMPLETE RESPONSE", "company": "Correvio", "source": "Correvio press release 2019-12-24; AdCom 11-2 against" } }, { "unii": "5BF646324K", "name": "VINFLUNINE", "moiety": "VINFLUNINE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EMA", "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Vinflunine", "vinflunine" ], "area": "Carcinoma, Transitional Cell;Urologic Neoplasms", "atc": "L01CA05", "indication": "Javlor is indicated in monotherapy for the treatment of adult patients with advanced or metastatic transitional-cell carcinoma of the urothelial tract after failure of a prior platinum-containing regimen. Efficacy and safety of vinflunine have not been studied in patients with performance status ? 2.", "approved": "21/09/2009", "flags": [], "foreign_status": "authorised", "status_by_source": { "EMA": "authorised", "EU national (Art57)": "authorised" }, "n_countries": 3, "countries": [ "Iceland", "Liechtenstein", "Norway" ], "pubmed": 0, "trials": 35, "us_orphan": "unknown", "chembl": [ { "disease": "Neoplasms", "phase": 3.0 }, { "disease": "Urogenital Neoplasms", "phase": 3.0 }, { "disease": "Urinary Bladder Neoplasms", "phase": 3.0 }, { "disease": "Head and Neck Neoplasms", "phase": 3.0 }, { "disease": "Breast Neoplasms", "phase": 3.0 }, { "disease": "Small Cell Lung Carcinoma", "phase": 2.0 }, { "disease": "Carcinoma, Non-Small-Cell Lung", "phase": 2.0 }, { "disease": "Carcinoma, Transitional Cell", "phase": 2.0 }, { "disease": "Penile Neoplasms", "phase": 2.0 }, { "disease": "Stomach Neoplasms", "phase": 2.0 }, { "disease": "Prostatic Neoplasms, Castration-Resistant", "phase": 2.0 }, { "disease": "Lung Neoplasms", "phase": 2.0 } ], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "05W904P57F", "name": "AMYLMETACRESOL", "moiety": "AMYLMETACRESOL", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)", "Health Canada" ], "orphan": false, "tier": "moiety-base", "examples": [ "AMYLMETACRESOL", "Amylmetacresol", "Amylmetacresol Bp" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised", "Health Canada": "authorised" }, "n_countries": 2, "countries": [ "Portugal", "United Kingdom (Northern Ireland)" ], "pubmed": 0, "trials": 1, "us_orphan": "unknown", "chembl": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "WRX4NFY03R", "name": "ETORICOXIB", "moiety": "ETORICOXIB", "inn": true, "usan": true, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Etoricoxib" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 30, "countries": [ "Austria", "Belgium", "Bulgaria", "Croatia", "Cyprus", "Czech Republic", "Denmark", "Estonia", "Finland", "France", "Germany", "Greece", "Hungary", "Iceland", "Ireland", "Italy", "Latvia", "Lithuania", "Luxembourg", "Malta", "Netherlands", "Norway", "Poland", "Portugal", "Romania", "Slovakia", "Slovenia", "Spain", "Sweden", "United Kingdom (Northern Ireland)" ], "pubmed": 12, "trials": 129, "us_orphan": "unknown", "chembl": [ { "disease": "Rheumatic Diseases", "phase": 4.0 }, { "disease": "Pain", "phase": 3.0 }, { "disease": "Arthritis, Rheumatoid", "phase": 3.0 }, { "disease": "Spondylitis, Ankylosing", "phase": 3.0 }, { "disease": "Osteoarthritis, Knee", "phase": 3.0 }, { "disease": "Wounds and Injuries", "phase": 3.0 }, { "disease": "Dysmenorrhea", "phase": 3.0 }, { "disease": "Toothache", "phase": 3.0 }, { "disease": "Low Back Pain", "phase": 3.0 }, { "disease": "Osteoarthritis", "phase": 3.0 }, { "disease": "HIV Infections", "phase": 2.0 }, { "disease": "Bone Neoplasms", "phase": 2.0 }, { "disease": "Prostatic Neoplasms, Castration-Resistant", "phase": 2.0 }, { "disease": "Melanoma", "phase": 1.0 }, { "disease": "Tuberculosis", "phase": 1.0 } ], "disease_terms": "Rheumatic Diseases", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "59XE10C19C", "name": "FUSIDIC ACID", "moiety": "FUSIDIC", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Anhydrous Fusidic Acid", "Fusidic Acid", "Fusidic Acid Hemihydrate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 30, "countries": [ "Austria", "Belgium", "Bulgaria", "Croatia", "Cyprus", "Czech Republic", "Denmark", "Estonia", "Finland", "France", "Germany", "Greece", "Hungary", "Iceland", "Ireland", "Italy", "Latvia", "Lithuania", "Luxembourg", "Malta", "Netherlands", "Norway", "Poland", "Portugal", "Romania", "Slovakia", "Slovenia", "Spain", "Sweden", "United Kingdom (Northern Ireland)" ], "pubmed": 55, "trials": 27, "us_orphan": "unknown", "chembl": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "2AE8M83G3E", "name": "RUPATADINE", "moiety": "RUPATADINE", "inn": true, "usan": true, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Rupatadine", "Rupatadine Fumarate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 30, "countries": [ "Austria", "Belgium", "Bulgaria", "Croatia", "Cyprus", "Czech Republic", "Denmark", "Estonia", "France", "Germany", "Greece", "Hungary", "Iceland", "Ireland", "Italy", "Latvia", "Liechtenstein", "Lithuania", "Luxembourg", "Malta", "Netherlands", "Norway", "Poland", "Portugal", "Romania", "Slovakia", "Slovenia", "Spain", "Sweden", "United Kingdom (Northern Ireland)" ], "pubmed": 7, "trials": 15, "us_orphan": "unknown", "chembl": [ { "disease": "Hypersensitivity", "phase": 4.0 }, { "disease": "Urticaria", "phase": 3.0 }, { "disease": "Arthritis, Rheumatoid", "phase": 3.0 }, { "disease": "Mastocytosis", "phase": 2.0 }, { "disease": "Cold Urticaria", "phase": 2.0 } ], "disease_terms": "Hypersensitivity", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "AB0BHP2FH0", "name": "AMOROLFINE", "moiety": "AMOROLFINE", "inn": true, "usan": true, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Amorolfine", "Amorolfine Hydrochloride" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 29, "countries": [ "Austria", "Belgium", "Bulgaria", "Croatia", "Cyprus", "Czech Republic", "Denmark", "Estonia", "Finland", "France", "Germany", "Greece", "Hungary", "Iceland", "Ireland", "Italy", "Latvia", "Lithuania", "Luxembourg", "Malta", "Norway", "Poland", "Portugal", "Romania", "Slovakia", "Slovenia", "Spain", "Sweden", "United Kingdom (Northern Ireland)" ], "pubmed": 2, "trials": 13, "us_orphan": "unknown", "chembl": [ { "disease": "Onychomycosis", "phase": 3.0 } ], "disease_terms": "Onychomycosis", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "WPY40FTH8K", "name": "XYLOMETAZOLINE", "moiety": "XYLOMETAZOLINE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Xylometazoline Hydrochloride" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT', 'UNAPPROVED DRUG OTHER']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 29, "countries": [ "Austria", "Belgium", "Bulgaria", "Croatia", "Cyprus", "Czech Republic", "Denmark", "Estonia", "Finland", "Germany", "Greece", "Hungary", "Iceland", "Ireland", "Italy", "Latvia", "Lithuania", "Luxembourg", "Malta", "Netherlands", "Norway", "Poland", "Portugal", "Romania", "Slovakia", "Slovenia", "Spain", "Sweden", "United Kingdom (Northern Ireland)" ], "pubmed": 10, "trials": 37, "us_orphan": "unknown", "chembl": [ { "disease": "Eye Manifestations", "phase": 4.0 }, { "disease": "Nasal Obstruction", "phase": 4.0 }, { "disease": "Common Cold", "phase": 3.0 } ], "disease_terms": "Eye Manifestations; Nasal Obstruction", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "4O21U048EF", "name": "BENZYDAMINE", "moiety": "BENZYDAMINE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Benzydamine Hydrochloride" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 28, "countries": [ "Austria", "Belgium", "Bulgaria", "Croatia", "Cyprus", "Czech Republic", "Denmark", "Finland", "France", "Germany", "Greece", "Hungary", "Ireland", "Italy", "Latvia", "Lithuania", "Luxembourg", "Malta", "Netherlands", "Norway", "Poland", "Portugal", "Romania", "Slovakia", "Slovenia", "Spain", "Sweden", "United Kingdom (Northern Ireland)" ], "pubmed": 10, "trials": 49, "us_orphan": "unknown", "chembl": [ { "disease": "Rheumatic Diseases", "phase": 4.0 }, { "disease": "Vaginitis", "phase": 4.0 }, { "disease": "Arthralgia", "phase": 4.0 }, { "disease": "Myalgia", "phase": 4.0 }, { "disease": "Stomatitis", "phase": 3.0 } ], "disease_terms": "Rheumatic Diseases; Vaginitis; Arthralgia; Myalgia", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "Q1J152VB1P", "name": "BROMHEXINE", "moiety": "BROMHEXINE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Bromhexine", "Bromhexine Hydrochloride", "Bromhexine Hydrochloride Bp" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['EXPORT ONLY']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 28, "countries": [ "Belgium", "Bulgaria", "Croatia", "Cyprus", "Czech Republic", "Denmark", "Estonia", "Finland", "France", "Germany", "Greece", "Hungary", "Iceland", "Ireland", "Italy", "Latvia", "Lithuania", "Luxembourg", "Malta", "Netherlands", "Norway", "Poland", "Portugal", "Romania", "Slovakia", "Slovenia", "Spain", "Sweden" ], "pubmed": 10, "trials": 30, "us_orphan": "unknown", "chembl": [ { "disease": "Malaria", "phase": 0.5 } ], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "CC6X0L40GW", "name": "MOXONIDINE", "moiety": "MOXONIDINE", "inn": true, "usan": true, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Moxonidine" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 28, "countries": [ "Austria", "Belgium", "Bulgaria", "Croatia", "Cyprus", "Czech Republic", "Denmark", "Estonia", "Finland", "France", "Germany", "Greece", "Hungary", "Italy", "Latvia", "Lithuania", "Luxembourg", "Malta", "Netherlands", "Norway", "Poland", "Portugal", "Romania", "Slovakia", "Slovenia", "Spain", "Sweden", "United Kingdom (Northern Ireland)" ], "pubmed": 2, "trials": 21, "us_orphan": "unknown", "chembl": [ { "disease": "Hypertension", "phase": 4.0 }, { "disease": "Heart Diseases", "phase": 3.0 }, { "disease": "Hypotension, Orthostatic", "phase": 0.5 } ], "disease_terms": "Hypertension", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "200168S0CL", "name": "AMBROXOL", "moiety": "AMBROXOL", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Ambroxol", "Ambroxol Hydrochloride", "Ambroxol Hydrochloride Ph. Eur." ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 27, "countries": [ "Austria", "Belgium", "Bulgaria", "Croatia", "Cyprus", "Czech Republic", "Denmark", "Estonia", "Finland", "France", "Germany", "Greece", "Hungary", "Iceland", "Italy", "Latvia", "Lithuania", "Luxembourg", "Malta", "Norway", "Poland", "Portugal", "Romania", "Slovakia", "Slovenia", "Spain", "Sweden" ], "pubmed": 40, "trials": 40, "us_orphan": "unknown", "chembl": [ { "disease": "Respiratory Tract Diseases", "phase": 3.0 }, { "disease": "Pharyngitis", "phase": 3.0 }, { "disease": "Parkinson Disease", "phase": 3.0 }, { "disease": "Cough", "phase": 2.0 }, { "disease": "Dementia", "phase": 2.0 }, { "disease": "Gaucher Disease", "phase": 2.0 }, { "disease": "Amyotrophic Lateral Sclerosis", "phase": 2.0 }, { "disease": "Lewy Body Disease", "phase": 1.0 } ], "disease_terms": "Respiratory Tract Diseases; Pharyngitis; Parkinson Disease", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "KZF0XM66JC", "name": "ETOFENAMATE", "moiety": "ETOFENAMATE", "inn": true, "usan": true, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Etofenamate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 27, "countries": [ "Austria", "Belgium", "Bulgaria", "Croatia", "Czech Republic", "Denmark", "Estonia", "Finland", "France", "Germany", "Greece", "Hungary", "Ireland", "Italy", "Latvia", "Lithuania", "Luxembourg", "Malta", "Netherlands", "Norway", "Poland", "Portugal", "Romania", "Slovakia", "Slovenia", "Spain", "Sweden" ], "pubmed": 0, "trials": 2, "us_orphan": "unknown", "chembl": [ { "disease": "Myalgia", "phase": 2.0 }, { "disease": "Arthralgia", "phase": 2.0 } ], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "C6T4514L4E", "name": "LEVOSIMENDAN", "moiety": "LEVOSIMENDAN", "inn": true, "usan": true, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Levosimendan" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "STEREO_NAME: name carries an enantiomer/racemate marker; check the counterpart's US status by hand", "US_SPONSOR_ACTIVE: Tenax Therapeutics TNX-103 (oral levosimendan, adult PH-HFpEF); Phase 3 LEVEL missed its primary endpoint 2026-08-10, LEVEL-2 ongoing; no NDA. A Tenax approval would bar the moiety for any Funnel-A programme" ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 27, "countries": [ "Austria", "Belgium", "Bulgaria", "Croatia", "Cyprus", "Czech Republic", "Denmark", "Estonia", "Finland", "France", "Germany", "Greece", "Hungary", "Iceland", "Ireland", "Italy", "Latvia", "Lithuania", "Netherlands", "Norway", "Poland", "Portugal", "Romania", "Slovakia", "Slovenia", "Spain", "Sweden" ], "pubmed": 117, "trials": 118, "us_orphan": "unknown", "chembl": [ { "disease": "Heart Failure", "phase": 3.0 }, { "disease": "ST Elevation Myocardial Infarction", "phase": 3.0 }, { "disease": "Cardiovascular Diseases", "phase": 3.0 }, { "disease": "Shock", "phase": 3.0 }, { "disease": "Amyotrophic Lateral Sclerosis", "phase": 3.0 }, { "disease": "Shock, Septic", "phase": 2.0 }, { "disease": "Hernias, Diaphragmatic, Congenital", "phase": 2.0 }, { "disease": "Acute Kidney Injury", "phase": 2.0 }, { "disease": "Muscular Diseases", "phase": 2.0 }, { "disease": "Stroke", "phase": 2.0 }, { "disease": "Coronary Disease", "phase": 2.0 }, { "disease": "Ischemic Attack, Transient", "phase": 2.0 }, { "disease": "Hemorrhage", "phase": 2.0 }, { "disease": "Heart Diseases", "phase": 2.0 } ], "disease_terms": "Heart Failure; ST Elevation Myocardial Infarction; Cardiovascular Diseases; Shock; Amyotrophic Lateral Sclerosis", "orpha": null, "eu_orphan_designation": { "use": "Treatment of amyotrophic lateral sclerosis", "status": "Withdrawn", "number": "EU/3/18/1980" }, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] }, "eu_orphan_designations": [ { "use": "Treatment of amyotrophic lateral sclerosis", "status": "Withdrawn", "number": "EU/3/18/1980" } ] }, { "unii": "E61Q31EK2F", "name": "CYPROTERONE", "moiety": "CYPROTERONE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Cyproterone Acetate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 26, "countries": [ "Austria", "Belgium", "Bulgaria", "Croatia", "Czech Republic", "Denmark", "Estonia", "France", "Germany", "Greece", "Hungary", "Ireland", "Italy", "Latvia", "Lithuania", "Luxembourg", "Netherlands", "Norway", "Poland", "Portugal", "Romania", "Slovakia", "Slovenia", "Spain", "Sweden", "United Kingdom (Northern Ireland)" ], "pubmed": 33, "trials": 66, "us_orphan": "unknown", "chembl": [ { "disease": "Polycystic Ovary Syndrome", "phase": 0.5 } ], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "GB2433A4M3", "name": "DAPOXETINE", "moiety": "DAPOXETINE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Dapoxetine" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 26, "countries": [ "Austria", "Belgium", "Bulgaria", "Croatia", "Cyprus", "Czech Republic", "Estonia", "Finland", "France", "Germany", "Greece", "Hungary", "Ireland", "Italy", "Lithuania", "Luxembourg", "Malta", "Netherlands", "Poland", "Portugal", "Romania", "Slovakia", "Slovenia", "Spain", "Sweden", "United Kingdom (Northern Ireland)" ], "pubmed": 1, "trials": 33, "us_orphan": "unknown", "chembl": [ { "disease": "Erectile Dysfunction", "phase": 3.0 }, { "disease": "Sexual Dysfunction, Physiological", "phase": 3.0 }, { "disease": "Premature Ejaculation", "phase": 3.0 } ], "disease_terms": "Erectile Dysfunction; Sexual Dysfunction, Physiological; Premature Ejaculation", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "7F80CC3NNV", "name": "MEBEVERINE", "moiety": "MEBEVERINE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Mebeverine", "Mebeverine Hydrochloride", "Mebeverine Hydrochloride Bp" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 26, "countries": [ "Austria", "Belgium", "Bulgaria", "Croatia", "Cyprus", "Czech Republic", "Denmark", "Estonia", "France", "Germany", "Greece", "Iceland", "Ireland", "Italy", "Latvia", "Lithuania", "Luxembourg", "Malta", "Netherlands", "Poland", "Portugal", "Romania", "Slovakia", "Slovenia", "Spain", "United Kingdom (Northern Ireland)" ], "pubmed": 2, "trials": 20, "us_orphan": "unknown", "chembl": [ { "disease": "Digestive System Diseases", "phase": 4.0 }, { "disease": "Irritable Bowel Syndrome", "phase": 3.0 } ], "disease_terms": "Digestive System Diseases", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "76K53XP4TO", "name": "RACECADOTRIL", "moiety": "RACECADOTRIL", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Racecadotril" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "STEREO_NAME: name carries an enantiomer/racemate marker; check the counterpart's US status by hand" ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 26, "countries": [ "Austria", "Belgium", "Bulgaria", "Croatia", "Czech Republic", "Denmark", "Estonia", "Finland", "France", "Germany", "Greece", "Hungary", "Ireland", "Italy", "Latvia", "Lithuania", "Luxembourg", "Netherlands", "Poland", "Portugal", "Romania", "Slovakia", "Slovenia", "Spain", "Sweden", "United Kingdom (Northern Ireland)" ], "pubmed": 23, "trials": 15, "us_orphan": "unknown", "chembl": [ { "disease": "Diarrhea", "phase": 3.0 }, { "disease": "Diarrhea, Infantile", "phase": 3.0 } ], "disease_terms": "Diarrhea; Diarrhea, Infantile", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "FF9X0205V2", "name": "TIBOLONE", "moiety": "TIBOLONE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Tibolone" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 26, "countries": [ "Austria", "Belgium", "Bulgaria", "Cyprus", "Czech Republic", "Denmark", "Finland", "France", "Germany", "Greece", "Hungary", "Iceland", "Ireland", "Italy", "Lithuania", "Luxembourg", "Netherlands", "Norway", "Poland", "Portugal", "Romania", "Slovakia", "Slovenia", "Spain", "Sweden", "United Kingdom (Northern Ireland)" ], "pubmed": 2, "trials": 28, "us_orphan": "unknown", "chembl": [ { "disease": "Osteoporosis", "phase": 3.0 }, { "disease": "Sexual Dysfunction, Physiological", "phase": 3.0 }, { "disease": "Breast Neoplasms", "phase": 3.0 }, { "disease": "Menopause", "phase": 3.0 }, { "disease": "Depressive Disorder, Major", "phase": 1.0 } ], "disease_terms": "Osteoporosis; Sexual Dysfunction, Physiological; Breast Neoplasms; Menopause", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "53S5U404NU", "name": "CYTISINICLINE", "moiety": "CYTISINICLINE", "inn": true, "usan": true, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Cytisinicline" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 25, "countries": [ "Austria", "Belgium", "Bulgaria", "Cyprus", "Czech Republic", "Denmark", "Finland", "France", "Germany", "Greece", "Hungary", "Ireland", "Italy", "Latvia", "Lithuania", "Malta", "Netherlands", "Norway", "Poland", "Portugal", "Romania", "Slovakia", "Spain", "Sweden", "United Kingdom (Northern Ireland)" ], "pubmed": 0, "trials": 34, "us_orphan": "unknown", "chembl": [ { "disease": "Tobacco Use Disorder", "phase": 3.0 }, { "disease": "Smoking Cessation", "phase": 3.0 }, { "disease": "Kidney Diseases", "phase": 1.0 } ], "disease_terms": "Tobacco Use Disorder; Smoking Cessation", "orpha": null, "us_crl": { "application": "NDA 218995", "date": "06/20/2026", "kind": "COMPLETE RESPONSE", "approval_status": "Unapproved", "company": "Achieve Life Sciences, Inc.", "letters": 1, "source": "openFDA CRL dataset" }, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "QG05NRB077", "name": "FENTICONAZOLE", "moiety": "FENTICONAZOLE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Fenticonazole Nitrate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 25, "countries": [ "Austria", "Belgium", "Bulgaria", "Croatia", "Cyprus", "Czech Republic", "Denmark", "Estonia", "France", "Germany", "Greece", "Hungary", "Ireland", "Italy", "Latvia", "Lithuania", "Luxembourg", "Netherlands", "Poland", "Portugal", "Romania", "Slovakia", "Slovenia", "Spain", "United Kingdom (Northern Ireland)" ], "pubmed": 0, "trials": 2, "us_orphan": "unknown", "chembl": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "ZH516LNZ10", "name": "PIRACETAM", "moiety": "PIRACETAM", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Piracetam" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 24, "countries": [ "Belgium", "Bulgaria", "Cyprus", "Czech Republic", "Finland", "France", "Germany", "Greece", "Hungary", "Italy", "Latvia", "Lithuania", "Luxembourg", "Malta", "Netherlands", "Norway", "Poland", "Portugal", "Romania", "Slovakia", "Slovenia", "Spain", "Sweden", "United Kingdom (Northern Ireland)" ], "pubmed": 17, "trials": 16, "us_orphan": "unknown", "chembl": [ { "disease": "Attention Deficit Disorder with Hyperactivity", "phase": 4.0 }, { "disease": "Movement Disorders", "phase": 3.0 }, { "disease": "Anxiety", "phase": 3.0 }, { "disease": "Postpoliomyelitis Syndrome", "phase": 3.0 }, { "disease": "Dementia", "phase": 3.0 }, { "disease": "Depressive Disorder", "phase": 3.0 }, { "disease": "Schizophrenia", "phase": 3.0 }, { "disease": "Cocaine-Related Disorders", "phase": 1.0 } ], "disease_terms": "Attention Deficit Disorder with Hyperactivity", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "88TWF8015Y", "name": "CARBETOCIN", "moiety": "CARBETOCIN", "inn": true, "usan": true, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Carbetocin" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 23, "countries": [ "Austria", "Belgium", "Bulgaria", "Czech Republic", "Denmark", "Estonia", "Finland", "France", "Germany", "Greece", "Ireland", "Italy", "Latvia", "Lithuania", "Luxembourg", "Netherlands", "Norway", "Poland", "Portugal", "Slovakia", "Spain", "Sweden", "United Kingdom (Northern Ireland)" ], "pubmed": 2, "trials": 103, "us_orphan": "unknown", "chembl": [ { "disease": "Postpartum Hemorrhage", "phase": 3.0 }, { "disease": "Prader-Willi Syndrome", "phase": 3.0 }, { "disease": "Uterine Inertia", "phase": 1.0 }, { "disease": "Leiomyoma", "phase": 1.0 } ], "disease_terms": "Postpartum Hemorrhage; Prader-Willi Syndrome", "orpha": null, "eu_orphan_designation": { "use": "Treatment of Prader-Willi syndrome", "status": "Withdrawn", "number": "EU/3/12/975" }, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] }, "eu_orphan_designations": [ { "use": "Treatment of Prader-Willi syndrome", "status": "Withdrawn", "number": "EU/3/12/975" } ] }, { "unii": "5587267Z69", "name": "DOMPERIDONE", "moiety": "DOMPERIDONE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Domperidone", "Domperidone Maleate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 23, "countries": [ "Austria", "Belgium", "Bulgaria", "Croatia", "Cyprus", "Denmark", "France", "Germany", "Greece", "Hungary", "Ireland", "Italy", "Liechtenstein", "Lithuania", "Luxembourg", "Malta", "Netherlands", "Portugal", "Romania", "Slovakia", "Spain", "Sweden", "United Kingdom (Northern Ireland)" ], "pubmed": 49, "trials": 83, "us_orphan": "unknown", "chembl": [ { "disease": "Digestive System Diseases", "phase": 4.0 }, { "disease": "Gastroparesis", "phase": 3.0 }, { "disease": "Gastroenteritis", "phase": 3.0 }, { "disease": "Gastroesophageal Reflux", "phase": 3.0 }, { "disease": "Dyspepsia", "phase": 3.0 }, { "disease": "Multiple Sclerosis, Relapsing-Remitting", "phase": 2.0 }, { "disease": "Multiple Sclerosis, Chronic Progressive", "phase": 2.0 }, { "disease": "Premature Birth", "phase": 2.0 }, { "disease": "Parkinson Disease", "phase": 2.0 } ], "disease_terms": "Digestive System Diseases", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "43B2M34G2V", "name": "FLUCLOXACILLIN", "moiety": "FLUCLOXACILLIN", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Flucloxacillin", "Flucloxacillin Magnesium Octahydrate", "Flucloxacillin Sodium" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 23, "countries": [ "Austria", "Belgium", "Croatia", "Czech Republic", "Denmark", "Estonia", "Finland", "France", "Germany", "Greece", "Hungary", "Iceland", "Ireland", "Italy", "Luxembourg", "Malta", "Netherlands", "Norway", "Portugal", "Slovakia", "Slovenia", "Sweden", "United Kingdom (Northern Ireland)" ], "pubmed": 62, "trials": 33, "us_orphan": "unknown", "chembl": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "81BMQ80QRL", "name": "ITOPRIDE", "moiety": "ITOPRIDE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Itopride Hydrochloride" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 23, "countries": [ "Austria", "Belgium", "Bulgaria", "Croatia", "Cyprus", "Czech Republic", "Denmark", "Estonia", "Finland", "Greece", "Hungary", "Ireland", "Italy", "Latvia", "Lithuania", "Luxembourg", "Malta", "Poland", "Portugal", "Romania", "Slovakia", "Spain", "United Kingdom (Northern Ireland)" ], "pubmed": 0, "trials": 33, "us_orphan": "unknown", "chembl": [ { "disease": "Dyspepsia", "phase": 3.0 }, { "disease": "Peritonitis", "phase": 3.0 }, { "disease": "Digestive System Diseases", "phase": 3.0 }, { "disease": "Irritable Bowel Syndrome", "phase": 2.0 }, { "disease": "Constipation", "phase": 2.0 }, { "disease": "Gastroparesis", "phase": 1.0 } ], "disease_terms": "Dyspepsia; Peritonitis; Digestive System Diseases", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "RPK779R03H", "name": "ACECLOFENAC", "moiety": "ACECLOFENAC", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Aceclofenac" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 22, "countries": [ "Belgium", "Bulgaria", "Cyprus", "Czech Republic", "Estonia", "France", "Germany", "Greece", "Hungary", "Ireland", "Italy", "Latvia", "Lithuania", "Luxembourg", "Netherlands", "Poland", "Portugal", "Romania", "Slovakia", "Spain", "Sweden", "United Kingdom (Northern Ireland)" ], "pubmed": 3, "trials": 22, "us_orphan": "unknown", "chembl": [ { "disease": "Rheumatic Diseases", "phase": 4.0 }, { "disease": "Myalgia", "phase": 4.0 }, { "disease": "Arthralgia", "phase": 4.0 }, { "disease": "Arthritis, Rheumatoid", "phase": 4.0 }, { "disease": "Spondylitis", "phase": 4.0 }, { "disease": "Osteoarthritis", "phase": 4.0 }, { "disease": "Pulpitis", "phase": 2.0 } ], "disease_terms": "Rheumatic Diseases; Myalgia; Arthralgia; Arthritis, Rheumatoid; Spondylitis; Osteoarthritis", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "N9A0A0R9S8", "name": "TRIMETAZIDINE", "moiety": "TRIMETAZIDINE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Trimetazidine", "Trimetazidine Dihydrochloride" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 22, "countries": [ "Austria", "Bulgaria", "Croatia", "Cyprus", "Czech Republic", "Denmark", "Estonia", "France", "Greece", "Hungary", "Ireland", "Italy", "Latvia", "Lithuania", "Luxembourg", "Malta", "Poland", "Portugal", "Romania", "Slovakia", "Slovenia", "Spain" ], "pubmed": 3, "trials": 40, "us_orphan": "unknown", "chembl": [ { "disease": "Cardiovascular Diseases", "phase": 4.0 }, { "disease": "Cardiomyopathy, Hypertrophic", "phase": 2.0 }, { "disease": "Hypertension, Pulmonary", "phase": 2.0 }, { "disease": "Cardiotoxicity", "phase": 2.0 }, { "disease": "Diabetic Nephropathies", "phase": 2.0 }, { "disease": "Non-alcoholic Fatty Liver Disease", "phase": 2.0 }, { "disease": "Heart Failure", "phase": 1.0 }, { "disease": "Acute-On-Chronic Liver Failure", "phase": 1.0 }, { "disease": "Peripheral Nervous System Diseases", "phase": 1.0 } ], "disease_terms": "Cardiovascular Diseases", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "250PJI13LM", "name": "MIANSERIN", "moiety": "MIANSERIN", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Mianserin", "Mianserin Hydrochloride" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 21, "countries": [ "Austria", "Belgium", "Cyprus", "Czech Republic", "Denmark", "Finland", "France", "Germany", "Hungary", "Iceland", "Italy", "Luxembourg", "Malta", "Netherlands", "Norway", "Poland", "Portugal", "Slovakia", "Spain", "Sweden", "United Kingdom (Northern Ireland)" ], "pubmed": 3, "trials": 5, "us_orphan": "unknown", "chembl": [ { "disease": "Depressive Disorder", "phase": 4.0 }, { "disease": "Anxiety", "phase": 3.0 }, { "disease": "Dementia", "phase": 3.0 }, { "disease": "Schizophrenia", "phase": 3.0 } ], "disease_terms": "Depressive Disorder", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "740J2QX53R", "name": "CARBOCYSTEINE", "moiety": "CARBOCISTEINE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Carbocisteine", "Carbocisteine Lysine", "Carbocisteine Sodium" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT', 'BULK INGREDIENT FOR HUMAN PRESCRIPTION COMPOUNDING']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 20, "countries": [ "Belgium", "Bulgaria", "Croatia", "Cyprus", "Estonia", "France", "Greece", "Hungary", "Ireland", "Italy", "Latvia", "Lithuania", "Luxembourg", "Malta", "Netherlands", "Poland", "Portugal", "Romania", "Spain", "United Kingdom (Northern Ireland)" ], "pubmed": 3, "trials": 10, "us_orphan": "unknown", "chembl": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "182ECH14UH", "name": "IOBITRIDOL", "moiety": "IOBITRIDOL", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Iobitridol" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 20, "countries": [ "Austria", "Belgium", "Croatia", "Czech Republic", "Denmark", "France", "Germany", "Greece", "Hungary", "Ireland", "Italy", "Luxembourg", "Netherlands", "Norway", "Portugal", "Romania", "Slovenia", "Spain", "Sweden", "United Kingdom (Northern Ireland)" ], "pubmed": 5, "trials": 9, "us_orphan": "unknown", "chembl": [ { "disease": "Acute Kidney Injury", "phase": 0.5 } ], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "9CLV70W7HS", "name": "NITRAZEPAM", "moiety": "NITRAZEPAM", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Nitrazepam", "Nitrazepam Bp" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 20, "countries": [ "Austria", "Belgium", "Croatia", "Cyprus", "Denmark", "Estonia", "Finland", "France", "Germany", "Hungary", "Italy", "Lithuania", "Luxembourg", "Malta", "Netherlands", "Norway", "Poland", "Romania", "Slovenia", "United Kingdom (Northern Ireland)" ], "pubmed": 20, "trials": 12, "us_orphan": "unknown", "chembl": [ { "disease": "Anxiety", "phase": 3.0 }, { "disease": "Dementia", "phase": 3.0 }, { "disease": "Depressive Disorder", "phase": 3.0 }, { "disease": "Sleep Wake Disorders", "phase": 3.0 }, { "disease": "Schizophrenia", "phase": 3.0 }, { "disease": "Epilepsy", "phase": 2.0 } ], "disease_terms": "Anxiety; Dementia; Depressive Disorder; Sleep Wake Disorders; Schizophrenia", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "GVV4Z879SP", "name": "SERTINDOLE", "moiety": "SERTINDOLE", "inn": true, "usan": true, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Sertindole" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 20, "countries": [ "Austria", "Belgium", "Bulgaria", "Croatia", "Czech Republic", "Denmark", "Estonia", "Finland", "Germany", "Greece", "Hungary", "Iceland", "Latvia", "Netherlands", "Norway", "Poland", "Romania", "Slovakia", "Spain", "Sweden" ], "pubmed": 9, "trials": 17, "us_orphan": "unknown", "chembl": [ { "disease": "Psychotic Disorders", "phase": 4.0 }, { "disease": "Anxiety", "phase": 3.0 }, { "disease": "Dementia", "phase": 3.0 }, { "disease": "Depressive Disorder", "phase": 3.0 }, { "disease": "Schizophrenia", "phase": 3.0 }, { "disease": "Cognitive Dysfunction", "phase": 3.0 } ], "disease_terms": "Psychotic Disorders", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "I00Q766CZ2", "name": "SULTHIAME", "moiety": "SULTIAME", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Sultiame" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 20, "countries": [ "Austria", "Belgium", "Czech Republic", "Denmark", "Estonia", "Finland", "Germany", "Hungary", "Ireland", "Italy", "Luxembourg", "Netherlands", "Norway", "Poland", "Portugal", "Romania", "Slovakia", "Spain", "Sweden", "United Kingdom (Northern Ireland)" ], "pubmed": 0, "trials": 5, "us_orphan": "unknown", "chembl": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "7QM776WJ5N", "name": "DIOSMIN", "moiety": "DIOSMIN", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Diosmin" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['UNAPPROVED DRUG OTHER']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 19, "countries": [ "Austria", "Belgium", "Bulgaria", "Croatia", "Czech Republic", "Estonia", "France", "Greece", "Hungary", "Italy", "Latvia", "Lithuania", "Malta", "Poland", "Portugal", "Romania", "Slovakia", "Slovenia", "Spain" ], "pubmed": 1, "trials": 55, "us_orphan": "unknown", "chembl": [ { "disease": "Cardiovascular Diseases", "phase": 4.0 }, { "disease": "Venous Insufficiency", "phase": 3.0 }, { "disease": "Arthritis, Rheumatoid", "phase": 3.0 }, { "disease": "Colitis, Ulcerative", "phase": 3.0 }, { "disease": "Peptic Ulcer", "phase": 3.0 }, { "disease": "Non-alcoholic Fatty Liver Disease", "phase": 3.0 }, { "disease": "Infertility", "phase": 2.0 }, { "disease": "Menorrhagia", "phase": 1.0 } ], "disease_terms": "Cardiovascular Diseases", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "TQD7Q784P1", "name": "EBASTINE", "moiety": "EBASTINE", "inn": true, "usan": true, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Ebastine" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 19, "countries": [ "Belgium", "Cyprus", "Denmark", "Estonia", "Finland", "France", "Germany", "Greece", "Iceland", "Italy", "Latvia", "Lithuania", "Luxembourg", "Netherlands", "Norway", "Poland", "Portugal", "Spain", "Sweden" ], "pubmed": 4, "trials": 10, "us_orphan": "unknown", "chembl": [ { "disease": "Hypersensitivity", "phase": 4.0 }, { "disease": "Irritable Bowel Syndrome", "phase": 3.0 } ], "disease_terms": "Hypersensitivity", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "290ZY759PI", "name": "ZOFENOPRIL", "moiety": "ZOFENOPRIL", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Zofenopril Calcium" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 19, "countries": [ "Belgium", "Bulgaria", "Croatia", "Estonia", "France", "Germany", "Greece", "Hungary", "Ireland", "Italy", "Latvia", "Lithuania", "Luxembourg", "Netherlands", "Poland", "Portugal", "Romania", "Slovakia", "Slovenia" ], "pubmed": 1, "trials": 6, "us_orphan": "unknown", "chembl": [ { "disease": "Cardiovascular Diseases", "phase": 2.0 } ], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "852A84Y8LS", "name": "HEXETIDINE", "moiety": "HEXETIDINE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Hexetidine" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 18, "countries": [ "Austria", "Belgium", "Croatia", "Cyprus", "Czech Republic", "Estonia", "France", "Germany", "Greece", "Ireland", "Latvia", "Luxembourg", "Malta", "Netherlands", "Portugal", "Romania", "Slovakia", "United Kingdom (Northern Ireland)" ], "pubmed": 0, "trials": 5, "us_orphan": "unknown", "chembl": [ { "disease": "Infections", "phase": 4.0 } ], "disease_terms": "Infections", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "260080034N", "name": "LACIDIPINE", "moiety": "LACIDIPINE", "inn": true, "usan": true, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Lacidipine" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 18, "countries": [ "Bulgaria", "Croatia", "Czech Republic", "Denmark", "Estonia", "Greece", "Hungary", "Italy", "Latvia", "Lithuania", "Malta", "Netherlands", "Poland", "Portugal", "Slovakia", "Slovenia", "Spain", "United Kingdom (Northern Ireland)" ], "pubmed": 1, "trials": 15, "us_orphan": "unknown", "chembl": [ { "disease": "Cardiovascular Diseases", "phase": 4.0 }, { "disease": "Hypertension", "phase": 3.0 }, { "disease": "Angina, Stable", "phase": 2.0 } ], "disease_terms": "Cardiovascular Diseases", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "A78GF17HJS", "name": "URAPIDIL", "moiety": "URAPIDIL", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Urapidil", "Urapidil Hydrochloride" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 18, "countries": [ "Austria", "Belgium", "Croatia", "Czech Republic", "Estonia", "France", "Germany", "Hungary", "Italy", "Latvia", "Luxembourg", "Netherlands", "Poland", "Portugal", "Romania", "Slovakia", "Slovenia", "Spain" ], "pubmed": 2, "trials": 32, "us_orphan": "unknown", "chembl": [ { "disease": "Hypertension", "phase": 3.0 }, { "disease": "Pre-Eclampsia", "phase": 3.0 }, { "disease": "Stroke", "phase": 3.0 }, { "disease": "Cerebrovascular Disorders", "phase": 3.0 } ], "disease_terms": "Hypertension; Pre-Eclampsia; Stroke; Cerebrovascular Disorders", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "M75MZG2236", "name": "BUTAMIRATE", "moiety": "BUTAMIRATE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Butamirate Citrate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 17, "countries": [ "Belgium", "Bulgaria", "Croatia", "Cyprus", "Czech Republic", "Denmark", "Estonia", "Greece", "Hungary", "Italy", "Latvia", "Luxembourg", "Malta", "Poland", "Romania", "Slovakia", "Slovenia" ], "pubmed": 2, "trials": 2, "us_orphan": "unknown", "chembl": [ { "disease": "Cough", "phase": 2.0 } ], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "3483C2H13H", "name": "HEXAMIDINE", "moiety": "HEXAMIDINE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Hexamidine Diisetionate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['OTC MONOGRAPH DRUG']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 17, "countries": [ "Belgium", "Bulgaria", "Czech Republic", "Denmark", "Finland", "France", "Germany", "Greece", "Iceland", "Italy", "Luxembourg", "Norway", "Poland", "Portugal", "Romania", "Slovakia", "Sweden" ], "pubmed": 1, "trials": 2, "us_orphan": "unknown", "chembl": [ { "disease": "Nasal Obstruction", "phase": 4.0 }, { "disease": "Eye Infections", "phase": 4.0 } ], "disease_terms": "Nasal Obstruction; Eye Infections", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "76J0853EKA", "name": "ERDOSTEINE", "moiety": "ERDOSTEINE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Erdosteine" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 16, "countries": [ "Austria", "Belgium", "Bulgaria", "Czech Republic", "Denmark", "Finland", "Greece", "Hungary", "Ireland", "Italy", "Luxembourg", "Poland", "Portugal", "Romania", "Slovakia", "United Kingdom (Northern Ireland)" ], "pubmed": 5, "trials": 10, "us_orphan": "unknown", "chembl": [ { "disease": "Pulmonary Disease, Chronic Obstructive", "phase": 3.0 }, { "disease": "Bronchitis, Chronic", "phase": 2.0 } ], "disease_terms": "Pulmonary Disease, Chronic Obstructive", "orpha": null, "eu_orphan_designation": { "use": "Treatment of lead toxicity", "status": "Positive", "number": "EU/3/12/1084" }, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] }, "eu_orphan_designations": [ { "use": "Treatment of lead toxicity", "status": "Positive", "number": "EU/3/12/1084" }, { "use": "Treatment of mercury toxicity", "status": "Positive", "number": "EU/3/12/1067" } ] }, { "unii": "73H7UDN6EC", "name": "MELDONIUM", "moiety": "MELDONIUM", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Meldonium", "Meldonium Dihydrate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 16, "countries": [ "Austria", "Bulgaria", "Croatia", "Cyprus", "Finland", "Ireland", "Italy", "Latvia", "Lithuania", "Luxembourg", "Malta", "Norway", "Poland", "Romania", "Slovakia", "Slovenia" ], "pubmed": 1, "trials": 7, "us_orphan": "unknown", "chembl": [ { "disease": "Cardiovascular Diseases", "phase": 2.0 }, { "disease": "Ischemic Stroke", "phase": 2.0 } ], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "PJ0Y7AZB63", "name": "MOCLOBEMIDE", "moiety": "MOCLOBEMIDE", "inn": true, "usan": true, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Moclobemide" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 16, "countries": [ "Austria", "Belgium", "Czech Republic", "Finland", "France", "Germany", "Hungary", "Ireland", "Netherlands", "Norway", "Poland", "Portugal", "Slovenia", "Spain", "Sweden", "United Kingdom (Northern Ireland)" ], "pubmed": 2, "trials": 9, "us_orphan": "unknown", "chembl": [ { "disease": "Depressive Disorder", "phase": 4.0 }, { "disease": "Anxiety", "phase": 3.0 }, { "disease": "Dementia", "phase": 3.0 }, { "disease": "Schizophrenia", "phase": 3.0 }, { "disease": "Tobacco Use Disorder", "phase": 2.0 }, { "disease": "Substance-Related Disorders", "phase": 2.0 } ], "disease_terms": "Depressive Disorder", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "SLE31693IV", "name": "TRAMAZOLINE", "moiety": "TRAMAZOLINE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Tramazoline Hydrochloride", "Tramazoline Hydrochloride Monohydrate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 16, "countries": [ "Austria", "Belgium", "Bulgaria", "Cyprus", "Czech Republic", "Finland", "Germany", "Greece", "Hungary", "Italy", "Luxembourg", "Netherlands", "Portugal", "Romania", "Slovakia", "Spain" ], "pubmed": 0, "trials": 2, "us_orphan": "unknown", "chembl": [ { "disease": "Nasal Obstruction", "phase": 2.0 } ], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "Y9449Q51XH", "name": "BEZAFIBRATE", "moiety": "BEZAFIBRATE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Bezafibrate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 15, "countries": [ "Austria", "Belgium", "Cyprus", "Finland", "France", "Germany", "Hungary", "Italy", "Luxembourg", "Netherlands", "Portugal", "Romania", "Spain", "Sweden", "United Kingdom (Northern Ireland)" ], "pubmed": 10, "trials": 27, "us_orphan": "unknown", "chembl": [ { "disease": "Cardiovascular Diseases", "phase": 4.0 }, { "disease": "Hyperlipidemia, Familial Combined", "phase": 3.0 }, { "disease": "Liver Cirrhosis, Biliary", "phase": 3.0 }, { "disease": "Cholangitis, Sclerosing", "phase": 3.0 }, { "disease": "Myelodysplastic Syndromes", "phase": 2.0 }, { "disease": "Diabetes Mellitus, Type 2", "phase": 2.0 }, { "disease": "Mitochondrial Diseases", "phase": 2.0 }, { "disease": "Bipolar Disorder", "phase": 2.0 } ], "disease_terms": "Cardiovascular Diseases", "orpha": null, "eu_orphan_designation": { "use": "Treatment of primary sclerosing cholangitis", "status": "Positive", "number": "EU/3/22/2675" }, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] }, "eu_orphan_designations": [ { "use": "Treatment of primary sclerosing cholangitis", "status": "Positive", "number": "EU/3/22/2675" } ] }, { "unii": "OZE0372S5A", "name": "OCTENIDINE", "moiety": "OCTENIDINE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Octenidine Dihydrochloride" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 15, "countries": [ "Austria", "Belgium", "Bulgaria", "Croatia", "Czech Republic", "Estonia", "Germany", "Hungary", "Latvia", "Lithuania", "Luxembourg", "Poland", "Romania", "Slovakia", "Slovenia" ], "pubmed": 2, "trials": 29, "us_orphan": "unknown", "chembl": [ { "disease": "Infections", "phase": 2.0 } ], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "21KOF230FA", "name": "ROXITHROMYCIN", "moiety": "ROXITHROMYCIN", "inn": true, "usan": true, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Roxithromycin" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 15, "countries": [ "Austria", "Bulgaria", "Cyprus", "Czech Republic", "Denmark", "Finland", "France", "Germany", "Greece", "Hungary", "Italy", "Malta", "Poland", "Spain", "Sweden" ], "pubmed": 31, "trials": 11, "us_orphan": "unknown", "chembl": [ { "disease": "Bacterial Infections", "phase": 4.0 } ], "disease_terms": "Bacterial Infections", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "6E20216Q0L", "name": "SOMATOSTATIN", "moiety": "SOMATOSTATIN", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Somatostatin", "Somatostatin Acetate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 15, "countries": [ "Austria", "Belgium", "Bulgaria", "Cyprus", "Czech Republic", "France", "Germany", "Greece", "Italy", "Luxembourg", "Poland", "Portugal", "Romania", "Slovenia", "Spain" ], "pubmed": 503, "trials": 359, "us_orphan": "unknown", "chembl": [ { "disease": "Carcinoma, Hepatocellular", "phase": 3.0 }, { "disease": "Hypertension, Portal", "phase": 3.0 }, { "disease": "Gallbladder Neoplasms", "phase": 3.0 }, { "disease": "Neuroendocrine Tumors", "phase": 3.0 }, { "disease": "Polycystic Kidney, Autosomal Dominant", "phase": 3.0 }, { "disease": "Pancreatitis", "phase": 3.0 }, { "disease": "Liver Failure", "phase": 3.0 }, { "disease": "Carcinoid Tumor", "phase": 2.0 }, { "disease": "Peptic Ulcer", "phase": 2.0 }, { "disease": "Diabetes Mellitus, Type 1", "phase": 2.0 }, { "disease": "Digestive System Diseases", "phase": 2.0 }, { "disease": "Prostatic Neoplasms", "phase": 1.0 }, { "disease": "Hypoglycemia", "phase": 1.0 } ], "disease_terms": "Carcinoma, Hepatocellular; Hypertension, Portal; Gallbladder Neoplasms; Neuroendocrine Tumors; Polycystic Kidney, Autosomal Dominant; Pancreatitis", "orpha": null, "us_crl": { "application": "NDA 208232", "date": "04/15/2016", "kind": "COMPLETE RESPONSE", "approval_status": "Approved", "company": "Camargo Pharmaceutical Services", "letters": 1, "source": "openFDA CRL dataset" }, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "LAH70H9JPH", "name": "TIAPRIDE", "moiety": "TIAPRIDE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Tiapride", "Tiapride Hydrochloride" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 15, "countries": [ "Austria", "Belgium", "Bulgaria", "Czech Republic", "France", "Germany", "Hungary", "Italy", "Lithuania", "Luxembourg", "Poland", "Portugal", "Romania", "Slovakia", "Spain" ], "pubmed": 11, "trials": 5, "us_orphan": "unknown", "chembl": [ { "disease": "Anxiety", "phase": 3.0 }, { "disease": "Dementia", "phase": 3.0 }, { "disease": "Tourette Syndrome", "phase": 3.0 }, { "disease": "Psychotic Disorders", "phase": 3.0 }, { "disease": "Depressive Disorder", "phase": 3.0 }, { "disease": "Schizophrenia", "phase": 3.0 }, { "disease": "Tic Disorders", "phase": 3.0 } ], "disease_terms": "Anxiety; Dementia; Tourette Syndrome; Psychotic Disorders; Depressive Disorder; Schizophrenia", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "Y92OUS2H9B", "name": "BENFOTIAMINE", "moiety": "BENFOTIAMINE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Benfotiamine" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 14, "countries": [ "Austria", "Bulgaria", "Czech Republic", "Estonia", "Germany", "Hungary", "Latvia", "Lithuania", "Poland", "Portugal", "Romania", "Slovakia", "Slovenia", "Spain" ], "pubmed": 2, "trials": 21, "us_orphan": "unknown", "chembl": [ { "disease": "Diabetic Neuropathies", "phase": 3.0 }, { "disease": "Alzheimer Disease", "phase": 2.0 }, { "disease": "Diabetes Mellitus, Type 2", "phase": 2.0 }, { "disease": "Diabetes Mellitus, Type 1", "phase": 1.0 } ], "disease_terms": "Diabetic Neuropathies", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "3O31P6T4G3", "name": "LEVODROPROPIZINE", "moiety": "LEVODROPROPIZINE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Levodropropizine" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "STEREO_NAME: name carries an enantiomer/racemate marker; check the counterpart's US status by hand" ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 14, "countries": [ "Belgium", "Czech Republic", "Finland", "Germany", "Greece", "Iceland", "Italy", "Luxembourg", "Malta", "Poland", "Portugal", "Romania", "Slovakia", "Spain" ], "pubmed": 9, "trials": 10, "us_orphan": "unknown", "chembl": [ { "disease": "Cough", "phase": 3.0 }, { "disease": "Bronchial Diseases", "phase": 3.0 }, { "disease": "Dyspnea", "phase": 2.0 } ], "disease_terms": "Cough; Bronchial Diseases", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "7D6EM3S13P", "name": "LYMECYCLINE", "moiety": "LYMECYCLINE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Lymecycline" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 14, "countries": [ "Austria", "Belgium", "Denmark", "Finland", "France", "Hungary", "Iceland", "Ireland", "Malta", "Norway", "Poland", "Portugal", "Sweden", "United Kingdom (Northern Ireland)" ], "pubmed": 1, "trials": 6, "us_orphan": "unknown", "chembl": [ { "disease": "Bacterial Infections", "phase": 4.0 }, { "disease": "Acne Vulgaris", "phase": 3.0 } ], "disease_terms": "Bacterial Infections", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "V4TKW1454M", "name": "NIMESULIDE", "moiety": "NIMESULIDE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Nimesulide" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 14, "countries": [ "Bulgaria", "Croatia", "Cyprus", "Czech Republic", "Greece", "Hungary", "Italy", "Latvia", "Lithuania", "Malta", "Poland", "Portugal", "Romania", "Slovakia" ], "pubmed": 34, "trials": 17, "us_orphan": "unknown", "chembl": [ { "disease": "Rheumatic Diseases", "phase": 4.0 }, { "disease": "Arthralgia", "phase": 4.0 }, { "disease": "Myalgia", "phase": 4.0 }, { "disease": "Breast Neoplasms", "phase": 2.0 }, { "disease": "Pain", "phase": 1.0 } ], "disease_terms": "Rheumatic Diseases; Arthralgia; Myalgia", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "947S0YZ36I", "name": "REBOXETINE", "moiety": "REBOXETINE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Reboxetine", "Reboxetine Methanesulfonate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 14, "countries": [ "Austria", "Belgium", "Bulgaria", "Croatia", "Germany", "Ireland", "Italy", "Luxembourg", "Poland", "Portugal", "Slovenia", "Spain", "Sweden", "United Kingdom (Northern Ireland)" ], "pubmed": 5, "trials": 41, "us_orphan": "unknown", "chembl": [ { "disease": "Depressive Disorder", "phase": 4.0 }, { "disease": "Anxiety", "phase": 3.0 }, { "disease": "Fibromyalgia", "phase": 3.0 }, { "disease": "Dementia", "phase": 3.0 }, { "disease": "Schizophrenia", "phase": 3.0 }, { "disease": "Fatigue", "phase": 3.0 }, { "disease": "Pain", "phase": 2.0 }, { "disease": "Attention Deficit Disorder with Hyperactivity", "phase": 2.0 }, { "disease": "Diabetic Neuropathies", "phase": 2.0 }, { "disease": "Depressive Disorder, Major", "phase": 2.0 }, { "disease": "Stress Disorders, Post-Traumatic", "phase": 2.0 } ], "disease_terms": "Depressive Disorder", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "00DPD30SOY", "name": "AMSACRINE", "moiety": "AMSACRINE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Amsacrine" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 13, "countries": [ "Austria", "Croatia", "Denmark", "Finland", "France", "Germany", "Iceland", "Ireland", "Italy", "Luxembourg", "Netherlands", "Norway", "Sweden" ], "pubmed": 16, "trials": 11, "us_orphan": "unknown", "chembl": [ { "disease": "Neoplasms", "phase": 4.0 }, { "disease": "Leukemia", "phase": 3.0 }, { "disease": "Leukemia, Myeloid, Acute", "phase": 3.0 }, { "disease": "Myelodysplastic Syndromes", "phase": 3.0 } ], "disease_terms": "Neoplasms", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "5921X1560Q", "name": "CALCIUM DOBESILATE", "moiety": "CALCIUM DOBESILATE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Calcium Dobesilate", "Calcium Dobesilate Monohydrate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 13, "countries": [ "Austria", "Bulgaria", "Cyprus", "Czech Republic", "France", "Greece", "Hungary", "Poland", "Portugal", "Romania", "Slovakia", "Slovenia", "Spain" ], "pubmed": 0, "trials": 806, "us_orphan": "unknown", "chembl": [ { "disease": "Varicose Veins", "phase": 4.0 }, { "disease": "Menorrhagia", "phase": 3.0 }, { "disease": "Infertility", "phase": 2.0 }, { "disease": "Diabetes Mellitus, Type 2", "phase": 2.0 }, { "disease": "Rosacea", "phase": 1.0 } ], "disease_terms": "Varicose Veins", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "G20G12V769", "name": "PROPAMIDINE", "moiety": "PROPAMIDINE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Propamidine Isetionate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 13, "countries": [ "Belgium", "Denmark", "Finland", "France", "Ireland", "Luxembourg", "Malta", "Netherlands", "Norway", "Portugal", "Romania", "Sweden", "United Kingdom (Northern Ireland)" ], "pubmed": 4, "trials": 1, "us_orphan": "unknown", "chembl": [ { "disease": "Acanthamoeba Keratitis", "phase": 3.0 }, { "disease": "Eye Infections", "phase": 3.0 } ], "disease_terms": "Acanthamoeba Keratitis; Eye Infections", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "P67IM25ID8", "name": "RILMENIDINE", "moiety": "RILMENIDINE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Rilmenidine", "Rilmenidine Dihydrogen Phosphate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 13, "countries": [ "Austria", "Bulgaria", "Czech Republic", "Estonia", "France", "Hungary", "Latvia", "Lithuania", "Luxembourg", "Poland", "Portugal", "Romania", "Slovakia" ], "pubmed": 1, "trials": 2, "us_orphan": "unknown", "chembl": [ { "disease": "Hypertension", "phase": 4.0 } ], "disease_terms": "Hypertension", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "H6630PU5ZJ", "name": "TAUROSELCHOLIC ACID", "moiety": "TAUROSELCHOLIC", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Tauroselcholic Acid" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 13, "countries": [ "Belgium", "Denmark", "Finland", "Hungary", "Ireland", "Italy", "Netherlands", "Norway", "Poland", "Portugal", "Slovakia", "Sweden", "United Kingdom (Northern Ireland)" ], "pubmed": 0, "trials": 0, "us_orphan": "unknown", "chembl": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "8KQ660G60G", "name": "CARBIMAZOLE", "moiety": "CARBIMAZOLE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Carbimazole", "Carbimazole Ph. Eur." ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 12, "countries": [ "Cyprus", "Denmark", "Finland", "France", "Germany", "Greece", "Ireland", "Malta", "Norway", "Portugal", "Spain", "United Kingdom (Northern Ireland)" ], "pubmed": 82, "trials": 7, "us_orphan": "unknown", "chembl": [ { "disease": "Thyroid Diseases", "phase": 4.0 }, { "disease": "Thyrotoxicosis", "phase": 3.0 } ], "disease_terms": "Thyroid Diseases", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "W13O82Z7HL", "name": "DOTHIEPIN", "moiety": "DOSULEPIN", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Dosulepin Hydrochloride", "Dosulepin Hydrochloride Bp" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 12, "countries": [ "Belgium", "Czech Republic", "Denmark", "France", "Germany", "Ireland", "Italy", "Luxembourg", "Netherlands", "Portugal", "Slovakia", "United Kingdom (Northern Ireland)" ], "pubmed": 1, "trials": 3, "us_orphan": "unknown", "chembl": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "24YL531VOH", "name": "ETHAMSYLATE", "moiety": "ETAMSILATE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Etamsilate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 12, "countries": [ "Belgium", "Bulgaria", "Czech Republic", "France", "Hungary", "Latvia", "Lithuania", "Luxembourg", "Poland", "Romania", "Slovakia", "Slovenia" ], "pubmed": 0, "trials": 0, "us_orphan": "unknown", "chembl": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "C7C2QDD75P", "name": "GLIQUIDONE", "moiety": "GLIQUIDONE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Gliquidone" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 12, "countries": [ "Austria", "Belgium", "Croatia", "Czech Republic", "Germany", "Hungary", "Italy", "Lithuania", "Luxembourg", "Romania", "Slovakia", "Slovenia" ], "pubmed": 0, "trials": 5, "us_orphan": "unknown", "chembl": [ { "disease": "Diabetes Mellitus", "phase": 4.0 } ], "disease_terms": "Diabetes Mellitus", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "CV71VTP0VN", "name": "IPIDACRINE", "moiety": "IPIDACRINE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Ipidacrine Hydrochloride" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 12, "countries": [ "Austria", "Bulgaria", "Croatia", "Finland", "Hungary", "Latvia", "Lithuania", "Norway", "Poland", "Romania", "Slovakia", "Slovenia" ], "pubmed": 0, "trials": 0, "us_orphan": "unknown", "chembl": [ { "disease": "Dementia", "phase": 2.0 } ], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "GRI7WFR424", "name": "ISOCONAZOLE", "moiety": "ISOCONAZOLE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Isoconazole Nitrate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 12, "countries": [ "Austria", "Cyprus", "Estonia", "France", "Greece", "Italy", "Latvia", "Lithuania", "Luxembourg", "Poland", "Portugal", "Romania" ], "pubmed": 3, "trials": 1, "us_orphan": "unknown", "chembl": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "GU56C842ZA", "name": "LORMETAZEPAM", "moiety": "LORMETAZEPAM", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Lormetazepam" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 12, "countries": [ "Austria", "Belgium", "Cyprus", "France", "Germany", "Italy", "Luxembourg", "Malta", "Netherlands", "Poland", "Spain", "United Kingdom (Northern Ireland)" ], "pubmed": 0, "trials": 6, "us_orphan": "unknown", "chembl": [ { "disease": "Anxiety", "phase": 3.0 }, { "disease": "Dementia", "phase": 3.0 }, { "disease": "Depressive Disorder", "phase": 3.0 }, { "disease": "Sleep Wake Disorders", "phase": 3.0 }, { "disease": "Schizophrenia", "phase": 3.0 } ], "disease_terms": "Anxiety; Dementia; Depressive Disorder; Sleep Wake Disorders; Schizophrenia", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "7Y4N11PXO8", "name": "TROXERUTIN", "moiety": "TROXERUTIN", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Troxerutin" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 12, "countries": [ "Bulgaria", "Czech Republic", "Estonia", "France", "Germany", "Italy", "Latvia", "Lithuania", "Poland", "Romania", "Slovakia", "Spain" ], "pubmed": 2, "trials": 6, "us_orphan": "unknown", "chembl": [ { "disease": "Cardiovascular Diseases", "phase": 4.0 }, { "disease": "Angina, Stable", "phase": 3.0 }, { "disease": "Inflammation", "phase": 1.0 } ], "disease_terms": "Cardiovascular Diseases", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "DRB57K47QC", "name": "CELIPROLOL", "moiety": "CELIPROLOL", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Celiprolol", "Celiprolol Hydrochloride" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 11, "countries": [ "Czech Republic", "Finland", "France", "Germany", "Greece", "Hungary", "Italy", "Netherlands", "Poland", "Slovakia", "United Kingdom (Northern Ireland)" ], "pubmed": 3, "trials": 4, "us_orphan": "unknown", "chembl": [ { "disease": "Cardiovascular Diseases", "phase": 4.0 }, { "disease": "Ehlers-Danlos Syndrome", "phase": 3.0 } ], "disease_terms": "Cardiovascular Diseases", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "KD510K1IQW", "name": "CHOLINE SALICYLATE", "moiety": "", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "direct", "examples": [ "Choline Salicylate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT', 'OTC MONOGRAPH DRUG']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 11, "countries": [ "Austria", "Cyprus", "Czech Republic", "Estonia", "Hungary", "Latvia", "Lithuania", "Malta", "Poland", "Portugal", "Slovakia" ], "pubmed": 3, "trials": 376, "us_orphan": "unknown", "chembl": [ { "disease": "Pain", "phase": 4.0 }, { "disease": "Fever", "phase": 4.0 } ], "disease_terms": "Pain; Fever", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "0813BZ6866", "name": "CLODRONIC ACID", "moiety": "CLODRONATE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Clodronate Disodium", "Clodronate Disodium Tetrahydrate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 11, "countries": [ "Austria", "Cyprus", "Czech Republic", "France", "Germany", "Italy", "Malta", "Netherlands", "Portugal", "Romania", "United Kingdom (Northern Ireland)" ], "pubmed": 20, "trials": 17, "us_orphan": "unknown", "chembl": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "0C5DBZ19HV", "name": "CLOMETHIAZOLE", "moiety": "CLOMETHIAZOLE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Clomethiazole", "Clomethiazole Edisilate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 11, "countries": [ "Cyprus", "Czech Republic", "Germany", "Hungary", "Iceland", "Norway", "Poland", "Slovenia", "Spain", "Sweden", "United Kingdom (Northern Ireland)" ], "pubmed": 2, "trials": 1, "us_orphan": "unknown", "chembl": [ { "disease": "Anxiety", "phase": 3.0 }, { "disease": "Dementia", "phase": 3.0 }, { "disease": "Depressive Disorder", "phase": 3.0 }, { "disease": "Schizophrenia", "phase": 3.0 } ], "disease_terms": "Anxiety; Dementia; Depressive Disorder; Schizophrenia", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "17432CG1KU", "name": "HYDROTALCITE", "moiety": "HYDROTALCITE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Hydrotalcite" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 11, "countries": [ "Austria", "Bulgaria", "Croatia", "Czech Republic", "France", "Germany", "Greece", "Hungary", "Luxembourg", "Slovakia", "Slovenia" ], "pubmed": 0, "trials": 7, "us_orphan": "unknown", "chembl": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "ER09126G7A", "name": "LORNOXICAM", "moiety": "LORNOXICAM", "inn": true, "usan": true, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Lornoxicam" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 11, "countries": [ "Austria", "Bulgaria", "Denmark", "Greece", "Hungary", "Latvia", "Lithuania", "Poland", "Romania", "Slovakia", "Spain" ], "pubmed": 4, "trials": 21, "us_orphan": "unknown", "chembl": [ { "disease": "Rheumatic Diseases", "phase": 4.0 }, { "disease": "Pain", "phase": 3.0 }, { "disease": "Alzheimer Disease", "phase": 2.0 }, { "disease": "Migraine Disorders", "phase": 2.0 } ], "disease_terms": "Rheumatic Diseases", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "543512OBTC", "name": "VINPOCETINE", "moiety": "VINPOCETINE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Vinpocetine" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 11, "countries": [ "Bulgaria", "Czech Republic", "Hungary", "Latvia", "Lithuania", "Poland", "Portugal", "Romania", "Slovakia", "Slovenia", "Spain" ], "pubmed": 1, "trials": 9, "us_orphan": "unknown", "chembl": [ { "disease": "Attention Deficit Disorder with Hyperactivity", "phase": 4.0 }, { "disease": "Diabetic Nephropathies", "phase": 2.0 }, { "disease": "Epilepsy", "phase": 1.0 } ], "disease_terms": "Attention Deficit Disorder with Hyperactivity", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "PXW8U3YXDV", "name": "BUSERELIN", "moiety": "BUSERELIN", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Buserelin Acetate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 10, "countries": [ "Austria", "Bulgaria", "Germany", "Greece", "Hungary", "Italy", "Netherlands", "Spain", "Sweden", "United Kingdom (Northern Ireland)" ], "pubmed": 12, "trials": 42, "us_orphan": "unknown", "chembl": [ { "disease": "Neoplasms", "phase": 4.0 }, { "disease": "Prostatic Neoplasms, Castration-Resistant", "phase": 3.0 }, { "disease": "Prostatic Neoplasms", "phase": 3.0 }, { "disease": "Infertility", "phase": 1.0 } ], "disease_terms": "Neoplasms", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "19KW7PI29F", "name": "CILAZAPRIL", "moiety": "CILAZAPRIL", "inn": false, "usan": true, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Cilazapril", "Cilazapril Monohydrate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 10, "countries": [ "Austria", "Belgium", "Czech Republic", "Germany", "Greece", "Hungary", "Italy", "Poland", "Portugal", "United Kingdom (Northern Ireland)" ], "pubmed": 3, "trials": 4, "us_orphan": "unknown", "chembl": [ { "disease": "Cardiovascular Diseases", "phase": 4.0 } ], "disease_terms": "Cardiovascular Diseases", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "98QS4N58TW", "name": "DROTAVERINE", "moiety": "DROTAVERINE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Drotaverine Hydrochloride" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 10, "countries": [ "Bulgaria", "Czech Republic", "Estonia", "Hungary", "Latvia", "Lithuania", "Malta", "Poland", "Romania", "Slovakia" ], "pubmed": 1, "trials": 6, "us_orphan": "unknown", "chembl": [ { "disease": "Digestive System Diseases", "phase": 3.0 }, { "disease": "Bile Duct Diseases", "phase": 3.0 } ], "disease_terms": "Digestive System Diseases; Bile Duct Diseases", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "J8WA3K39B7", "name": "MELPERONE", "moiety": "MELPERONE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Melperone Hydrochloride" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 10, "countries": [ "Austria", "Czech Republic", "Denmark", "Estonia", "Germany", "Iceland", "Latvia", "Lithuania", "Portugal", "Sweden" ], "pubmed": 0, "trials": 4, "us_orphan": "unknown", "chembl": [ { "disease": "Psychotic Disorders", "phase": 3.0 }, { "disease": "Anxiety", "phase": 3.0 }, { "disease": "Dementia", "phase": 3.0 }, { "disease": "Depressive Disorder", "phase": 3.0 }, { "disease": "Schizophrenia", "phase": 3.0 } ], "disease_terms": "Psychotic Disorders; Anxiety; Dementia; Depressive Disorder; Schizophrenia", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "80Q9QWN15M", "name": "QUINAGOLIDE", "moiety": "QUINAGOLIDE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Quinagolide", "Quinagolide Hydrochloride" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 10, "countries": [ "Austria", "Czech Republic", "France", "Germany", "Greece", "Hungary", "Netherlands", "Poland", "Slovakia", "United Kingdom (Northern Ireland)" ], "pubmed": 1, "trials": 6, "us_orphan": "unknown", "chembl": [ { "disease": "Ovarian Hyperstimulation Syndrome", "phase": 2.0 } ], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "FCB9EGG971", "name": "RALTITREXED", "moiety": "RALTITREXED", "inn": true, "usan": true, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Raltitrexed" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 10, "countries": [ "Austria", "Belgium", "France", "Hungary", "Italy", "Luxembourg", "Malta", "Portugal", "Spain", "United Kingdom (Northern Ireland)" ], "pubmed": 8, "trials": 87, "us_orphan": "unknown", "chembl": [ { "disease": "Neoplasms", "phase": 4.0 }, { "disease": "Mesothelioma", "phase": 3.0 }, { "disease": "Carcinoma, Squamous Cell", "phase": 3.0 }, { "disease": "Colorectal Neoplasms", "phase": 3.0 }, { "disease": "Carcinoma, Hepatocellular", "phase": 3.0 }, { "disease": "Liver Neoplasms", "phase": 3.0 }, { "disease": "Rectal Neoplasms", "phase": 2.0 }, { "disease": "Esophageal Neoplasms", "phase": 2.0 }, { "disease": "Stomach Neoplasms", "phase": 2.0 }, { "disease": "Pancreatic Neoplasms", "phase": 2.0 }, { "disease": "Adenoma, Bile Duct", "phase": 2.0 }, { "disease": "Nasopharyngeal Carcinoma", "phase": 2.0 }, { "disease": "Precursor Cell Lymphoblastic Leukemia-Lymphoma", "phase": 1.0 } ], "disease_terms": "Neoplasms", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "5V141XK28X", "name": "ACEMETACIN", "moiety": "ACEMETACIN", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Acemetacin" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 9, "countries": [ "Austria", "Croatia", "Germany", "Greece", "Hungary", "Luxembourg", "Malta", "Poland", "Portugal" ], "pubmed": 2, "trials": 2, "us_orphan": "unknown", "chembl": [ { "disease": "Rheumatic Diseases", "phase": 4.0 } ], "disease_terms": "Rheumatic Diseases", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "2M3055079H", "name": "BRIVUDINE", "moiety": "BRIVUDINE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Brivudine" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 9, "countries": [ "Austria", "Germany", "Greece", "Italy", "Portugal", "Romania", "Slovakia", "Slovenia", "Spain" ], "pubmed": 2, "trials": 2, "us_orphan": "unknown", "chembl": [ { "disease": "Virus Diseases", "phase": 2.0 }, { "disease": "Pancreatic Neoplasms", "phase": 2.0 }, { "disease": "Colorectal Neoplasms", "phase": 1.0 } ], "orpha": null, "eu_orphan_designation": { "use": "Treatment of pancreatic cancer", "status": "Positive", "number": "EU/3/09/703" }, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] }, "eu_orphan_designations": [ { "use": "Treatment of pancreatic cancer", "status": "Positive", "number": "EU/3/09/703" } ] }, { "unii": "5XZM1R3DKF", "name": "BROTIZOLAM", "moiety": "BROTIZOLAM", "inn": true, "usan": true, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Brotizolam" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 9, "countries": [ "Austria", "Belgium", "Germany", "Hungary", "Italy", "Luxembourg", "Netherlands", "Portugal", "Spain" ], "pubmed": 1, "trials": 10, "us_orphan": "unknown", "chembl": [ { "disease": "Sleep Initiation and Maintenance Disorders", "phase": 3.0 }, { "disease": "Anxiety", "phase": 3.0 }, { "disease": "Dementia", "phase": 3.0 }, { "disease": "Depressive Disorder", "phase": 3.0 }, { "disease": "Sleep Wake Disorders", "phase": 3.0 }, { "disease": "Schizophrenia", "phase": 3.0 } ], "disease_terms": "Sleep Initiation and Maintenance Disorders; Anxiety; Dementia; Depressive Disorder; Sleep Wake Disorders; Schizophrenia", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "ZB6F8MY53V", "name": "ETILEFRINE", "moiety": "ETILEFRINE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Etilefrine Hydrochloride" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 9, "countries": [ "Austria", "Belgium", "Finland", "France", "Germany", "Italy", "Luxembourg", "Poland", "Sweden" ], "pubmed": 7, "trials": 2, "us_orphan": "unknown", "chembl": [ { "disease": "Cardiovascular Diseases", "phase": 4.0 }, { "disease": "Ascites", "phase": 2.0 } ], "disease_terms": "Cardiovascular Diseases", "orpha": null, "eu_orphan_designation": { "use": "Treatment of low-flow priapism", "status": "Positive", "number": "EU/3/02/122" }, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] }, "eu_orphan_designations": [ { "use": "Treatment of low-flow priapism", "status": "Positive", "number": "EU/3/02/122" } ] }, { "unii": "R7PLA2DM0J", "name": "FLUNARIZINE", "moiety": "FLUNARIZINE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Flunarizine", "Flunarizine Hydrochloride" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 9, "countries": [ "Bulgaria", "France", "Germany", "Italy", "Liechtenstein", "Netherlands", "Poland", "Portugal", "Spain" ], "pubmed": 53, "trials": 21, "us_orphan": "unknown", "chembl": [ { "disease": "Vertigo", "phase": 4.0 }, { "disease": "Migraine Disorders", "phase": 1.0 } ], "disease_terms": "Vertigo", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "JCV8365FWN", "name": "NICERGOLINE", "moiety": "NICERGOLINE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Nicergoline" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 9, "countries": [ "Austria", "Bulgaria", "Germany", "Hungary", "Latvia", "Poland", "Portugal", "Romania", "Spain" ], "pubmed": 1, "trials": 6, "us_orphan": "unknown", "chembl": [ { "disease": "Cardiovascular Diseases", "phase": 4.0 }, { "disease": "Anxiety", "phase": 3.0 }, { "disease": "Dementia", "phase": 3.0 }, { "disease": "Depressive Disorder", "phase": 3.0 }, { "disease": "Schizophrenia", "phase": 3.0 }, { "disease": "Deglutition Disorders", "phase": 3.0 } ], "disease_terms": "Cardiovascular Diseases", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "C2W72OJ5ZU", "name": "TACALCITOL", "moiety": "TACALCITOL", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Tacalcitol", "Tacalcitol Monohydrate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 9, "countries": [ "Austria", "Belgium", "Czech Republic", "Germany", "Hungary", "Luxembourg", "Poland", "Slovakia", "United Kingdom (Northern Ireland)" ], "pubmed": 1, "trials": 2, "us_orphan": "unknown", "chembl": [ { "disease": "Psoriasis", "phase": 4.0 } ], "disease_terms": "Psoriasis", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "03QB156W6I", "name": "TEMOCILLIN", "moiety": "TEMOCILLIN", "inn": true, "usan": true, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Temocillin Sodium" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 9, "countries": [ "Belgium", "France", "Germany", "Ireland", "Luxembourg", "Netherlands", "Spain", "Sweden", "United Kingdom (Northern Ireland)" ], "pubmed": 5, "trials": 16, "us_orphan": "unknown", "chembl": [ { "disease": "Urinary Tract Infections", "phase": 3.0 }, { "disease": "Bacterial Infections", "phase": 3.0 }, { "disease": "Bacteremia", "phase": 3.0 } ], "disease_terms": "Urinary Tract Infections; Bacterial Infections; Bacteremia", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "0F32U78V2Q", "name": "CHLOROXYLENOL", "moiety": "CHLOROXYLENOL", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Chloroxylenol", "Chloroxylenol Bp" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT', 'OTC MONOGRAPH DRUG', 'OTC MONOGRAPH NOT FINAL', 'UNAPPROVED DRUG OTHER']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 8, "countries": [ "Belgium", "Germany", "Greece", "Ireland", "Luxembourg", "Malta", "Netherlands", "United Kingdom (Northern Ireland)" ], "pubmed": 0, "trials": 9, "us_orphan": "unknown", "chembl": [ { "disease": "Inflammation", "phase": 4.0 } ], "disease_terms": "Inflammation", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "620X0222FQ", "name": "FLUNITRAZEPAM", "moiety": "FLUNITRAZEPAM", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Flunitrazepam" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 8, "countries": [ "Austria", "Belgium", "Cyprus", "Germany", "Greece", "Italy", "Luxembourg", "Netherlands" ], "pubmed": 19, "trials": 2, "us_orphan": "unknown", "chembl": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "N2Z8ALG4U5", "name": "LYNESTRENOL", "moiety": "LYNESTRENOL", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Lynestrenol" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 8, "countries": [ "Austria", "Belgium", "Czech Republic", "Finland", "Luxembourg", "Netherlands", "Poland", "Sweden" ], "pubmed": 6, "trials": 4, "us_orphan": "unknown", "chembl": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "6V88E24N5T", "name": "MAGALDRATE", "moiety": "MAGALDRATE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Magaldrate", "Magaldrate Anhydrous" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT', 'DRUG FOR FURTHER PROCESSING']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 8, "countries": [ "Austria", "Belgium", "Germany", "Greece", "Italy", "Luxembourg", "Portugal", "Spain" ], "pubmed": 0, "trials": 4, "us_orphan": "unknown", "chembl": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "I7GG1YUC5V", "name": "METILDIGOXIN", "moiety": "METILDIGOXIN", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Metildigoxin" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 8, "countries": [ "Austria", "Belgium", "Bulgaria", "Croatia", "Germany", "Portugal", "Slovenia", "Spain" ], "pubmed": 0, "trials": 0, "us_orphan": "unknown", "chembl": [ { "disease": "Cardiovascular Diseases", "phase": 4.0 } ], "disease_terms": "Cardiovascular Diseases", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "42H8PQ0NMJ", "name": "NAFTIDROFURYL", "moiety": "NAFTIDROFURYL", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Naftidrofuryl Hydrogen Oxalate", "Naftidrofuryl Hydrogen Oxalate Ph.Eur." ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 8, "countries": [ "Bulgaria", "Cyprus", "Czech Republic", "Estonia", "France", "Latvia", "Slovakia", "United Kingdom (Northern Ireland)" ], "pubmed": 1, "trials": 0, "us_orphan": "unknown", "chembl": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "T1X8S697GT", "name": "THIOCOLCHICOSIDE", "moiety": "THIOCOLCHICOSIDE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Thiocolchicoside" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 8, "countries": [ "Bulgaria", "Cyprus", "Czech Republic", "France", "Greece", "Italy", "Poland", "Portugal" ], "pubmed": 3, "trials": 15, "us_orphan": "unknown", "chembl": [ { "disease": "Low Back Pain", "phase": 3.0 } ], "disease_terms": "Low Back Pain", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "U3RSY48JW5", "name": "BENZOCAINE", "moiety": "BENZOCAINE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Benzocaine" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT', 'BULK INGREDIENT FOR HUMAN PRESCRIPTION COMPOUNDING', 'DRUG FOR FURTHER PROCESSING', 'OTC MONOGRAPH DRUG', 'OTC MONOGRAPH NOT FINAL', 'UNAPPROVED DRUG OTHER']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 7, "countries": [ "Germany", "Malta", "Netherlands", "Poland", "Portugal", "Spain", "United Kingdom (Northern Ireland)" ], "pubmed": 49, "trials": 7250, "us_orphan": "unknown", "chembl": [ { "disease": "Pruritus", "phase": 4.0 }, { "disease": "Anesthesia", "phase": 4.0 }, { "disease": "Hemorrhoids", "phase": 4.0 }, { "disease": "Pharyngitis", "phase": 4.0 }, { "disease": "Sunburn", "phase": 4.0 }, { "disease": "Burns", "phase": 4.0 }, { "disease": "Toothache", "phase": 4.0 }, { "disease": "Nasal Obstruction", "phase": 4.0 }, { "disease": "Inflammation", "phase": 4.0 }, { "disease": "Insect Bites and Stings", "phase": 4.0 }, { "disease": "Premature Ejaculation", "phase": 4.0 }, { "disease": "Eczema", "phase": 3.0 }, { "disease": "Otitis Media", "phase": 3.0 }, { "disease": "Asthma", "phase": 2.0 }, { "disease": "Obesity", "phase": 1.0 } ], "disease_terms": "Pruritus; Anesthesia; Hemorrhoids; Pharyngitis; Sunburn; Burns", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "536BQ2JVC7", "name": "CITICOLINE", "moiety": "CITICOLINE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Citicoline", "Citicoline Sodium" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['UNAPPROVED HOMEOPATHIC']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 7, "countries": [ "Austria", "Bulgaria", "Italy", "Malta", "Poland", "Portugal", "Spain" ], "pubmed": 6, "trials": 72, "us_orphan": "unknown", "chembl": [ { "disease": "Stroke", "phase": 3.0 }, { "disease": "Amphetamine-Related Disorders", "phase": 3.0 }, { "disease": "Attention Deficit Disorder with Hyperactivity", "phase": 3.0 }, { "disease": "Cerebral Infarction", "phase": 3.0 }, { "disease": "Brain Ischemia", "phase": 3.0 }, { "disease": "Brain Injuries", "phase": 3.0 }, { "disease": "Ischemic Stroke", "phase": 3.0 }, { "disease": "Glaucoma, Open-Angle", "phase": 3.0 }, { "disease": "Marijuana Abuse", "phase": 2.0 }, { "disease": "Alcoholism", "phase": 2.0 } ], "disease_terms": "Stroke; Amphetamine-Related Disorders; Attention Deficit Disorder with Hyperactivity; Cerebral Infarction; Brain Ischemia; Brain Injuries", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "9902116282", "name": "MYRTECAINE", "moiety": "MYRTECAINE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Myrtecaine, Diethylamine Salicylate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 7, "countries": [ "Austria", "Czech Republic", "Germany", "Greece", "Portugal", "Slovakia", "Spain" ], "pubmed": 0, "trials": 0, "us_orphan": "unknown", "chembl": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "4U5MP5IUD8", "name": "NIFLUMIC ACID", "moiety": "NIFLUMIC", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Niflumic Acid" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 7, "countries": [ "Belgium", "France", "Greece", "Hungary", "Italy", "Luxembourg", "Spain" ], "pubmed": 9, "trials": 1, "us_orphan": "unknown", "chembl": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "5402501F0W", "name": "PIPAMPERONE", "moiety": "PIPAMPERONE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Pipamperone", "Pipamperone Hydrochloride" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 7, "countries": [ "Belgium", "Denmark", "France", "Germany", "Greece", "Luxembourg", "Netherlands" ], "pubmed": 3, "trials": 6, "us_orphan": "unknown", "chembl": [ { "disease": "Psychotic Disorders", "phase": 3.0 }, { "disease": "Anxiety", "phase": 3.0 }, { "disease": "Dementia", "phase": 3.0 }, { "disease": "Depressive Disorder, Major", "phase": 3.0 }, { "disease": "Depressive Disorder", "phase": 3.0 }, { "disease": "Schizophrenia", "phase": 3.0 } ], "disease_terms": "Psychotic Disorders; Anxiety; Dementia; Depressive Disorder, Major; Depressive Disorder; Schizophrenia", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "DO22K1PRDJ", "name": "PIRIBEDIL", "moiety": "PIRIBEDIL", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Piribedil" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 7, "countries": [ "France", "Germany", "Lithuania", "Luxembourg", "Poland", "Portugal", "Romania" ], "pubmed": 0, "trials": 5, "us_orphan": "unknown", "chembl": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "4RP92LYZ2F", "name": "PIRITRAMIDE", "moiety": "PIRITRAMIDE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Piritramide" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 7, "countries": [ "Austria", "Belgium", "Czech Republic", "Germany", "Luxembourg", "Netherlands", "Slovenia" ], "pubmed": 18, "trials": 25, "us_orphan": "unknown", "chembl": [ { "disease": "Pain", "phase": 3.0 } ], "disease_terms": "Pain", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "0BY8440V3N", "name": "PIZOTYLINE", "moiety": "PIZOTIFEN", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Pizotifen", "Pizotifen Malate", "Pizotifen Malate Bp" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 7, "countries": [ "Cyprus", "France", "Ireland", "Netherlands", "Slovakia", "Spain", "United Kingdom (Northern Ireland)" ], "pubmed": 8, "trials": 2, "us_orphan": "unknown", "chembl": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "501Q5EQ1GM", "name": "SULPROSTONE", "moiety": "SULPROSTONE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Sulprostone" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 7, "countries": [ "Austria", "France", "Germany", "Hungary", "Italy", "Netherlands", "Portugal" ], "pubmed": 8, "trials": 5, "us_orphan": "unknown", "chembl": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "M671F9NLEA", "name": "CANRENOATE POTASSIUM", "moiety": "POTASSIUM CANRENOATE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Potassium Canrenoate", "Potassium Canrenoate, Trometamol" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 6, "countries": [ "Austria", "Czech Republic", "France", "Germany", "Italy", "Poland" ], "pubmed": 2, "trials": 20, "us_orphan": "unknown", "chembl": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "68P0556B0U", "name": "CINOLAZEPAM", "moiety": "CINOLAZEPAM", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Cinolazepam" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 6, "countries": [ "Austria", "Bulgaria", "Czech Republic", "Hungary", "Romania", "Slovakia" ], "pubmed": 0, "trials": 0, "us_orphan": "unknown", "chembl": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "K253365DXI", "name": "DIFLUCORTOLONE", "moiety": "DIFLUCORTOLONE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Diflucortolone Valerate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 6, "countries": [ "Austria", "France", "Germany", "Italy", "Portugal", "Spain" ], "pubmed": 4, "trials": 3, "us_orphan": "unknown", "chembl": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "776S0UP252", "name": "FLUTRIMAZOLE", "moiety": "FLUTRIMAZOLE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Flutrimazole" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 6, "countries": [ "Austria", "Czech Republic", "Greece", "Hungary", "Slovakia", "Spain" ], "pubmed": 0, "trials": 0, "us_orphan": "unknown", "chembl": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "BW7H1TJS22", "name": "IMIDAPRIL", "moiety": "IMIDAPRIL", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Imidapril Hydrochloride" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 6, "countries": [ "Austria", "Czech Republic", "Poland", "Portugal", "Spain", "United Kingdom (Northern Ireland)" ], "pubmed": 0, "trials": 5, "us_orphan": "unknown", "chembl": [ { "disease": "Cardiovascular Diseases", "phase": 4.0 }, { "disease": "Essential Hypertension", "phase": 3.0 }, { "disease": "Polycystic Kidney, Autosomal Dominant", "phase": 2.0 } ], "disease_terms": "Cardiovascular Diseases", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "JTG7R315LK", "name": "LEVOSULPIRIDE", "moiety": "LEVOSULPIRIDE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Levosulpiride" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "STEREO_NAME: name carries an enantiomer/racemate marker; check the counterpart's US status by hand" ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 6, "countries": [ "Bulgaria", "Cyprus", "Italy", "Malta", "Romania", "Spain" ], "pubmed": 2, "trials": 5, "us_orphan": "unknown", "chembl": [ { "disease": "Psychotic Disorders", "phase": 2.0 }, { "disease": "Psychomotor Agitation", "phase": 2.0 }, { "disease": "Dyspepsia", "phase": 1.0 } ], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "759N8462G8", "name": "LOPRAZOLAM", "moiety": "LOPRAZOLAM", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Loprazolam", "Loprazolam Mesilate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 6, "countries": [ "Belgium", "France", "Luxembourg", "Portugal", "Spain", "United Kingdom (Northern Ireland)" ], "pubmed": 0, "trials": 0, "us_orphan": "unknown", "chembl": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "0SRQ75X9I9", "name": "MESTEROLONE", "moiety": "MESTEROLONE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Mesterolone" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 6, "countries": [ "Belgium", "Greece", "Luxembourg", "Portugal", "Spain", "United Kingdom (Northern Ireland)" ], "pubmed": 1, "trials": 0, "us_orphan": "unknown", "chembl": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "U60U6P08SP", "name": "NIFURATEL", "moiety": "NIFURATEL", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Nifuratel" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 6, "countries": [ "Bulgaria", "Czech Republic", "Italy", "Lithuania", "Poland", "Slovakia" ], "pubmed": 4, "trials": 0, "us_orphan": "unknown", "chembl": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "10F89177CO", "name": "NOMEGESTROL", "moiety": "NOMEGESTROL", "inn": true, "usan": true, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Nomegestrol Acetate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 6, "countries": [ "Belgium", "France", "Italy", "Luxembourg", "Poland", "Portugal" ], "pubmed": 0, "trials": 22, "us_orphan": "unknown", "chembl": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "9E75Q6SUUB", "name": "PRIDINOL", "moiety": "PRIDINOL", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Pridinol Hydrochloride", "Pridinol Mesilate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 6, "countries": [ "Austria", "Germany", "Italy", "Poland", "Spain", "United Kingdom (Northern Ireland)" ], "pubmed": 0, "trials": 1, "us_orphan": "unknown", "chembl": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "UZC80HAU42", "name": "TOFISOPAM", "moiety": "TOFISOPAM", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Tofisopam" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 6, "countries": [ "Bulgaria", "Czech Republic", "Hungary", "Latvia", "Lithuania", "Slovakia" ], "pubmed": 1, "trials": 2, "us_orphan": "unknown", "chembl": [ { "disease": "Anxiety", "phase": 2.0 } ], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "1Z0YFI05OO", "name": "TRIFLUSAL", "moiety": "TRIFLUSAL", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Triflusal" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 6, "countries": [ "Bulgaria", "Greece", "Hungary", "Portugal", "Romania", "Spain" ], "pubmed": 0, "trials": 6, "us_orphan": "unknown", "chembl": [ { "disease": "Thrombosis", "phase": 3.0 }, { "disease": "Insulin Resistance", "phase": 2.0 } ], "disease_terms": "Thrombosis", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "EJ27X76M46", "name": "UBIDECARENONE", "moiety": "UBIDECARENONE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Ubidecarenone" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT FOR HUMAN PRESCRIPTION COMPOUNDING', 'UNAPPROVED DRUG OTHER', 'UNAPPROVED HOMEOPATHIC']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 6, "countries": [ "Denmark", "Hungary", "Italy", "Latvia", "Poland", "Portugal" ], "pubmed": 0, "trials": 285, "us_orphan": "unknown", "chembl": [ { "disease": "Cardiovascular Diseases", "phase": 3.0 }, { "disease": "Pre-Eclampsia", "phase": 3.0 }, { "disease": "Fatigue Syndrome, Chronic", "phase": 3.0 }, { "disease": "Multiple Organ Failure", "phase": 3.0 }, { "disease": "Carcinoma, Hepatocellular", "phase": 2.0 }, { "disease": "Death, Sudden, Cardiac", "phase": 2.0 }, { "disease": "Heart Failure", "phase": 2.0 }, { "disease": "Sepsis", "phase": 2.0 }, { "disease": "Carcinoma, Squamous Cell", "phase": 2.0 }, { "disease": "Heart Arrest", "phase": 2.0 }, { "disease": "Heart Failure, Diastolic", "phase": 2.0 }, { "disease": "Glioblastoma", "phase": 2.0 }, { "disease": "Coronary Disease", "phase": 2.0 }, { "disease": "Depressive Disorder", "phase": 2.0 }, { "disease": "Polycystic Ovary Syndrome", "phase": 2.0 }, { "disease": "Gliosarcoma", "phase": 1.0 }, { "disease": "Carcinoma, Basal Cell", "phase": 1.0 }, { "disease": "Cerebellar Ataxia", "phase": 1.0 }, { "disease": "Breast Neoplasms", "phase": 1.0 }, { "disease": "Wounds and Injuries", "phase": 0.5 } ], "disease_terms": "Cardiovascular Diseases; Pre-Eclampsia; Fatigue Syndrome, Chronic; Multiple Organ Failure", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "2K3L8O9SOV", "name": "CHLOROPYRAMINE", "moiety": "CHLOROPYRAMINE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Chloropyramine Hydrochloride" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 5, "countries": [ "Bulgaria", "Croatia", "Hungary", "Latvia", "Lithuania" ], "pubmed": 3, "trials": 1, "us_orphan": "unknown", "chembl": [ { "disease": "Pruritus", "phase": 2.0 }, { "disease": "Hypersensitivity", "phase": 2.0 } ], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "F8252JGO9S", "name": "CIPROFIBRATE", "moiety": "CIPROFIBRATE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Ciprofibrate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 5, "countries": [ "Bulgaria", "France", "Netherlands", "Poland", "United Kingdom (Northern Ireland)" ], "pubmed": 1, "trials": 4, "us_orphan": "unknown", "chembl": [ { "disease": "Cardiovascular Diseases", "phase": 4.0 } ], "disease_terms": "Cardiovascular Diseases", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "C7Z4ITI7L7", "name": "ENOXIMONE", "moiety": "ENOXIMONE", "inn": true, "usan": true, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Enoximone" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 5, "countries": [ "France", "Germany", "Italy", "Netherlands", "United Kingdom (Northern Ireland)" ], "pubmed": 20, "trials": 8, "us_orphan": "unknown", "chembl": [ { "disease": "Cardiovascular Diseases", "phase": 4.0 }, { "disease": "Heart Failure", "phase": 3.0 }, { "disease": "Coronary Disease", "phase": 3.0 } ], "disease_terms": "Cardiovascular Diseases", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "P0J3387W3S", "name": "MEDAZEPAM", "moiety": "MEDAZEPAM", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Medazepam" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 5, "countries": [ "Germany", "Hungary", "Lithuania", "Poland", "Romania" ], "pubmed": 3, "trials": 2, "us_orphan": "unknown", "chembl": [ { "disease": "Anxiety", "phase": 4.0 } ], "disease_terms": "Anxiety", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "244O1F90NA", "name": "MIZOLASTINE", "moiety": "MIZOLASTINE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Mizolastine" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 5, "countries": [ "France", "Germany", "Greece", "Italy", "Netherlands" ], "pubmed": 2, "trials": 1, "us_orphan": "unknown", "chembl": [ { "disease": "Hypersensitivity", "phase": 4.0 } ], "disease_terms": "Hypersensitivity", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "260456HAM0", "name": "NICORANDIL", "moiety": "NICORANDIL", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Nicorandil" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 5, "countries": [ "Austria", "Denmark", "France", "Netherlands", "United Kingdom (Northern Ireland)" ], "pubmed": 14, "trials": 44, "us_orphan": "unknown", "chembl": [ { "disease": "Cardiovascular Diseases", "phase": 4.0 }, { "disease": "Myocardial Infarction", "phase": 3.0 }, { "disease": "Dementia", "phase": 2.0 }, { "disease": "Premature Birth", "phase": 2.0 }, { "disease": "Coronary Disease", "phase": 2.0 }, { "disease": "Cognitive Dysfunction", "phase": 2.0 }, { "disease": "Radiation Pneumonitis", "phase": 2.0 } ], "disease_terms": "Cardiovascular Diseases", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "PM5LI0P38J", "name": "NIFUROXAZIDE", "moiety": "NIFUROXAZIDE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Nifuroxazide" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 5, "countries": [ "Bulgaria", "Czech Republic", "Germany", "Greece", "Poland" ], "pubmed": 4, "trials": 3, "us_orphan": "unknown", "chembl": [ { "disease": "Brain Diseases", "phase": 3.0 }, { "disease": "Inflammatory Bowel Diseases", "phase": 2.0 } ], "disease_terms": "Brain Diseases", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "A8M33244M6", "name": "NITROXOLINE", "moiety": "NITROXOLINE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Nitroxoline" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 5, "countries": [ "Bulgaria", "Croatia", "Germany", "Poland", "Romania" ], "pubmed": 0, "trials": 0, "us_orphan": "unknown", "chembl": [ { "disease": "Bacterial Infections", "phase": 4.0 } ], "disease_terms": "Bacterial Infections", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "42NCM1BW43", "name": "SULBUTIAMINE", "moiety": "SULBUTIAMINE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Sulbutiamine" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 5, "countries": [ "France", "Malta", "Portugal", "Romania", "Spain" ], "pubmed": 1, "trials": 1, "us_orphan": "unknown", "chembl": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "3G943U18KM", "name": "TOLFENAMIC ACID", "moiety": "TOLFENAMIC", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Tolfenamic Acid" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 5, "countries": [ "Denmark", "Finland", "Greece", "Norway", "Poland" ], "pubmed": 4, "trials": 2, "us_orphan": "unknown", "chembl": [], "orpha": null, "eu_orphan_designation": { "use": "treatment of behavioural variant frontotemporal dementia", "status": "Positive", "number": "EU/3/16/1614" }, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] }, "eu_orphan_designations": [ { "use": "treatment of behavioural variant frontotemporal dementia", "status": "Positive", "number": "EU/3/16/1614" }, { "use": "Treatment of progressive supranuclear palsy", "status": "Positive", "number": "EU/3/16/1613" } ] }, { "unii": "K9AY9IR2SD", "name": "ACIPIMOX", "moiety": "ACIPIMOX", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Acipimox" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 4, "countries": [ "Denmark", "Italy", "Netherlands", "United Kingdom (Northern Ireland)" ], "pubmed": 1, "trials": 25, "us_orphan": "unknown", "chembl": [ { "disease": "Cardiovascular Diseases", "phase": 4.0 }, { "disease": "Diabetes Mellitus, Type 1", "phase": 3.0 }, { "disease": "Metabolic Syndrome", "phase": 2.0 }, { "disease": "Heart Failure", "phase": 2.0 }, { "disease": "Bulimia Nervosa", "phase": 2.0 }, { "disease": "Diabetes Mellitus, Type 2", "phase": 2.0 }, { "disease": "Feeding and Eating Disorders", "phase": 2.0 } ], "disease_terms": "Cardiovascular Diseases", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "7LP2MPO46S", "name": "ADEMETIONINE", "moiety": "ADEMETIONINE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Ademetionine" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['UNAPPROVED HOMEOPATHIC']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 4, "countries": [ "Bulgaria", "Czech Republic", "Italy", "Lithuania" ], "pubmed": 0, "trials": 69, "us_orphan": "unknown", "chembl": [ { "disease": "Non-alcoholic Fatty Liver Disease", "phase": 3.0 }, { "disease": "Cholestasis, Intrahepatic", "phase": 3.0 }, { "disease": "Liver Diseases", "phase": 3.0 }, { "disease": "Hepatitis", "phase": 3.0 }, { "disease": "Liver Cirrhosis, Biliary", "phase": 2.0 }, { "disease": "Depressive Disorder, Major", "phase": 2.0 }, { "disease": "Depressive Disorder", "phase": 2.0 }, { "disease": "Liver Cirrhosis, Alcoholic", "phase": 2.0 }, { "disease": "Hepatitis C", "phase": 2.0 }, { "disease": "Tobacco Use Disorder", "phase": 2.0 }, { "disease": "Hyperhomocysteinemia", "phase": 1.0 } ], "disease_terms": "Non-alcoholic Fatty Liver Disease; Cholestasis, Intrahepatic; Liver Diseases; Hepatitis", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "P55703ZRZY", "name": "ALIZAPRIDE", "moiety": "ALIZAPRIDE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Alizapride", "Alizapride Hydrochloride" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 4, "countries": [ "Belgium", "France", "Italy", "Luxembourg" ], "pubmed": 3, "trials": 3, "us_orphan": "unknown", "chembl": [ { "disease": "Digestive System Diseases", "phase": 3.0 } ], "disease_terms": "Digestive System Diseases", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "T3CHA1B51H", "name": "ANTIPYRINE", "moiety": "PHENAZONE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Phenazone" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT', 'UNAPPROVED HOMEOPATHIC']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 4, "countries": [ "Germany", "Ireland", "Lithuania", "Malta" ], "pubmed": 2, "trials": 47, "us_orphan": "unknown", "chembl": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "2VBY96ASWJ", "name": "BARNIDIPINE", "moiety": "BARNIDIPINE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Barnidipine Hydrochloride" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 4, "countries": [ "Italy", "Netherlands", "Poland", "Spain" ], "pubmed": 0, "trials": 2, "us_orphan": "unknown", "chembl": [ { "disease": "Cardiovascular Diseases", "phase": 2.0 } ], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "W4K0AE8XW9", "name": "BICLOTYMOL", "moiety": "BICLOTYMOL", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Biclotymol" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 4, "countries": [ "France", "Latvia", "Lithuania", "Luxembourg" ], "pubmed": 0, "trials": 0, "us_orphan": "unknown", "chembl": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "LYH6F7I22E", "name": "BROMPERIDOL", "moiety": "BROMPERIDOL", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Bromperidol", "Bromperidol Decanoate", "Bromperidol Lactate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 4, "countries": [ "Belgium", "Germany", "Luxembourg", "Netherlands" ], "pubmed": 0, "trials": 3, "us_orphan": "unknown", "chembl": [ { "disease": "Psychotic Disorders", "phase": 4.0 }, { "disease": "Anxiety", "phase": 3.0 }, { "disease": "Dementia", "phase": 3.0 }, { "disease": "Depressive Disorder", "phase": 3.0 }, { "disease": "Schizophrenia", "phase": 3.0 } ], "disease_terms": "Psychotic Disorders", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "MM6384NG73", "name": "CARBENOXOLONE", "moiety": "CARBENOXOLONE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Carbenoxolone Sodium" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 4, "countries": [ "Hungary", "Ireland", "Lithuania", "Malta" ], "pubmed": 19, "trials": 0, "us_orphan": "unknown", "chembl": [ { "disease": "Gastroesophageal Reflux", "phase": 4.0 }, { "disease": "Peptic Ulcer", "phase": 4.0 } ], "disease_terms": "Gastroesophageal Reflux; Peptic Ulcer", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "R8I97I2L24", "name": "CINITAPRIDE", "moiety": "CINITAPRIDE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Cinitapride", "Cinitapride Tartrate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 4, "countries": [ "Croatia", "Czech Republic", "Malta", "Spain" ], "pubmed": 1, "trials": 1, "us_orphan": "unknown", "chembl": [ { "disease": "Dyspepsia", "phase": 3.0 }, { "disease": "Digestive System Diseases", "phase": 3.0 } ], "disease_terms": "Dyspepsia; Digestive System Diseases", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "LT69WY1J6D", "name": "CLOBETASONE", "moiety": "CLOBETASONE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Clobetasone Butyrate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 4, "countries": [ "Ireland", "Italy", "Sweden", "United Kingdom (Northern Ireland)" ], "pubmed": 3, "trials": 5, "us_orphan": "unknown", "chembl": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "69M5L7BXEK", "name": "CLOPERASTINE", "moiety": "CLOPERASTINE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Cloperastine Hydrochloride" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 4, "countries": [ "Belgium", "Italy", "Luxembourg", "Spain" ], "pubmed": 4, "trials": 0, "us_orphan": "unknown", "chembl": [ { "disease": "Cough", "phase": 2.0 } ], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "ZCN055599V", "name": "CLOTIAZEPAM", "moiety": "CLOTIAZEPAM", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Clotiazepam" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 4, "countries": [ "Belgium", "France", "Italy", "Luxembourg" ], "pubmed": 0, "trials": 1, "us_orphan": "unknown", "chembl": [ { "disease": "Anxiety", "phase": 4.0 } ], "disease_terms": "Anxiety", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "4HU6J11EL5", "name": "DIACEREIN", "moiety": "DIACEREIN", "inn": true, "usan": true, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Diacerein" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 4, "countries": [ "Austria", "France", "Greece", "Italy" ], "pubmed": 4, "trials": 39, "us_orphan": "unknown", "chembl": [ { "disease": "Rheumatic Diseases", "phase": 4.0 }, { "disease": "Osteoarthritis, Hip", "phase": 3.0 }, { "disease": "Osteoarthritis, Knee", "phase": 3.0 }, { "disease": "Osteoarthritis", "phase": 3.0 }, { "disease": "Arthritis, Rheumatoid", "phase": 2.0 }, { "disease": "Gout", "phase": 2.0 }, { "disease": "Thrombocytopenia", "phase": 2.0 }, { "disease": "Diabetes Mellitus, Type 2", "phase": 2.0 }, { "disease": "Epidermolysis Bullosa Simplex", "phase": 2.0 }, { "disease": "Pemphigoid, Bullous", "phase": 2.0 }, { "disease": "Epidermolysis Bullosa", "phase": 2.0 }, { "disease": "Epidermolysis Bullosa Dystrophica", "phase": 1.0 }, { "disease": "Epidermolysis Bullosa, Junctional", "phase": 1.0 } ], "disease_terms": "Rheumatic Diseases", "orpha": null, "eu_orphan_designation": { "use": "Treatment of epidermolysis bullosa", "status": "Positive", "number": "EU/3/14/1236" }, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] }, "eu_orphan_designations": [ { "use": "Treatment of epidermolysis bullosa", "status": "Positive", "number": "EU/3/14/1236" }, { "use": "Treatment of epidermolysis bullosa", "status": "Positive", "number": "EU/3/19/2161" }, { "use": "Treatment of epidermolysis bullosa", "status": "Negative", "number": "-" } ] }, { "unii": "269M3QL74S", "name": "DIBROMPROPAMIDINE", "moiety": "DIBROMPROPAMIDINE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Dibrompropamidine Diisetionate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 4, "countries": [ "Cyprus", "Ireland", "Malta", "United Kingdom (Northern Ireland)" ], "pubmed": 0, "trials": 1, "us_orphan": "unknown", "chembl": [ { "disease": "Eye Infections", "phase": 2.0 } ], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "VJB5FW9W9J", "name": "ETHYL LOFLAZEPATE", "moiety": "ETHYL LOFLAZEPATE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Ethyl Loflazepate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 4, "countries": [ "Belgium", "France", "Luxembourg", "Portugal" ], "pubmed": 0, "trials": 0, "us_orphan": "unknown", "chembl": [ { "disease": "Anxiety", "phase": 2.0 } ], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "22M9P70OQ9", "name": "FENOTEROL", "moiety": "FENOTEROL", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Fenoterol Hydrobromide" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT FOR HUMAN PRESCRIPTION COMPOUNDING']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 4, "countries": [ "Estonia", "Germany", "Lithuania", "Poland" ], "pubmed": 12, "trials": 25, "us_orphan": "unknown", "chembl": [ { "disease": "Lung Diseases, Obstructive", "phase": 4.0 }, { "disease": "Pulmonary Disease, Chronic Obstructive", "phase": 3.0 }, { "disease": "Asthma", "phase": 3.0 }, { "disease": "Heart Failure", "phase": 1.0 } ], "disease_terms": "Lung Diseases, Obstructive", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "C5QA4GLR9M", "name": "FLUSPIRILENE", "moiety": "FLUSPIRILENE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Fluspirilene" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 4, "countries": [ "Belgium", "Germany", "Luxembourg", "Netherlands" ], "pubmed": 0, "trials": 19, "us_orphan": "unknown", "chembl": [ { "disease": "Psychotic Disorders", "phase": 4.0 }, { "disease": "Anxiety", "phase": 3.0 }, { "disease": "Dementia", "phase": 3.0 }, { "disease": "Depressive Disorder", "phase": 3.0 }, { "disease": "Schizophrenia", "phase": 3.0 } ], "disease_terms": "Psychotic Disorders", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "G9L6B3W684", "name": "HEXOPRENALINE", "moiety": "HEXOPRENALINE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Hexoprenaline Sulfate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 4, "countries": [ "Austria", "Bulgaria", "Czech Republic", "Romania" ], "pubmed": 1, "trials": 0, "us_orphan": "unknown", "chembl": [ { "disease": "Lung Diseases, Obstructive", "phase": 2.0 } ], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "3T5NG4Q468", "name": "HYMECROMONE", "moiety": "HYMECROMONE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Hymecromone" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 4, "countries": [ "Czech Republic", "France", "Poland", "Slovakia" ], "pubmed": 0, "trials": 6, "us_orphan": "unknown", "chembl": [ { "disease": "Cholangitis, Sclerosing", "phase": 2.0 }, { "disease": "Hypertension, Pulmonary", "phase": 2.0 }, { "disease": "Respiratory Tract Diseases", "phase": 1.0 } ], "orpha": null, "eu_orphan_designation": { "use": "Treatment of spinal cord injury", "status": "Positive", "number": "EU/3/22/2584" }, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] }, "eu_orphan_designations": [ { "use": "Treatment of spinal cord injury", "status": "Positive", "number": "EU/3/22/2584" } ] }, { "unii": "6T9949G4LZ", "name": "INDOBUFEN", "moiety": "INDOBUFEN", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Indobufen" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 4, "countries": [ "Czech Republic", "Italy", "Portugal", "Slovakia" ], "pubmed": 0, "trials": 14, "us_orphan": "unknown", "chembl": [ { "disease": "Thrombosis", "phase": 3.0 }, { "disease": "Atrial Fibrillation", "phase": 3.0 } ], "disease_terms": "Thrombosis; Atrial Fibrillation", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "C1LJO185Q9", "name": "OXITRIPTAN", "moiety": "OXITRIPTAN", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Oxitriptan" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['UNAPPROVED HOMEOPATHIC']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 4, "countries": [ "France", "Italy", "Lithuania", "Portugal" ], "pubmed": 0, "trials": 30, "us_orphan": "unknown", "chembl": [ { "disease": "Depressive Disorder", "phase": 4.0 }, { "disease": "Sleep Wake Disorders", "phase": 3.0 }, { "disease": "Spinal Cord Injuries", "phase": 2.0 }, { "disease": "Depressive Disorder, Major", "phase": 2.0 }, { "disease": "Obesity", "phase": 2.0 } ], "disease_terms": "Depressive Disorder", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "2H52Z9F2Q5", "name": "PEFLOXACIN", "moiety": "PEFLOXACIN", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Pefloxacin", "Pefloxacin Mesilate", "Pefloxacin Mesilate Dihydrate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 4, "countries": [ "Czech Republic", "Greece", "Romania", "Slovakia" ], "pubmed": 12, "trials": 2, "us_orphan": "unknown", "chembl": [ { "disease": "Bacterial Infections", "phase": 4.0 } ], "disease_terms": "Bacterial Infections", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "3405M6FD73", "name": "PERICIAZINE", "moiety": "PERICIAZINE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Periciazine" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 4, "countries": [ "France", "Italy", "Spain", "United Kingdom (Northern Ireland)" ], "pubmed": 1, "trials": 1, "us_orphan": "unknown", "chembl": [ { "disease": "Psychotic Disorders", "phase": 4.0 } ], "disease_terms": "Psychotic Disorders", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "GXM4PER6WZ", "name": "PROMESTRIENE", "moiety": "PROMESTRIENE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Promestriene" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 4, "countries": [ "France", "Italy", "Portugal", "Spain" ], "pubmed": 0, "trials": 10, "us_orphan": "unknown", "chembl": [ { "disease": "Hypospadias", "phase": 3.0 } ], "disease_terms": "Hypospadias", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "5O6VWA87VA", "name": "PROTHIPENDYL", "moiety": "PROTHIPENDYL", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Prothipendyl Hydrochloride", "Prothipendyl Hydrochloride Monohydrate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 4, "countries": [ "Austria", "Belgium", "Germany", "Luxembourg" ], "pubmed": 0, "trials": 2, "us_orphan": "unknown", "chembl": [ { "disease": "Psychotic Disorders", "phase": 4.0 }, { "disease": "Anxiety", "phase": 3.0 }, { "disease": "Dementia", "phase": 3.0 }, { "disease": "Depressive Disorder", "phase": 3.0 }, { "disease": "Schizophrenia", "phase": 3.0 } ], "disease_terms": "Psychotic Disorders", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "06KB629TKV", "name": "TOLNAFTATE", "moiety": "TOLNAFTATE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Tolnaftate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT', 'DRUG FOR FURTHER PROCESSING', 'OTC MONOGRAPH DRUG', 'OTC MONOGRAPH FINAL', 'UNAPPROVED DRUG OTHER', 'UNAPPROVED HOMEOPATHIC']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 4, "countries": [ "Germany", "Hungary", "Ireland", "United Kingdom (Northern Ireland)" ], "pubmed": 1, "trials": 34, "us_orphan": "unknown", "chembl": [ { "disease": "Infections", "phase": 4.0 }, { "disease": "Tinea", "phase": 4.0 }, { "disease": "Tinea Pedis", "phase": 4.0 }, { "disease": "Pruritus", "phase": 4.0 }, { "disease": "Eczema", "phase": 3.0 } ], "disease_terms": "Infections; Tinea; Tinea Pedis; Pruritus", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "RSA8KO39WH", "name": "VINDESINE", "moiety": "VINDESINE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Vindesine Sulfate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 4, "countries": [ "France", "Germany", "Italy", "Spain" ], "pubmed": 26, "trials": 85, "us_orphan": "unknown", "chembl": [ { "disease": "Neoplasms", "phase": 4.0 }, { "disease": "Precursor Cell Lymphoblastic Leukemia-Lymphoma", "phase": 3.0 }, { "disease": "Lymphoma, Large B-Cell, Diffuse", "phase": 3.0 }, { "disease": "Leukemia", "phase": 3.0 }, { "disease": "Lymphoma", "phase": 3.0 }, { "disease": "Neuroblastoma", "phase": 3.0 }, { "disease": "Uterine Cervical Neoplasms", "phase": 3.0 }, { "disease": "Lung Neoplasms", "phase": 3.0 }, { "disease": "Leukemia-Lymphoma, Adult T-Cell", "phase": 3.0 }, { "disease": "Precursor T-Cell Lymphoblastic Leukemia-Lymphoma", "phase": 3.0 }, { "disease": "Lymphoma, T-Cell, Peripheral", "phase": 2.0 }, { "disease": "Small Cell Lung Carcinoma", "phase": 2.0 }, { "disease": "Lymphoma, Follicular", "phase": 2.0 }, { "disease": "Lymphoma, Non-Hodgkin", "phase": 1.0 } ], "disease_terms": "Neoplasms", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "BYK4592A3Q", "name": "AMBAZONE", "moiety": "AMBAZONE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Ambazone Monohydrate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 3, "countries": [ "Lithuania", "Poland", "Romania" ], "pubmed": 0, "trials": 0, "us_orphan": "unknown", "chembl": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "Y6BHZ28O92", "name": "BAMIPINE", "moiety": "BAMIPINE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Bamipine Lactate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 3, "countries": [ "Austria", "Germany", "Luxembourg" ], "pubmed": 0, "trials": 0, "us_orphan": "unknown", "chembl": [ { "disease": "Pruritus", "phase": 2.0 }, { "disease": "Hypersensitivity", "phase": 2.0 } ], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "4POG0RL69O", "name": "BENZBROMARONE", "moiety": "BENZBROMARONE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Benzbromarone" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 3, "countries": [ "Germany", "Netherlands", "Spain" ], "pubmed": 3, "trials": 13, "us_orphan": "unknown", "chembl": [ { "disease": "Gout", "phase": 4.0 }, { "disease": "Hyperuricemia", "phase": 2.0 }, { "disease": "Familial Primary Pulmonary Hypertension", "phase": 2.0 } ], "disease_terms": "Gout", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "9M363FGA7J", "name": "BORNAPRINE", "moiety": "BORNAPRINE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Bornaprine Hydrochloride" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 3, "countries": [ "Austria", "Germany", "Italy" ], "pubmed": 0, "trials": 0, "us_orphan": "unknown", "chembl": [ { "disease": "Parkinson Disease", "phase": 2.0 } ], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "I0A84520Y9", "name": "CLEBOPRIDE", "moiety": "CLEBOPRIDE", "inn": true, "usan": true, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Clebopride Hydrogen Maleate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 3, "countries": [ "Italy", "Portugal", "Spain" ], "pubmed": 0, "trials": 0, "us_orphan": "unknown", "chembl": [ { "disease": "Digestive System Diseases", "phase": 4.0 } ], "disease_terms": "Digestive System Diseases", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "Z05HCY0X1T", "name": "CLOTHIAPINE", "moiety": "CLOTIAPINE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Clotiapine" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 3, "countries": [ "Belgium", "Italy", "Spain" ], "pubmed": 1, "trials": 2, "us_orphan": "unknown", "chembl": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "48TCX9A1VT", "name": "CYSTINE", "moiety": "L CYSTINE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Cystine", "L-Cystine" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT', 'BULK INGREDIENT FOR HUMAN PRESCRIPTION COMPOUNDING', 'UNAPPROVED HOMEOPATHIC']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 3, "countries": [ "France", "Italy", "Spain" ], "pubmed": 4, "trials": 31, "us_orphan": "unknown", "chembl": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "WH41D8433D", "name": "ERGONOVINE", "moiety": "ERGOMETRINE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Ergometrine Maleate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 3, "countries": [ "Greece", "Romania", "United Kingdom (Northern Ireland)" ], "pubmed": 3, "trials": 27, "us_orphan": "unknown", "chembl": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "WIX85M1A6R", "name": "ETHACRIDINE", "moiety": "ETHACRIDINE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Ethacridine Lactate", "Ethacridine Lactate Monohydrate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 3, "countries": [ "Bulgaria", "Germany", "Poland" ], "pubmed": 0, "trials": 2, "us_orphan": "unknown", "chembl": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "X24X82MX4X", "name": "ETIFOXINE", "moiety": "ETIFOXINE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Etifoxine Hydrochloride" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 3, "countries": [ "Bulgaria", "France", "Malta" ], "pubmed": 0, "trials": 2, "us_orphan": "unknown", "chembl": [ { "disease": "Anxiety", "phase": 3.0 } ], "disease_terms": "Anxiety", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "FA517NS3N7", "name": "FLUPREDNIDENE", "moiety": "FLUPREDNIDENE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Fluprednidene Acetate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 3, "countries": [ "Austria", "Germany", "Luxembourg" ], "pubmed": 0, "trials": 0, "us_orphan": "unknown", "chembl": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "6CL9Y5YZEQ", "name": "NADIFLOXACIN", "moiety": "NADIFLOXACIN", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Nadifloxacin" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 3, "countries": [ "Germany", "Italy", "Spain" ], "pubmed": 1, "trials": 3, "us_orphan": "unknown", "chembl": [ { "disease": "Acne Vulgaris", "phase": 3.0 } ], "disease_terms": "Acne Vulgaris", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "15B0523P5L", "name": "NAFTAZONE", "moiety": "NAFTAZONE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Naftazone" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 3, "countries": [ "Belgium", "France", "Luxembourg" ], "pubmed": 0, "trials": 0, "us_orphan": "unknown", "chembl": [ { "disease": "Cardiovascular Diseases", "phase": 4.0 } ], "disease_terms": "Cardiovascular Diseases", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "D23ZXO613C", "name": "OPIPRAMOL", "moiety": "OPIPRAMOL", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Opipramol Hydrochloride" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 3, "countries": [ "Austria", "Germany", "Poland" ], "pubmed": 0, "trials": 3, "us_orphan": "unknown", "chembl": [ { "disease": "Anxiety", "phase": 3.0 }, { "disease": "Dementia", "phase": 3.0 }, { "disease": "Depressive Disorder", "phase": 3.0 }, { "disease": "Schizophrenia", "phase": 3.0 }, { "disease": "Substance-Related Disorders", "phase": 0.5 } ], "disease_terms": "Anxiety; Dementia; Depressive Disorder; Schizophrenia", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "62XCK0G93T", "name": "ORNIDAZOLE", "moiety": "ORNIDAZOLE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Ornidazole" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 3, "countries": [ "Belgium", "France", "Luxembourg" ], "pubmed": 6, "trials": 9, "us_orphan": "unknown", "chembl": [ { "disease": "Bacterial Infections", "phase": 4.0 }, { "disease": "Amebiasis", "phase": 4.0 }, { "disease": "Rectal Neoplasms", "phase": 3.0 }, { "disease": "Colonic Neoplasms", "phase": 3.0 } ], "disease_terms": "Bacterial Infections; Amebiasis", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "Q0XV76B96L", "name": "OXACEPROL", "moiety": "OXACEPROL", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Oxaceprol" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 3, "countries": [ "Croatia", "Germany", "Romania" ], "pubmed": 0, "trials": 0, "us_orphan": "unknown", "chembl": [ { "disease": "Rheumatic Diseases", "phase": 2.0 } ], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "EEO29FZT86", "name": "PADELIPORFIN", "moiety": "PADELIPORFIN", "inn": true, "usan": true, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Padeliporfin" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 3, "countries": [ "Iceland", "Liechtenstein", "Norway" ], "pubmed": 0, "trials": 12, "us_orphan": "unknown", "chembl": [ { "disease": "Neoplasms", "phase": 4.0 }, { "disease": "Carcinoma, Non-Small-Cell Lung", "phase": 2.0 }, { "disease": "Cholangiocarcinoma", "phase": 2.0 }, { "disease": "Prostatic Neoplasms, Castration-Resistant", "phase": 2.0 }, { "disease": "Urinary Bladder Neoplasms", "phase": 1.0 }, { "disease": "Kidney Neoplasms", "phase": 1.0 }, { "disease": "Prostatic Neoplasms", "phase": 1.0 } ], "disease_terms": "Neoplasms", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "G083B71P98", "name": "PENTETREOTIDE", "moiety": "PENTETREOTIDE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Pentetreotide" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 3, "countries": [ "Luxembourg", "Netherlands", "Portugal" ], "pubmed": 7, "trials": 15, "us_orphan": "unknown", "chembl": [ { "disease": "Neuroendocrine Tumors", "phase": 2.0 } ], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "4449F8I3LE", "name": "PRAMIRACETAM", "moiety": "PRAMIRACETAM", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Pramiracetam Sulfate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 3, "countries": [ "Latvia", "Lithuania", "Romania" ], "pubmed": 0, "trials": 0, "us_orphan": "unknown", "chembl": [ { "disease": "Attention Deficit Disorder with Hyperactivity", "phase": 2.0 } ], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "J42298IESW", "name": "PRULIFLOXACIN", "moiety": "PRULIFLOXACIN", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Prulifloxacin" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 3, "countries": [ "Greece", "Italy", "Portugal" ], "pubmed": 1, "trials": 8, "us_orphan": "unknown", "chembl": [ { "disease": "Bronchitis, Chronic", "phase": 3.0 }, { "disease": "Urinary Tract Infections", "phase": 3.0 }, { "disease": "Gastroenteritis", "phase": 3.0 }, { "disease": "Bacterial Infections", "phase": 3.0 }, { "disease": "Prostatitis", "phase": 2.0 } ], "disease_terms": "Bronchitis, Chronic; Urinary Tract Infections; Gastroenteritis; Bacterial Infections", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "1199LEX5N8", "name": "TERIZIDONE", "moiety": "TERIZIDONE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Terizidone" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 3, "countries": [ "Germany", "Latvia", "Lithuania" ], "pubmed": 3, "trials": 4, "us_orphan": "unknown", "chembl": [ { "disease": "Tuberculosis, Multidrug-Resistant", "phase": 2.0 }, { "disease": "Tuberculosis", "phase": 2.0 } ], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "1LS1T6R34C", "name": "TIAPROFENIC ACID", "moiety": "TIAPROFENIC", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Tiaprofenic Acid" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 3, "countries": [ "France", "Italy", "United Kingdom (Northern Ireland)" ], "pubmed": 1, "trials": 1, "us_orphan": "unknown", "chembl": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "40257685LM", "name": "AZIDAMFENICOL", "moiety": "AZIDAMFENICOL", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Azidamfenicol" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 2, "countries": [ "Cyprus", "Greece" ], "pubmed": 0, "trials": 0, "us_orphan": "unknown", "chembl": [ { "disease": "Eye Infections", "phase": 2.0 } ], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "6I97Z6S135", "name": "BENCYCLANE", "moiety": "BENCYCLANE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Bencyclane Fumarate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 2, "countries": [ "Hungary", "Poland" ], "pubmed": 0, "trials": 0, "us_orphan": "unknown", "chembl": [ { "disease": "Cardiovascular Diseases", "phase": 2.0 } ], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "97O6X78C53", "name": "BENPERIDOL", "moiety": "BENPERIDOL", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Benperidol" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 2, "countries": [ "Germany", "United Kingdom (Northern Ireland)" ], "pubmed": 2, "trials": 4, "us_orphan": "unknown", "chembl": [ { "disease": "Psychotic Disorders", "phase": 4.0 }, { "disease": "Anxiety", "phase": 3.0 }, { "disease": "Dementia", "phase": 3.0 }, { "disease": "Depressive Disorder", "phase": 3.0 }, { "disease": "Schizophrenia", "phase": 3.0 } ], "disease_terms": "Psychotic Disorders", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "SDS4N642GG", "name": "CHLORMADINONE", "moiety": "CHLORMADINONE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Chlormadinone Acetate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 2, "countries": [ "France", "Germany" ], "pubmed": 7, "trials": 4, "us_orphan": "unknown", "chembl": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "3UVD77BP8R", "name": "CHLORPHENOXAMINE", "moiety": "CHLORPHENOXAMINE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Chlorphenoxamine Hydrochloride" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 2, "countries": [ "Malta", "Portugal" ], "pubmed": 0, "trials": 0, "us_orphan": "unknown", "chembl": [ { "disease": "Pruritus", "phase": 4.0 }, { "disease": "Hypersensitivity", "phase": 4.0 } ], "disease_terms": "Pruritus; Hypersensitivity", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "D6VHC87LLS", "name": "CHLORQUINALDOL", "moiety": "CHLORQUINALDOL", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Chlorquinaldol" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 2, "countries": [ "Poland", "Romania" ], "pubmed": 0, "trials": 1, "us_orphan": "unknown", "chembl": [ { "disease": "Amebiasis", "phase": 4.0 } ], "disease_terms": "Amebiasis", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "60M22SGW66", "name": "CHOLINE ALFOSCERATE", "moiety": "CHOLINE ALFOSCERATE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Choline Alfoscerate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT', 'UNAPPROVED HOMEOPATHIC']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 2, "countries": [ "Italy", "Poland" ], "pubmed": 2, "trials": 28, "us_orphan": "unknown", "chembl": [ { "disease": "Diabetes Mellitus, Type 2", "phase": 3.0 }, { "disease": "Cognitive Dysfunction", "phase": 2.0 } ], "disease_terms": "Diabetes Mellitus, Type 2", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "17S83WON0I", "name": "CLOPAMIDE", "moiety": "CLOPAMIDE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Clopamide" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 2, "countries": [ "Hungary", "Poland" ], "pubmed": 1, "trials": 0, "us_orphan": "unknown", "chembl": [ { "disease": "Cardiovascular Diseases", "phase": 2.0 } ], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "7560BB0BO3", "name": "CLOROPHENE", "moiety": "CLOROFENE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Clorofene" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 2, "countries": [ "Belgium", "Luxembourg" ], "pubmed": 0, "trials": 0, "us_orphan": "unknown", "chembl": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "F0R1QK73KB", "name": "COBAMAMIDE", "moiety": "COBAMAMIDE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Cobamamide" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['UNAPPROVED DRUG OTHER']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 2, "countries": [ "Italy", "Portugal" ], "pubmed": 0, "trials": 3, "us_orphan": "unknown", "chembl": [ { "disease": "Anemia", "phase": 2.0 }, { "disease": "Dermatitis, Atopic", "phase": 2.0 } ], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "A2JGV5CNU4", "name": "CYAMEMAZINE", "moiety": "CYAMEMAZINE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Cyamemazine", "Cyamemazine Tartrate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 2, "countries": [ "France", "Portugal" ], "pubmed": 1, "trials": 1, "us_orphan": "unknown", "chembl": [ { "disease": "Psychotic Disorders", "phase": 2.0 } ], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "NH5000009I", "name": "DEHYDROCHOLIC ACID", "moiety": "DEHYDROCHOLIC", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Dehydrocholic Acid" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 2, "countries": [ "Poland", "Romania" ], "pubmed": 5, "trials": 0, "us_orphan": "unknown", "chembl": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "PCU411F5L6", "name": "DIHYDRALAZINE", "moiety": "DIHYDRALAZINE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Dihydralazine Mesilate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 2, "countries": [ "France", "Germany" ], "pubmed": 4, "trials": 2, "us_orphan": "unknown", "chembl": [ { "disease": "Hypertension", "phase": 3.0 }, { "disease": "Aortic Valve Stenosis", "phase": 3.0 } ], "disease_terms": "Hypertension; Aortic Valve Stenosis", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "L164909TBI", "name": "ETOFYLLINE", "moiety": "ETOFYLLINE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Etofylline" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 2, "countries": [ "Czech Republic", "Slovakia" ], "pubmed": 1, "trials": 0, "us_orphan": "unknown", "chembl": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "94WNJ5U8L7", "name": "FELBINAC", "moiety": "FELBINAC", "inn": true, "usan": true, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Felbinac" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 2, "countries": [ "Ireland", "United Kingdom (Northern Ireland)" ], "pubmed": 1, "trials": 6, "us_orphan": "unknown", "chembl": [ { "disease": "Myalgia", "phase": 4.0 }, { "disease": "Arthralgia", "phase": 4.0 }, { "disease": "Pain", "phase": 3.0 }, { "disease": "Thromboembolism", "phase": 3.0 }, { "disease": "Neoplasms", "phase": 2.0 }, { "disease": "Acute Kidney Injury", "phase": 1.0 } ], "disease_terms": "Myalgia; Arthralgia", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "X3FZE77O60", "name": "FENIPENTOL", "moiety": "FENIPENTOL", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Fenipentol" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 2, "countries": [ "Czech Republic", "Slovakia" ], "pubmed": 0, "trials": 0, "us_orphan": "unknown", "chembl": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "R8M46911LR", "name": "FLUBENDAZOLE", "moiety": "FLUBENDAZOLE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Flubendazole" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 2, "countries": [ "France", "Portugal" ], "pubmed": 2, "trials": 1, "us_orphan": "unknown", "chembl": [ { "disease": "Helminthiasis", "phase": 2.0 } ], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "EQ35YMS20Q", "name": "FLUINDIONE", "moiety": "FLUINDIONE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Fluindione" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 2, "countries": [ "France", "Luxembourg" ], "pubmed": 3, "trials": 14, "us_orphan": "unknown", "chembl": [ { "disease": "Thrombosis", "phase": 4.0 } ], "disease_terms": "Thrombosis", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "A571S2B3JE", "name": "GAXILOSE", "moiety": "GAXILOSE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Gaxilose" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 2, "countries": [ "Portugal", "Spain" ], "pubmed": 1, "trials": 1, "us_orphan": "unknown", "chembl": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "0Z802HMY7H", "name": "INDORAMIN", "moiety": "INDORAMIN", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Indoramin", "Indoramin Hydrochloride" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 2, "countries": [ "Ireland", "United Kingdom (Northern Ireland)" ], "pubmed": 0, "trials": 0, "us_orphan": "unknown", "chembl": [ { "disease": "Hypertension", "phase": 4.0 } ], "disease_terms": "Hypertension", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "903A9K181P", "name": "IPRAZOCHROME", "moiety": "IPRAZOCHROME", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Iprazochrome" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 2, "countries": [ "Hungary", "Poland" ], "pubmed": 0, "trials": 0, "us_orphan": "unknown", "chembl": [ { "disease": "Migraine Disorders", "phase": 2.0 } ], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "OCA4JT7PAW", "name": "LOFEPRAMINE", "moiety": "LOFEPRAMINE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Lofepramine", "Lofepramine Hydrochloride" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 2, "countries": [ "Ireland", "United Kingdom (Northern Ireland)" ], "pubmed": 0, "trials": 0, "us_orphan": "unknown", "chembl": [ { "disease": "Depressive Disorder", "phase": 4.0 } ], "disease_terms": "Depressive Disorder", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "G5B860B0G1", "name": "LUTETIUM LU-177 ZADAVOTIDE GURAXETAN", "moiety": "LUTETIUM 177LU ZADAVOTIDE GURAXETAN", "inn": true, "usan": true, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Lutetium (177lu) Zadavotide Guraxetan" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 2, "countries": [ "Austria", "Estonia" ], "pubmed": 0, "trials": 0, "us_orphan": "unknown", "chembl": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "1501393LY5", "name": "METERGOLINE", "moiety": "METERGOLINE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Metergoline" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 2, "countries": [ "Germany", "Italy" ], "pubmed": 0, "trials": 0, "us_orphan": "unknown", "chembl": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "S5969B6237", "name": "MEXAZOLAM", "moiety": "MEXAZOLAM", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Mexazolam" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 2, "countries": [ "Malta", "Portugal" ], "pubmed": 0, "trials": 0, "us_orphan": "unknown", "chembl": [ { "disease": "Anxiety", "phase": 2.0 } ], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "R133MWH7X1", "name": "MORNIFLUMATE", "moiety": "MORNIFLUMATE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Morniflumate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 2, "countries": [ "France", "Italy" ], "pubmed": 4, "trials": 0, "us_orphan": "unknown", "chembl": [ { "disease": "Rheumatic Diseases", "phase": 2.0 } ], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "25TLU22Q8H", "name": "PENFLURIDOL", "moiety": "PENFLURIDOL", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Penfluridol" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 2, "countries": [ "France", "Netherlands" ], "pubmed": 4, "trials": 1, "us_orphan": "unknown", "chembl": [ { "disease": "Psychotic Disorders", "phase": 2.0 } ], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "EFA30X554H", "name": "PHENETICILLIN", "moiety": "PHENETICILLIN", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Pheneticillin" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 2, "countries": [ "Belgium", "Netherlands" ], "pubmed": 0, "trials": 2, "us_orphan": "unknown", "chembl": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "LPP64AWZ7L", "name": "PHOLCODINE", "moiety": "PHOLCODINE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Pholcodine", "Pholcodine Monohydrate", "Pholcodine Ph. Eur." ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 2, "countries": [ "Ireland", "United Kingdom (Northern Ireland)" ], "pubmed": 2, "trials": 2, "us_orphan": "unknown", "chembl": [ { "disease": "Cough", "phase": 4.0 } ], "disease_terms": "Cough", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "362QBC4NL0", "name": "PIKETOPROFEN", "moiety": "PIKETOPROFEN", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Piketoprofen", "Piketoprofen Hydrochloride" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 2, "countries": [ "Portugal", "Spain" ], "pubmed": 0, "trials": 0, "us_orphan": "unknown", "chembl": [ { "disease": "Arthralgia", "phase": 2.0 }, { "disease": "Myalgia", "phase": 2.0 } ], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "27L0EP6IZK", "name": "PIRENOXINE", "moiety": "PIRENOXINE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Pirenoxine", "Pirenoxine Sodium" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 2, "countries": [ "Poland", "Portugal" ], "pubmed": 0, "trials": 0, "us_orphan": "unknown", "chembl": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "S491HH391H", "name": "PRENOXDIAZINE", "moiety": "PRENOXDIAZIN", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Prenoxdiazin Hydrochloride" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 2, "countries": [ "Bulgaria", "Hungary" ], "pubmed": 0, "trials": 0, "us_orphan": "unknown", "chembl": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "76YOO33643", "name": "PROTIONAMIDE", "moiety": "PROTIONAMIDE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Protionamide" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 2, "countries": [ "Germany", "Lithuania" ], "pubmed": 2, "trials": 15, "us_orphan": "unknown", "chembl": [ { "disease": "Tuberculosis", "phase": 4.0 }, { "disease": "Tuberculosis, Pulmonary", "phase": 3.0 } ], "disease_terms": "Tuberculosis", "orpha": { "code": "3389", "name": "Tuberculosis", "rarity": "rare", "rarity_evidence": "Orphanet 1-5 / 10 000 (Europe) \u2248 100,499 in the US, under 200,000", "us_prevalence_est": 100499, "onset": "mixed", "onset_evidence": "Orphanet age of onset: All ages \u2014 spans pediatric and adult" }, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "PASS", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "Orphanet 1-5 / 10 000 (Europe) \u2248 100,499 in the US, under 200,000" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "Orphanet age of onset: All ages \u2014 spans pediatric and adult" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "AK5Q5FZH2R", "name": "PYRITINOL", "moiety": "PYRITINOL", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Pyritinol Hydrochloride" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 2, "countries": [ "Portugal", "Romania" ], "pubmed": 0, "trials": 1, "us_orphan": "unknown", "chembl": [ { "disease": "Attention Deficit Disorder with Hyperactivity", "phase": 4.0 }, { "disease": "Anxiety", "phase": 3.0 }, { "disease": "Dementia", "phase": 3.0 }, { "disease": "Depressive Disorder", "phase": 3.0 }, { "disease": "Schizophrenia", "phase": 3.0 } ], "disease_terms": "Attention Deficit Disorder with Hyperactivity", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "W9A18RJ49B", "name": "QUIFENADINE", "moiety": "QUIFENADINE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Quifenadine Hydrochloride" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 2, "countries": [ "Latvia", "Lithuania" ], "pubmed": 0, "trials": 0, "us_orphan": "unknown", "chembl": [ { "disease": "Hypersensitivity", "phase": 2.0 } ], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "9164VT6ANB", "name": "SODIUM PICOFOSFATE", "moiety": "SODIUM PICOFOSFATE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Sodium Picofosfate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 2, "countries": [ "Lithuania", "United Kingdom (Northern Ireland)" ], "pubmed": 0, "trials": 0, "us_orphan": "unknown", "chembl": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "X81L78B3RB", "name": "SPAGLUMIC ACID", "moiety": "SPAGLUMIC", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Spaglumic Acid" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 2, "countries": [ "Portugal", "Spain" ], "pubmed": 0, "trials": 0, "us_orphan": "unknown", "chembl": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "Z7N0Y673NU", "name": "TRIBENOSIDE", "moiety": "TRIBENOSIDE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Tribenoside" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 2, "countries": [ "Czech Republic", "Slovakia" ], "pubmed": 2, "trials": 2, "us_orphan": "unknown", "chembl": [ { "disease": "Cardiovascular Diseases", "phase": 2.0 }, { "disease": "Hemorrhoids", "phase": 2.0 } ], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "IN1233R0JO", "name": "TRIMECAINE", "moiety": "TRIMECAINE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Trimecaine Hydrochloride" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 2, "countries": [ "Czech Republic", "Slovakia" ], "pubmed": 1, "trials": 1, "us_orphan": "unknown", "chembl": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "RUA6CWU76G", "name": "VALPROMIDE", "moiety": "VALPROMIDE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Valpromide" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 2, "countries": [ "France", "Italy" ], "pubmed": 9, "trials": 1, "us_orphan": "unknown", "chembl": [ { "disease": "Epilepsy", "phase": 2.0 } ], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "G54D0HMY25", "name": "VINBURNINE", "moiety": "VINBURNINE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Vinburnine" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 2, "countries": [ "Portugal", "Spain" ], "pubmed": 0, "trials": 0, "us_orphan": "unknown", "chembl": [ { "disease": "Cardiovascular Diseases", "phase": 2.0 } ], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "4S9EY0NUEC", "name": "XIPAMIDE", "moiety": "XIPAMIDE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Xipamide" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 2, "countries": [ "Germany", "United Kingdom (Northern Ireland)" ], "pubmed": 0, "trials": 0, "us_orphan": "unknown", "chembl": [ { "disease": "Cardiovascular Diseases", "phase": 4.0 } ], "disease_terms": "Cardiovascular Diseases", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "U29O83JAZW", "name": "ZOTEPINE", "moiety": "ZOTEPINE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Zotepine" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 2, "countries": [ "Portugal", "Slovakia" ], "pubmed": 0, "trials": 3, "us_orphan": "unknown", "chembl": [ { "disease": "Psychotic Disorders", "phase": 4.0 } ], "disease_terms": "Psychotic Disorders", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "3VQ38D63M7", "name": "ALFATRADIOL", "moiety": "ALFATRADIOL", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Alfatradiol" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT FOR HUMAN PRESCRIPTION COMPOUNDING']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Germany" ], "pubmed": 0, "trials": 0, "us_orphan": "unknown", "chembl": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "568Z59H7ZJ", "name": "ALMAGATE", "moiety": "ALMAGATE", "inn": true, "usan": true, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Almagate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Spain" ], "pubmed": 1, "trials": 0, "us_orphan": "unknown", "chembl": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "OZQ8O62H53", "name": "ALMASILATE", "moiety": "ALMASILATE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Almasilate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Germany" ], "pubmed": 0, "trials": 0, "us_orphan": "unknown", "chembl": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "TYR2U59WMA", "name": "AMITRIPTYLINOXIDE", "moiety": "AMITRIPTYLINOXIDE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Amitriptylinoxide Dihydrate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Germany" ], "pubmed": 0, "trials": 2, "us_orphan": "unknown", "chembl": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "08J2K08A3Y", "name": "ANDROSTANOLONE", "moiety": "ANDROSTANOLONE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Androstanolone" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "France" ], "pubmed": 1, "trials": 0, "us_orphan": "unknown", "chembl": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "5L16LKN964", "name": "ANIRACETAM", "moiety": "ANIRACETAM", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Aniracetam" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Greece" ], "pubmed": 0, "trials": 0, "us_orphan": "unknown", "chembl": [ { "disease": "Attention Deficit Disorder with Hyperactivity", "phase": 2.0 } ], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "U8CJK0JH5M", "name": "ANTHRALIN", "moiety": "DITHRANOL", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Dithranol Bp" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT FOR HUMAN PRESCRIPTION COMPOUNDING']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "United Kingdom (Northern Ireland)" ], "pubmed": 4, "trials": 6, "us_orphan": "unknown", "chembl": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "VLL71EBS9Z", "name": "ARLIPOIC ACID", "moiety": "LIPOIC", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Lipoic Acid Trometamol Salt" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Portugal" ], "pubmed": 42, "trials": 241, "us_orphan": "unknown", "chembl": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "3AA6IZ48YK", "name": "BENPROPERINE", "moiety": "BENPROPERINE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Benproperine Phosphate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Germany" ], "pubmed": 0, "trials": 0, "us_orphan": "unknown", "chembl": [ { "disease": "Cough", "phase": 2.0 } ], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "75473V2YZK", "name": "BROMOPRIDE", "moiety": "BROMOPRIDE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Bromopride" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Italy" ], "pubmed": 1, "trials": 1, "us_orphan": "unknown", "chembl": [ { "disease": "Dyspepsia", "phase": 3.0 }, { "disease": "Digestive System Diseases", "phase": 3.0 } ], "disease_terms": "Dyspepsia; Digestive System Diseases", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "6PU1E16C9W", "name": "BRONOPOL", "moiety": "BRONOPOL", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Bronopol" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Germany" ], "pubmed": 2, "trials": 4, "us_orphan": "unknown", "chembl": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "6931E8LH07", "name": "CARBALDRATE", "moiety": "CARBALDRATE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Carbaldrate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Germany" ], "pubmed": 0, "trials": 0, "us_orphan": "unknown", "chembl": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "81F061RQS4", "name": "CARBAZOCHROME", "moiety": "CARBAZOCHROME", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Carbazochrome" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Romania" ], "pubmed": 1, "trials": 1, "us_orphan": "unknown", "chembl": [ { "disease": "Hemorrhage", "phase": 2.0 } ], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "XJ73B0K6KB", "name": "CAROVERINE", "moiety": "CAROVERINE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Caroverine" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Austria" ], "pubmed": 0, "trials": 3, "us_orphan": "unknown", "chembl": [ { "disease": "Digestive System Diseases", "phase": 2.0 } ], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "8P4W949T8K", "name": "CEFATRIZINE", "moiety": "CEFATRIZINE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Cefatrizine" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Greece" ], "pubmed": 6, "trials": 2, "us_orphan": "unknown", "chembl": [ { "disease": "Bacterial Infections", "phase": 2.0 } ], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "HM4YQM8WRC", "name": "CHLOROBUTANOL", "moiety": "CHLOROBUTANOL", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Chlorobutanol Bp" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "United Kingdom (Northern Ireland)" ], "pubmed": 2, "trials": 7, "us_orphan": "unknown", "chembl": [ { "disease": "Nausea", "phase": 4.0 } ], "disease_terms": "Nausea", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "36W53O7109", "name": "CHLOROCRESOL", "moiety": "CHLOROCRESOL", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Chlorocresol" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Germany" ], "pubmed": 0, "trials": 2, "us_orphan": "unknown", "chembl": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "CHG7QC509W", "name": "CICLETANINE", "moiety": "CICLETANINE", "inn": true, "usan": true, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Cicletanine Hydrochloride" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "France" ], "pubmed": 0, "trials": 2, "us_orphan": "unknown", "chembl": [ { "disease": "Hypertension, Pulmonary", "phase": 2.0 }, { "disease": "Cardiovascular Diseases", "phase": 2.0 } ], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "Z7489237QT", "name": "CIFENLINE", "moiety": "CIBENZOLINE", "inn": true, "usan": true, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Cibenzoline Succinate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "France" ], "pubmed": 3, "trials": 3, "us_orphan": "unknown", "chembl": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "704083NI0R", "name": "CLOFOCTOL", "moiety": "CLOFOCTOL", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Clofoctol" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Italy" ], "pubmed": 0, "trials": 0, "us_orphan": "unknown", "chembl": [ { "disease": "Bacterial Infections", "phase": 2.0 } ], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "V7DXN0M42R", "name": "CLONIXIN", "moiety": "CLONIXIN", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Clonixin" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Portugal" ], "pubmed": 0, "trials": 1, "us_orphan": "unknown", "chembl": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "SYP56O3GJG", "name": "CLOPREDNOL", "moiety": "CLOPREDNOL", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Cloprednol" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Germany" ], "pubmed": 0, "trials": 0, "us_orphan": "unknown", "chembl": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "GYL649Z0HY", "name": "CLOXAZOLAM", "moiety": "CLOXAZOLAM", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Cloxazolam" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Portugal" ], "pubmed": 0, "trials": 0, "us_orphan": "unknown", "chembl": [ { "disease": "Anxiety", "phase": 2.0 } ], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "YM183K0H63", "name": "CORTIVAZOL", "moiety": "CORTIVAZOL", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Cortivazol" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "France" ], "pubmed": 2, "trials": 4, "us_orphan": "unknown", "chembl": [ { "disease": "Pain", "phase": 2.0 } ], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "O91W32476G", "name": "DELORAZEPAM", "moiety": "DELORAZEPAM", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Delorazepam" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Italy" ], "pubmed": 2, "trials": 1, "us_orphan": "unknown", "chembl": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "2N9HM3X95F", "name": "DEMOXYTOCIN", "moiety": "DEMOXYTOCIN", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Demoxytocin" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Latvia" ], "pubmed": 0, "trials": 0, "us_orphan": "unknown", "chembl": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "W656S9I00W", "name": "DENOTIVIR", "moiety": "DENOTIVIR", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Denotivir" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Poland" ], "pubmed": 0, "trials": 0, "us_orphan": "unknown", "chembl": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "772EN3BH6I", "name": "DIMETHISOQUIN", "moiety": "QUINISOCAINE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Quinisocaine Hydrochloride" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Germany" ], "pubmed": 0, "trials": 0, "us_orphan": "unknown", "chembl": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "MPM23GMO7Z", "name": "DOXOFYLLINE", "moiety": "DOXOFYLLINE", "inn": true, "usan": true, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Doxofylline" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT FOR HUMAN PRESCRIPTION COMPOUNDING']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Italy" ], "pubmed": 4, "trials": 7, "us_orphan": "unknown", "chembl": [ { "disease": "Lung Diseases, Obstructive", "phase": 3.0 }, { "disease": "Asthma", "phase": 3.0 } ], "disease_terms": "Lung Diseases, Obstructive; Asthma", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "V7O1U41C9B", "name": "EBERCONAZOLE", "moiety": "EBERCONAZOLE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Eberconazole" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Spain" ], "pubmed": 0, "trials": 0, "us_orphan": "unknown", "chembl": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "A76XI0HL37", "name": "ETIZOLAM", "moiety": "ETIZOLAM", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Etizolam" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Italy" ], "pubmed": 1, "trials": 1, "us_orphan": "unknown", "chembl": [ { "disease": "Anxiety", "phase": 2.0 } ], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "60GCX7Y6BH", "name": "FLUFENAMIC ACID", "moiety": "FLUFENAMIC", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Flufenamic Acid" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Germany" ], "pubmed": 3, "trials": 2, "us_orphan": "unknown", "chembl": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "M7522JYX1H", "name": "FOSFRUCTOSE", "moiety": "FRUCTOSE 1 6", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Fructose-1.6-Diphosphate Sodium Salt" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Italy" ], "pubmed": 35, "trials": 39, "us_orphan": "unknown", "chembl": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "GQ7JL9P5I2", "name": "FOTEMUSTINE", "moiety": "FOTEMUSTINE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Fotemustine" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "France" ], "pubmed": 4, "trials": 14, "us_orphan": "unknown", "chembl": [ { "disease": "Neoplasms", "phase": 3.0 }, { "disease": "Melanoma", "phase": 3.0 }, { "disease": "Uveal Melanoma", "phase": 3.0 }, { "disease": "Glioblastoma", "phase": 2.0 } ], "disease_terms": "Neoplasms; Melanoma; Uveal Melanoma", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "4BOC774388", "name": "FRAMYCETIN", "moiety": "FRAMYCETIN", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Framycetin Sulfate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Germany" ], "pubmed": 0, "trials": 2, "us_orphan": "unknown", "chembl": [ { "disease": "Nasal Obstruction", "phase": 3.0 }, { "disease": "Eye Infections", "phase": 3.0 }, { "disease": "Osteomyelitis", "phase": 0.5 } ], "disease_terms": "Nasal Obstruction; Eye Infections", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "7OW73204U1", "name": "FUMAGILLIN", "moiety": "FUMAGILLIN", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Fumagillin" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "France" ], "pubmed": 4, "trials": 0, "us_orphan": "unknown", "chembl": [ { "disease": "Amebiasis", "phase": 2.0 } ], "orpha": null, "eu_orphan_designation": { "use": "Treatment of diarrhoea associated with intestinal microsporidial infection", "status": "Withdrawn", "number": "EU/3/01/081" }, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] }, "eu_orphan_designations": [ { "use": "Treatment of diarrhoea associated with intestinal microsporidial infection", "status": "Withdrawn", "number": "EU/3/01/081" } ] }, { "unii": "4V7M9137X9", "name": "GABEXATE", "moiety": "GABEXATE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Gabexate Mesilate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Italy" ], "pubmed": 2, "trials": 9, "us_orphan": "unknown", "chembl": [ { "disease": "Liver Diseases", "phase": 3.0 } ], "disease_terms": "Liver Diseases", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "T3C89M417N", "name": "GLUTARAL", "moiety": "GLUTARAL", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Glutaral" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Germany" ], "pubmed": 0, "trials": 41, "us_orphan": "unknown", "chembl": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "3DQS188SY5", "name": "HEPTAMINOL", "moiety": "HEPTAMINOL", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Heptaminol Hydrochloride" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "France" ], "pubmed": 0, "trials": 1, "us_orphan": "unknown", "chembl": [ { "disease": "Cardiovascular Diseases", "phase": 2.0 } ], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "8ZCA2I2L11", "name": "IBOPAMINE", "moiety": "IBOPAMINE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Ibopamine Hydrochloride" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Italy" ], "pubmed": 0, "trials": 0, "us_orphan": "unknown", "chembl": [ { "disease": "Cardiovascular Diseases", "phase": 2.0 } ], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "D892HFI3XA", "name": "IPRONIAZID", "moiety": "IPRONIAZID", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Iproniazid Phosphate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "France" ], "pubmed": 0, "trials": 0, "us_orphan": "unknown", "chembl": [ { "disease": "Depressive Disorder", "phase": 4.0 } ], "disease_terms": "Depressive Disorder", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "WVZ7K9P0JY", "name": "ISOTHIPENDYL", "moiety": "ISOTHIPENDYL", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Isothipendyl Hydrochloride" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "France" ], "pubmed": 0, "trials": 0, "us_orphan": "unknown", "chembl": [ { "disease": "Pruritus", "phase": 2.0 }, { "disease": "Hypersensitivity", "phase": 2.0 } ], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "HV13HFS217", "name": "JOSAMYCIN", "moiety": "JOSAMYCIN", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Josamycin", "Josamycin Propionate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "France" ], "pubmed": 26, "trials": 1, "us_orphan": "unknown", "chembl": [ { "disease": "Premature Birth", "phase": 3.0 }, { "disease": "Bacterial Infections", "phase": 3.0 } ], "disease_terms": "Premature Birth; Bacterial Infections", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "EK22QV7701", "name": "LIDOFENIN", "moiety": "LIDOFENIN", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Lidofenin" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Hungary" ], "pubmed": 0, "trials": 1, "us_orphan": "unknown", "chembl": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "077DN93G5B", "name": "MEDROGESTONE", "moiety": "MEDROGESTONE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Medrogestone" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "France" ], "pubmed": 0, "trials": 0, "us_orphan": "unknown", "chembl": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "ZF3786Q2E8", "name": "MELARSOPROL", "moiety": "MELARSOPROL", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Melarsoprol" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "France" ], "pubmed": 3, "trials": 1, "us_orphan": "unknown", "chembl": [ { "disease": "Trypanosomiasis", "phase": 3.0 }, { "disease": "Leishmaniasis", "phase": 3.0 } ], "disease_terms": "Trypanosomiasis; Leishmaniasis", "orpha": null, "eu_orphan_designation": { "use": "Treatment of African trypanosomiasis", "status": "Positive", "number": "EU/3/12/1068" }, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] }, "eu_orphan_designations": [ { "use": "Treatment of African trypanosomiasis", "status": "Positive", "number": "EU/3/12/1068" } ] }, { "unii": "CZ87T54W8W", "name": "MEPHENOXALONE", "moiety": "MEPHENOXALONE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Mephenoxalone" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Czech Republic" ], "pubmed": 0, "trials": 18, "us_orphan": "unknown", "chembl": [ { "disease": "Anxiety", "phase": 2.0 } ], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "M0T18YH28D", "name": "MERBROMIN", "moiety": "MERBROMIN", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Merbromin" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Spain" ], "pubmed": 0, "trials": 1, "us_orphan": "unknown", "chembl": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "BR1SN1JS2W", "name": "METHYLCOBALAMIN", "moiety": "METHYLCOBALAMIN", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Methylcobalamin" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT', 'BULK INGREDIENT FOR HUMAN PRESCRIPTION COMPOUNDING', 'DRUG FOR FURTHER PROCESSING', 'UNAPPROVED DRUG OTHER', 'UNAPPROVED HOMEOPATHIC']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Cyprus" ], "pubmed": 17, "trials": 627, "us_orphan": "unknown", "chembl": [ { "disease": "Autistic Disorder", "phase": 2.0 }, { "disease": "Multiple Sclerosis", "phase": 1.0 } ], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "238S75V9AV", "name": "METOPIMAZINE", "moiety": "METOPIMAZINE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Metopimazine" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "France" ], "pubmed": 4, "trials": 2, "us_orphan": "unknown", "chembl": [ { "disease": "Nausea", "phase": 2.0 } ], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "PW8QYA7KI0", "name": "MOXISYLYTE", "moiety": "MOXISYLYTE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Moxisylyte Hydrochloride" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "France" ], "pubmed": 0, "trials": 0, "us_orphan": "unknown", "chembl": [ { "disease": "Cardiovascular Diseases", "phase": 4.0 }, { "disease": "Erectile Dysfunction", "phase": 4.0 } ], "disease_terms": "Cardiovascular Diseases; Erectile Dysfunction", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "8U27U3RIN4", "name": "NERIDRONIC ACID", "moiety": "NERIDRONATE", "inn": true, "usan": true, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Neridronate Sodium" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Italy" ], "pubmed": 13, "trials": 7, "us_orphan": "unknown", "chembl": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "368IVD6M32", "name": "NIKETHAMIDE", "moiety": "NIKETHAMIDE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Nikethamide" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Latvia" ], "pubmed": 0, "trials": 0, "us_orphan": "unknown", "chembl": [ { "disease": "Respiratory Tract Diseases", "phase": 4.0 } ], "disease_terms": "Respiratory Tract Diseases", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "469ULX0H4G", "name": "NIMORAZOLE", "moiety": "NIMORAZOLE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Nimorazole" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Norway" ], "pubmed": 0, "trials": 10, "us_orphan": "unknown", "chembl": [ { "disease": "Amebiasis", "phase": 4.0 }, { "disease": "Carcinoma, Squamous Cell", "phase": 3.0 } ], "disease_terms": "Amebiasis", "orpha": null, "eu_orphan_designation": { "use": "Treatment of squamous-cell carcinoma of the head and neck in patients undergoing radiotherapy", "status": "Withdrawn", "number": "EU/3/10/842" }, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] }, "eu_orphan_designations": [ { "use": "Treatment of squamous-cell carcinoma of the head and neck in patients undergoing radiotherapy", "status": "Withdrawn", "number": "EU/3/10/842" } ] }, { "unii": "S846B891OR", "name": "NONIVAMIDE", "moiety": "NONIVAMIDE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Nonivamide" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['OTC MONOGRAPH DRUG', 'UNAPPROVED DRUG OTHER']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Germany" ], "pubmed": 0, "trials": 2, "us_orphan": "unknown", "chembl": [ { "disease": "Low Back Pain", "phase": 3.0 } ], "disease_terms": "Low Back Pain", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "67220MCM01", "name": "NORDAZEPAM", "moiety": "NORDAZEPAM", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Nordazepam" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Italy" ], "pubmed": 0, "trials": 0, "us_orphan": "unknown", "chembl": [ { "disease": "Anxiety", "phase": 2.0 } ], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "P7W01638W6", "name": "NORETHANDROLONE", "moiety": "NORETHANDROLONE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Norethandrolone" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "France" ], "pubmed": 2, "trials": 1, "us_orphan": "unknown", "chembl": [ { "disease": "Leukemia, Myeloid, Acute", "phase": 3.0 } ], "disease_terms": "Leukemia, Myeloid, Acute", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "J31IL9Z2EE", "name": "OXATOMIDE", "moiety": "OXATOMIDE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Oxatomide", "Oxatomide Monohydrate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Italy" ], "pubmed": 10, "trials": 1, "us_orphan": "unknown", "chembl": [ { "disease": "Hypersensitivity", "phase": 4.0 }, { "disease": "Muscular Dystrophy, Duchenne", "phase": 2.0 } ], "disease_terms": "Hypersensitivity", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "T2M58D6LA8", "name": "PHENIBUT", "moiety": "4 AMINO 3 PHENYLBUTYRIC", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "4-Amino-3-Phenylbutyric Acid" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Latvia" ], "pubmed": 0, "trials": 2, "us_orphan": "unknown", "chembl": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "6QK969R2IF", "name": "PHENOLPHTHALEIN", "moiety": "PHENOLPHTHALEIN", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Phenolphthalein" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['UNAPPROVED HOMEOPATHIC']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Hungary" ], "pubmed": 4, "trials": 3, "us_orphan": "unknown", "chembl": [ { "disease": "Constipation", "phase": 4.0 } ], "disease_terms": "Constipation", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "707484X33X", "name": "PHENOTHRIN", "moiety": "PHENOTHRIN", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Phenothrin" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Greece" ], "pubmed": 2, "trials": 0, "us_orphan": "unknown", "chembl": [ { "disease": "Ectoparasitic Infestations", "phase": 4.0 } ], "disease_terms": "Ectoparasitic Infestations", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "4YC2PY3AEU", "name": "PICLOXYDINE", "moiety": "PICLOXYDINE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Picloxydine Dihydrochloride" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "France" ], "pubmed": 0, "trials": 0, "us_orphan": "unknown", "chembl": [ { "disease": "Eye Infections", "phase": 2.0 } ], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "785363R681", "name": "PIDOTIMOD", "moiety": "PIDOTIMOD", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Pidotimod" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Italy" ], "pubmed": 18, "trials": 1, "us_orphan": "unknown", "chembl": [ { "disease": "Neoplasms", "phase": 2.0 } ], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "5286RBZ882", "name": "PINAZEPAM", "moiety": "PINAZEPAM", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Pinazepam" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Italy" ], "pubmed": 0, "trials": 1, "us_orphan": "unknown", "chembl": [ { "disease": "Anxiety", "phase": 2.0 } ], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "LT12J5HVR8", "name": "PIPEMIDIC ACID", "moiety": "PIPEMIDIC", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Pipemidic Acid Trihydrate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Italy" ], "pubmed": 3, "trials": 0, "us_orphan": "unknown", "chembl": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "L903J9JPYV", "name": "PIPOTIAZINE", "moiety": "PIPOTIAZINE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Pipotiazine", "Pipotiazine Palmitate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "France" ], "pubmed": 0, "trials": 3, "us_orphan": "unknown", "chembl": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "3G0285N20N", "name": "PIRENZEPINE", "moiety": "PIRENZEPINE", "inn": true, "usan": true, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Pirenzepine Dihydrochloride Monohydrate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Germany" ], "pubmed": 7, "trials": 4, "us_orphan": "unknown", "chembl": [ { "disease": "Peptic Ulcer", "phase": 4.0 }, { "disease": "Gastroesophageal Reflux", "phase": 4.0 }, { "disease": "Peripheral Nervous System Diseases", "phase": 2.0 }, { "disease": "Diabetic Neuropathies", "phase": 2.0 }, { "disease": "Stomach Ulcer", "phase": 1.0 } ], "disease_terms": "Peptic Ulcer; Gastroesophageal Reflux", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "DQ6KK6GV93", "name": "PIRETANIDE", "moiety": "PIRETANIDE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Piretanide" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "France" ], "pubmed": 0, "trials": 0, "us_orphan": "unknown", "chembl": [ { "disease": "Cardiovascular Diseases", "phase": 4.0 } ], "disease_terms": "Cardiovascular Diseases", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "V39YPH45FZ", "name": "PIRLINDOLE", "moiety": "PIRLINDOLE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Pirlindole Hydrochloride" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Portugal" ], "pubmed": 0, "trials": 0, "us_orphan": "unknown", "chembl": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "7E8Q32N75N", "name": "PIROXICAM CINNAMATE", "moiety": "PIROXICAM CINNAMATE", "inn": false, "usan": true, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Piroxicam Cinnamate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Greece" ], "pubmed": 0, "trials": 0, "us_orphan": "unknown", "chembl": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "2R7O1ET613", "name": "PRANOPROFEN", "moiety": "PRANOPROFEN", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Pranoprofen" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Poland" ], "pubmed": 1, "trials": 8, "us_orphan": "unknown", "chembl": [ { "disease": "Eye Diseases", "phase": 2.0 } ], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "CY3RNB3K4T", "name": "PROFLAVINE", "moiety": "PROFLAVINE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Proflavine Hemisulfate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "United Kingdom (Northern Ireland)" ], "pubmed": 0, "trials": 22, "us_orphan": "unknown", "chembl": [ { "disease": "Carcinoma, Squamous Cell", "phase": 2.0 }, { "disease": "Barrett Esophagus", "phase": 2.0 }, { "disease": "Uterine Neoplasms", "phase": 2.0 }, { "disease": "Genital Neoplasms, Female", "phase": 0.5 }, { "disease": "Mouth Neoplasms", "phase": 0.5 } ], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "11941YC6RN", "name": "REPROTEROL", "moiety": "REPROTEROL", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Reproterol Hydrochloride" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Germany" ], "pubmed": 1, "trials": 1, "us_orphan": "unknown", "chembl": [ { "disease": "Lung Diseases, Obstructive", "phase": 4.0 }, { "disease": "Asthma", "phase": 2.0 } ], "disease_terms": "Lung Diseases, Obstructive", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "VI08KS44V0", "name": "ROCIVERINE", "moiety": "ROCIVERINE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Rociverine" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Italy" ], "pubmed": 0, "trials": 0, "us_orphan": "unknown", "chembl": [ { "disease": "Digestive System Diseases", "phase": 2.0 } ], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "Y521XM2900", "name": "RUFLOXACIN", "moiety": "RUFLOXACIN", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Rufloxacin Hydrochloride" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Italy" ], "pubmed": 0, "trials": 0, "us_orphan": "unknown", "chembl": [ { "disease": "Bacterial Infections", "phase": 2.0 } ], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "5G06TVY3R7", "name": "RUTIN", "moiety": "RUTOSIDE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Rutoside Trihydrate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['UNAPPROVED DRUG OTHER', 'UNAPPROVED HOMEOPATHIC']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Germany" ], "pubmed": 3, "trials": 55, "us_orphan": "unknown", "chembl": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "CKS1YQ9W1J", "name": "SODIUM AUROTIOSULFATE", "moiety": "SODIUM AUROTIOSULFATE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Sodium Aurotiosulfate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Germany" ], "pubmed": 0, "trials": 3, "us_orphan": "unknown", "chembl": [ { "disease": "Rheumatic Diseases", "phase": 2.0 } ], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "1W6L629B4K", "name": "SULISOBENZONE", "moiety": "SULISOBENZONE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Sulisobenzone" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Germany" ], "pubmed": 0, "trials": 0, "us_orphan": "unknown", "chembl": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "55FE6NPG89", "name": "TEMGICOLURIL", "moiety": "TEMGICOLURIL", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Temgicoluril" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Latvia" ], "pubmed": 0, "trials": 0, "us_orphan": "unknown", "chembl": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "0D771IS0FH", "name": "THIRAM", "moiety": "THIRAM", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Thiram" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Germany" ], "pubmed": 0, "trials": 0, "us_orphan": "unknown", "chembl": [ { "disease": "Ectoparasitic Infestations", "phase": 2.0 } ], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "H1187LU49Q", "name": "TREPIBUTONE", "moiety": "TREPIBUTONE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Trepibutone" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Portugal" ], "pubmed": 0, "trials": 0, "us_orphan": "unknown", "chembl": [ { "disease": "Digestive System Diseases", "phase": 2.0 } ], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "1M2IB31DTS", "name": "TRIMEPERIDINE", "moiety": "TRIMEPERIDINE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Trimeperidine Hydrochloride" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Latvia" ], "pubmed": 1, "trials": 3, "us_orphan": "unknown", "chembl": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "H64JRU6GJ0", "name": "TROFOSFAMIDE", "moiety": "TROFOSFAMIDE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Trofosfamide" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Germany" ], "pubmed": 11, "trials": 4, "us_orphan": "unknown", "chembl": [ { "disease": "Sarcoma", "phase": 3.0 }, { "disease": "Neoplasms", "phase": 3.0 }, { "disease": "Medulloblastoma", "phase": 2.0 }, { "disease": "Melanoma", "phase": 1.0 } ], "disease_terms": "Sarcoma; Neoplasms", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "H191JFG8WA", "name": "TROMANTADINE", "moiety": "TROMANTADINE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Tromantadine Hydrochloride" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Portugal" ], "pubmed": 0, "trials": 0, "us_orphan": "unknown", "chembl": [ { "disease": "Virus Diseases", "phase": 2.0 } ], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "C27HY5RFU5", "name": "TROPATEPINE", "moiety": "TROPATEPINE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Tropatepine", "Tropatepine Hydrochloride" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "France" ], "pubmed": 0, "trials": 0, "us_orphan": "unknown", "chembl": [ { "disease": "Parkinson Disease", "phase": 2.0 } ], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "996XVD0JHT", "name": "VINCAMINE", "moiety": "VINCAMINE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Vincamine" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Romania" ], "pubmed": 2, "trials": 0, "us_orphan": "unknown", "chembl": [ { "disease": "Cardiovascular Diseases", "phase": 4.0 } ], "disease_terms": "Cardiovascular Diseases", "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "RQ12GMY0FZ", "name": "XIBORNOL", "moiety": "XIBORNOL", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Xibornol" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Italy" ], "pubmed": 1, "trials": 0, "us_orphan": "unknown", "chembl": [ { "disease": "Bacterial Infections", "phase": 2.0 } ], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "K16AME9I3V", "name": "ATALUREN", "moiety": "ATALUREN", "inn": true, "usan": true, "type": "chemical", "sources": [ "EMA" ], "orphan": false, "tier": "exact-moiety", "examples": [ "ataluren" ], "area": "Muscular Dystrophy, Duchenne", "atc": "M09AX03", "indication": "Translarna is indicated for the treatment of Duchenne muscular dystrophy resulting from a nonsense mutation in the dystrophin gene, in ambulatory patients aged 2 years and older. Efficacy has not been demonstrated in non-ambulatory patients. The presence of a nonsense mutation in the dystrophin gene should be determined by genetic testing.", "approved": "31/07/2014", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "previously_authorised", "status_by_source": { "EMA": "previously_authorised" }, "n_countries": 0, "countries": [], "pubmed": 34, "trials": 34, "us_orphan": "unknown", "chembl": [ { "disease": "Muscular Dystrophy, Duchenne", "phase": 4.0 }, { "disease": "Cystic Fibrosis", "phase": 3.0 }, { "disease": "Epilepsy", "phase": 2.0 }, { "disease": "Hemophilia A", "phase": 2.0 }, { "disease": "Hemophilia B", "phase": 2.0 }, { "disease": "Aniridia", "phase": 2.0 } ], "orpha": { "code": "98896", "name": "Duchenne muscular dystrophy", "rarity": "rare", "rarity_evidence": "Orphanet 1-9 / 100 000 (Worldwide) \u2248 16,750 in the US, under 200,000", "us_prevalence_est": 16750, "onset": "pediatric", "onset_evidence": "Orphanet age of onset: Childhood \u2014 within birth-18" }, "eu_orphan_designation": { "use": "Treatment of Becker muscular dystrophy", "status": "Withdrawn", "number": "EU/3/12/1010" }, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "PASS", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "Orphanet 1-9 / 100 000 (Worldwide) \u2248 16,750 in the US, under 200,000" }, { "key": "primarily_pediatric", "status": "PASS", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "Orphanet age of onset: Childhood \u2014 within birth-18" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] }, "eu_orphan_designations": [ { "use": "Treatment of Becker muscular dystrophy", "status": "Withdrawn", "number": "EU/3/12/1010" }, { "use": "Treatment of aniridia", "status": "Withdrawn", "number": "EU/3/15/1561" }, { "use": "Treatment of mucopolysaccharidosis type I", "status": "Withdrawn", "number": "EU/3/14/1380" } ] }, { "unii": "T4RS462G19", "name": "AUMOLERTINIB", "moiety": "AUMOLERTINIB", "inn": true, "usan": true, "type": "chemical", "sources": [ "EMA" ], "orphan": false, "tier": "moiety-base", "examples": [ "Aumolertinib mesilate" ], "area": "Carcinoma, Non-Small-Cell Lung", "atc": "L01EB", "indication": "Aumseqa as monotherapy is indicated for:- the first-line treatment of adult patients with advanced non-small cell lung cancer (NSCLC)whose tumours have EGFR exon 19 deletions or exon 21 (L858R) substitution mutations (forbiomarker-based patient selection, see section 4.2).- the treatment of adult patients with advanced EGFR T790M mutation-positive NSCLC (forbiomarker-based patient selection, see section 4.2)", "approved": "12/02/2026", "flags": [], "foreign_status": "previously_authorised", "status_by_source": { "EMA": "previously_authorised" }, "n_countries": 0, "countries": [], "pubmed": 0, "trials": 80, "us_orphan": "unknown", "chembl": [ { "disease": "Carcinoma, Non-Small-Cell Lung", "phase": 3.0 }, { "disease": "Lung Neoplasms", "phase": 3.0 }, { "disease": "Neoplasms", "phase": 3.0 }, { "disease": "Carcinoma, Squamous Cell", "phase": 2.0 }, { "disease": "Carcinoma", "phase": 1.0 }, { "disease": "Liver Diseases", "phase": 1.0 } ], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "337G83N988", "name": "LASOFOXIFENE", "moiety": "LASOFOXIFENE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EMA" ], "orphan": false, "tier": "moiety-base", "examples": [ "Lasofoxifene tartrate" ], "area": "Osteoporosis, Postmenopausal", "atc": "G03", "indication": "Fablyn is indicated for the treatment of osteoporosis in postmenopausal women at increased risk of fracture. A significant reduction in the incidence of vertebral and non-vertebral fractures but not hip fractures has been demonstrated (see section 5.1). When determining the choice of Fablyn or other therapies, including oestrogens, for a postmenopausal woman, consideration should be given to menopausal symptoms, effects on uterine and breast tissues, and cardiovascular risks and benefits (see section 5.1).", "approved": "24/02/2009", "flags": [], "foreign_status": "previously_authorised", "status_by_source": { "EMA": "previously_authorised" }, "n_countries": 0, "countries": [], "pubmed": 0, "trials": 9, "us_orphan": "unknown", "chembl": [ { "disease": "Osteoporosis", "phase": 3.0 }, { "disease": "Breast Neoplasms", "phase": 3.0 }, { "disease": "Hemangiosarcoma", "phase": 2.0 } ], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "A2I8C7HI9T", "name": "METHYLPARABEN", "moiety": "E 218", "inn": false, "usan": true, "type": "chemical", "sources": [ "Swissmedic" ], "orphan": false, "tier": "exact-moiety", "examples": [ "E 218" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT FOR HUMAN PRESCRIPTION COMPOUNDING', 'OTC MONOGRAPH FINAL', 'UNAPPROVED HOMEOPATHIC']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "Swissmedic": "authorised" }, "n_countries": 0, "countries": [], "pubmed": 0, "trials": 0, "us_orphan": "unknown", "chembl": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "483AU1Y5CZ", "name": "PERFLENAPENT", "moiety": "DODECAFLUOROPENTANE", "inn": true, "usan": true, "type": "chemical", "sources": [ "EMA" ], "orphan": false, "tier": "exact-moiety", "examples": [ "dodecafluoropentane" ], "area": "Echocardiography", "atc": "V08DA", "indication": "EchoGen is a transpulmonary echocardiographic contrast agent for use in patients with suspected or established cardiovascular disease to provide opacification of cardiac chambers, enhance left ventricular border delineation with resulting improvement in wall motion visualisation. EchoGen should only be used in patients where the study without contrast enhancement is inconclusive.", "approved": "17/07/1998", "flags": [], "foreign_status": "previously_authorised", "status_by_source": { "EMA": "previously_authorised" }, "n_countries": 0, "countries": [], "pubmed": 0, "trials": 6, "us_orphan": "unknown", "chembl": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "F5SXN2KNMR", "name": "PIXANTRONE", "moiety": "PIXANTRONE", "inn": true, "usan": true, "type": "chemical", "sources": [ "EMA" ], "orphan": false, "tier": "moiety-base", "examples": [ "pixantrone dimaleate" ], "area": "Lymphoma, Non-Hodgkin", "atc": "L01DB11", "indication": "Pixuvri is indicated as monotherapy for the treatment of adult patients with multiply relapsed or refractory aggressive non-Hodgkin B-cell lymphomas (NHL). The benefit of pixantrone treatment has not been established in patients when used as fifth-line or greater chemotherapy in patients who are refractory to last therapy.", "approved": "10/05/2012", "flags": [], "foreign_status": "previously_authorised", "status_by_source": { "EMA": "previously_authorised" }, "n_countries": 0, "countries": [], "pubmed": 3, "trials": 18, "us_orphan": "unknown", "chembl": [ { "disease": "Neoplasms", "phase": 4.0 }, { "disease": "Lymphoma", "phase": 3.0 }, { "disease": "Lymphoma, Large B-Cell, Diffuse", "phase": 3.0 }, { "disease": "Lymphoma, Non-Hodgkin", "phase": 3.0 }, { "disease": "Lymphoma, Follicular", "phase": 3.0 }, { "disease": "Leukemia, Lymphocytic, Chronic, B-Cell", "phase": 1.0 }, { "disease": "Leukemia, Myeloid, Acute", "phase": 1.0 } ], "orpha": { "code": "547", "name": "Non-Hodgkin lymphoma", "rarity": "rare", "rarity_evidence": "Orphanet 1-5 / 10 000 (Europe) \u2248 100,499 in the US, under 200,000", "us_prevalence_est": 100499, "onset": "mixed", "onset_evidence": "Orphanet age of onset: Adolescent, Adult, Childhood, Elderly, Infancy \u2014 spans pediatric and adult" }, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "PASS", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "Orphanet 1-5 / 10 000 (Europe) \u2248 100,499 in the US, under 200,000" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "Orphanet age of onset: Adolescent, Adult, Childhood, Elderly, Infancy \u2014 spans pediatric and adult" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "RML78EN3XE", "name": "RIMONABANT", "moiety": "RIMONABANT", "inn": true, "usan": true, "type": "chemical", "sources": [ "EMA" ], "orphan": false, "tier": "exact-moiety", "examples": [ "rimonabant" ], "area": "Obesity", "atc": "A08AX01", "indication": "As an adjunct to diet and exercise for the treatment of obese patients (BMI 30 kg/m2), or overweight patients (BMI 27 kg/m2) with associated risk factor(s), such as type 2 diabetes or dyslipidaemia (see section 5.1).", "approved": "19/06/2006", "flags": [], "foreign_status": "previously_authorised", "status_by_source": { "EMA": "previously_authorised" }, "n_countries": 0, "countries": [], "pubmed": 10, "trials": 48, "us_orphan": "unknown", "chembl": [ { "disease": "Obesity", "phase": 4.0 }, { "disease": "Cardiovascular Diseases", "phase": 3.0 }, { "disease": "Metabolic Syndrome", "phase": 3.0 }, { "disease": "Fatty Liver", "phase": 3.0 }, { "disease": "Smoking Cessation", "phase": 3.0 }, { "disease": "Prediabetic State", "phase": 3.0 }, { "disease": "Lipid Metabolism Disorders", "phase": 3.0 }, { "disease": "Weight Loss", "phase": 3.0 }, { "disease": "Coronary Artery Disease", "phase": 3.0 }, { "disease": "Arteriosclerosis", "phase": 3.0 }, { "disease": "Diabetes Mellitus, Type 2", "phase": 3.0 }, { "disease": "Prader-Willi Syndrome", "phase": 3.0 }, { "disease": "Tobacco Use Disorder", "phase": 2.0 }, { "disease": "Alcohol Drinking", "phase": 2.0 }, { "disease": "Spinal Cord Injuries", "phase": 2.0 }, { "disease": "Pain", "phase": 1.0 }, { "disease": "Marijuana Abuse", "phase": 1.0 } ], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "J9QH779MEM", "name": "SITAXENTAN", "moiety": "SITAXENTAN", "inn": true, "usan": false, "type": "chemical", "sources": [ "EMA" ], "orphan": false, "tier": "moiety-base", "examples": [ "sitaxentan sodium" ], "area": "Hypertension, Pulmonary", "atc": "C02KX03", "indication": "Treatment of patients with pulmonary arterial hypertension (PAH) classified as WHO functional class III, to improve exercise capacity. Efficacy has been shown in primary pulmonary hypertension and in pulmonary hypertension associated with connective tissue disease.", "approved": "10/08/2006", "flags": [], "foreign_status": "previously_authorised", "status_by_source": { "EMA": "previously_authorised" }, "n_countries": 0, "countries": [], "pubmed": 10, "trials": 24, "us_orphan": "unknown", "chembl": [ { "disease": "Hypertension", "phase": 4.0 }, { "disease": "Hypertension, Pulmonary", "phase": 3.0 }, { "disease": "Renal Insufficiency, Chronic", "phase": 2.0 }, { "disease": "Proteinuria", "phase": 2.0 }, { "disease": "Asthma", "phase": 2.0 } ], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "PASS", "decidability": "data", "citation": "505(b)(2) reliance", "note": "published evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "FU21S769PF", "name": "TEMOPORFIN", "moiety": "TEMOPORFIN", "inn": true, "usan": true, "type": "chemical", "sources": [ "EMA" ], "orphan": false, "tier": "exact-moiety", "examples": [ "temoporfin" ], "area": "Head and Neck Neoplasms;Carcinoma, Squamous Cell", "atc": "L01XD05", "indication": "Foscan is indicated for the palliative treatment of patients with advanced head and neck squamous cell carcinoma failing prior therapies and unsuitable for radiotherapy, surgery or systemic chemotherapy.", "approved": "24/10/2001", "flags": [], "foreign_status": "authorised", "status_by_source": { "EMA": "authorised" }, "n_countries": 0, "countries": [], "pubmed": 0, "trials": 8, "us_orphan": "unknown", "chembl": [ { "disease": "Carcinoma, Squamous Cell", "phase": 4.0 }, { "disease": "Head and Neck Neoplasms", "phase": 4.0 }, { "disease": "Neoplasms", "phase": 4.0 }, { "disease": "Bile Duct Neoplasms", "phase": 2.0 }, { "disease": "Nasopharyngeal Carcinoma", "phase": 2.0 }, { "disease": "Carcinoma, Non-Small-Cell Lung", "phase": 1.0 }, { "disease": "Carcinoma", "phase": 1.0 } ], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } } ], "unnamed": [ { "unii": "40UB1H6Q6T", "name": "ETARFOLATIDE", "moiety": "ETARFOLATIDE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EMA" ], "orphan": true, "tier": "exact-moiety", "examples": [ "Etarfolatide" ], "area": "Ovarian Neoplasms;Radionuclide Imaging", "atc": "V09", "indication": "Indicated for single photon emission computed tomography (SPECT) imaging", "approved": "", "flags": [], "foreign_status": "never_authorised", "status_by_source": { "EMA": "never_authorised" }, "n_countries": 0, "countries": [], "orpha": null }, { "unii": "0GH9JX37C8", "name": "BUTYLSCOPOLAMINE BROMIDE", "moiety": "HYOSCINE BUTYLBROMIDE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)", "Health Canada" ], "orphan": false, "tier": "exact-moiety", "examples": [ "HYOSCINE BUTYLBROMIDE", "Hyoscine Butylbromide" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT', 'BULK INGREDIENT FOR HUMAN PRESCRIPTION COMPOUNDING']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised", "Health Canada": "authorised" }, "n_countries": 28, "countries": [ "Austria", "Belgium", "Bulgaria", "Croatia", "Cyprus", "Czech Republic", "Denmark", "Estonia", "Finland", "France", "Germany", "Greece", "Hungary", "Ireland", "Italy", "Latvia", "Luxembourg", "Malta", "Netherlands", "Norway", "Poland", "Portugal", "Romania", "Slovakia", "Slovenia", "Spain", "Sweden", "United Kingdom (Northern Ireland)" ], "orpha": null }, { "unii": "JL5DK93RCL", "name": "MELATONIN", "moiety": "MELATONIN", "inn": false, "usan": false, "type": "chemical", "sources": [ "EMA", "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Melatonin", "melatonin" ], "area": "Sleep Initiation and Maintenance Disorders;Autistic Disorder", "atc": "N05CH01", "indication": "Slenyto is indicated for: treatment of insomnia in children and adolescents aged 2-18 years with Autism Spectrum Disorder (ASD), and / or neurogenetic disorders with aberrant diurnal melatonin secretion and /or nocturnal awakenings, where sleep hygiene measures have been insufficient. treatment of insomnia in children and adolescents aged 6-17 years with attention-deficit hyperactivity disorder (ADHD) where sleep hygiene measures have been insufficient.", "approved": "20/09/2018", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT', 'BULK INGREDIENT FOR HUMAN PRESCRIPTION COMPOUNDING', 'UNAPPROVED HOMEOPATHIC']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EMA": "authorised", "EU national (Art57)": "authorised" }, "n_countries": 28, "countries": [ "Austria", "Belgium", "Cyprus", "Czech Republic", "Denmark", "Estonia", "Finland", "France", "Germany", "Greece", "Hungary", "Iceland", "Ireland", "Italy", "Latvia", "Liechtenstein", "Lithuania", "Luxembourg", "Malta", "Netherlands", "Norway", "Poland", "Portugal", "Slovakia", "Slovenia", "Spain", "Sweden", "United Kingdom (Northern Ireland)" ], "orpha": null, "eu_orphan_designation": { "use": "Treatment of non-24-hour sleep-wake disorders in blind people with no light perception", "status": "Withdrawn", "number": "EU/3/05/274" }, "eu_orphan_designations": [ { "use": "Treatment of perinatal asphyxia", "status": "Positive", "number": "EU/3/12/978" }, { "use": "Treatment of perinatal asphyxia", "status": "Positive", "number": "EU/3/23/2757" }, { "use": "Treatment of retinopathy of prematurity", "status": "Negative", "number": "-" }, { "use": "Prevention of spaceflight-related radiation and microgravity", "status": "Negative", "number": "N/A" }, { "use": "Treatment of pre-eclampsia", "status": "Positive", "number": "EU/3/21/2567" }, { "use": "Prevention of retinopathy of prematurity", "status": "Positive", "number": "EU/3/21/2453" }, { "use": "Treatment of retinitis pigmentosa", "status": "Positive", "number": "EU/3/21/2439" }, { "use": "Treatment of non-traumatic spontaneous intracerebral haemorrhage", "status": "Positive", "number": "EU/3/21/2468" }, { "use": "Treatment of intracerebral haemorrhage", "status": "Negative", "number": "-" }, { "use": "Treatment of perinatal asphyxia", "status": "Positive", "number": "EU/3/18/2127" }, { "use": "Treatment of acute radiation syndrome", "status": "Positive", "number": "EU/3/18/2077" }, { "use": "Treatment of Smith-Magenis syndrome", "status": "Positive", "number": "EU/3/16/1755" }, { "use": "Treatment of necrotising enterocolitis", "status": "Positive", "number": "EU/3/16/1682" }, { "use": "Treatment of neonatal sepsis", "status": "Positive", "number": "EU/3/16/1683" }, { "use": "Treatment of non-traumatic subarachnoid haemorrhage", "status": "Negative", "number": "-" }, { "use": "Treatment of neonatal encephalopathy", "status": "Positive", "number": "EU/3/18/1996" }, { "use": "Treatment of partial deep dermal and full-thickness burns", "status": "Negative", "number": "-" }, { "use": "Treatment of non-24-hour sleep-wake disorders in blind people with no light perception", "status": "Withdrawn", "number": "EU/3/05/274" } ] }, { "unii": "I38ZP9992A", "name": "MAGNESIUM", "moiety": "", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)", "Swissmedic" ], "orphan": false, "tier": "direct", "examples": [ "Magnesium", "magnesium" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['UNAPPROVED DRUG OTHER', 'UNAPPROVED HOMEOPATHIC']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised", "Swissmedic": "authorised" }, "n_countries": 18, "countries": [ "Austria", "Belgium", "Bulgaria", "Denmark", "Finland", "Germany", "Greece", "Hungary", "Ireland", "Latvia", "Lithuania", "Luxembourg", "Netherlands", "Norway", "Poland", "Slovenia", "Sweden", "United Kingdom (Northern Ireland)" ], "orpha": null }, { "unii": "1U477369SN", "name": "LUTETIUM CHLORIDE LU-177", "moiety": "LUTETIUM 177LU", "inn": false, "usan": false, "type": "chemical", "sources": [ "EMA", "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Lutetium (177lu) Chloride", "lutetium (177Lu) chloride" ], "area": "Radionuclide Imaging", "atc": "V10X", "indication": "Theralugand is a radiopharmaceutical precursor, and it is not intended for direct use in patients. It is to be used only for the radiolabelling of carrier molecules that have been specifically developed and authorised for radiolabelling with lutetium (177Lu) chloride.", "approved": "22/11/2024", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT FOR HUMAN PRESCRIPTION COMPOUNDING', 'DRUG FOR FURTHER PROCESSING']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EMA": "authorised", "EU national (Art57)": "authorised" }, "n_countries": 13, "countries": [ "Austria", "Belgium", "France", "Germany", "Iceland", "Italy", "Liechtenstein", "Netherlands", "Norway", "Portugal", "Spain", "Sweden", "United Kingdom (Northern Ireland)" ], "orpha": null }, { "unii": "CUB7JI0JV3", "name": "CETYLPYRIDINIUM", "moiety": "CETYLPYRIDINIUM", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)", "Health Canada" ], "orphan": false, "tier": "moiety-base", "examples": [ "CETYLPYRIDINIUM CHLORIDE", "Cetylpyridinium Chloride", "Cetylpyridinium Chloride Monohydrate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT', 'DRUG FOR FURTHER PROCESSING', 'OTC MONOGRAPH DRUG', 'UNAPPROVED DRUG OTHER']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised", "Health Canada": "authorised" }, "n_countries": 10, "countries": [ "Austria", "Czech Republic", "France", "Germany", "Italy", "Malta", "Norway", "Poland", "Slovenia", "United Kingdom (Northern Ireland)" ], "orpha": null }, { "unii": "J8L3BWC4BD", "name": "YTTRIUM CITRATE Y-90", "moiety": "YTTRIUM 90Y", "inn": false, "usan": false, "type": "chemical", "sources": [ "EMA", "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Yttrium (90y) Citrate", "yttrium (90Y) chloride" ], "area": "Radionuclide Imaging", "atc": "V09", "indication": "To be used only for the radiolabelling of carrier molecules, which have been specifically developed and authorised for radiolabelling with this radionuclide.Radiopharmaceutical precursor - Not intended for direct use in patients.", "approved": "19/01/2006", "flags": [], "foreign_status": "authorised", "status_by_source": { "EMA": "authorised", "EU national (Art57)": "authorised" }, "n_countries": 10, "countries": [ "Belgium", "Czech Republic", "France", "Germany", "Hungary", "Italy", "Luxembourg", "Netherlands", "Slovakia", "Spain" ], "orpha": null }, { "unii": "N20HL7Q941", "name": "CAMPHOR (NATURAL)", "moiety": "CAMPHOR", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)", "Health Canada", "Swissmedic" ], "orphan": false, "tier": "exact-moiety", "examples": [ "CAMPHOR", "Camphor", "D-Camphor" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT', 'DRUG FOR FURTHER PROCESSING', 'OTC MONOGRAPH DRUG', 'OTC MONOGRAPH NOT FINAL', 'UNAPPROVED DRUG OTHER', 'UNAPPROVED HOMEOPATHIC']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised", "Health Canada": "authorised", "Swissmedic": "authorised" }, "n_countries": 6, "countries": [ "Bulgaria", "Estonia", "Germany", "Italy", "Poland", "United Kingdom (Northern Ireland)" ], "orpha": null }, { "unii": "339NCG44TV", "name": "PHENOL", "moiety": "PHENOL", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)", "Health Canada" ], "orphan": false, "tier": "moiety-base", "examples": [ "PHENOL", "Phenol", "Phenol Bp" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT', 'DRUG FOR FURTHER PROCESSING', 'OTC MONOGRAPH DRUG', 'OTC MONOGRAPH NOT FINAL', 'UNAPPROVED HOMEOPATHIC']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised", "Health Canada": "authorised" }, "n_countries": 3, "countries": [ "Germany", "Italy", "United Kingdom (Northern Ireland)" ], "orpha": null }, { "unii": "FLI06WS32H", "name": "CHLORAL", "moiety": "CHLORAL", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)", "Health Canada" ], "orphan": false, "tier": "moiety-base", "examples": [ "CHLORAL HYDRATE", "Chloral Hydrate", "Chloral Hydrate Bp" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT', 'BULK INGREDIENT FOR HUMAN PRESCRIPTION COMPOUNDING']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised", "Health Canada": "authorised" }, "n_countries": 2, "countries": [ "Germany", "United Kingdom (Northern Ireland)" ], "orpha": null }, { "unii": "9NEZ333N27", "name": "SODIUM", "moiety": "", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)", "Swissmedic" ], "orphan": false, "tier": "direct", "examples": [ "Sodium", "natrium" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['OTC MONOGRAPH DRUG', 'UNAPPROVED DRUG OTHER', 'UNAPPROVED HOMEOPATHIC']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised", "Swissmedic": "authorised" }, "n_countries": 2, "countries": [ "France", "Spain" ], "orpha": null }, { "unii": "J41CSQ7QDS", "name": "ZINC", "moiety": "", "inn": false, "usan": false, "type": "chemical", "sources": [ "EMA", "EU national (Art57)" ], "orphan": false, "tier": "direct", "examples": [ "Zinc", "zinc" ], "area": "Hepatolenticular Degeneration", "atc": "A16AX05", "indication": "Treatment of Wilson's disease.", "approved": "12/10/2004", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['DRUG FOR FURTHER PROCESSING', 'UNAPPROVED DRUG OTHER', 'UNAPPROVED HOMEOPATHIC']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EMA": "authorised", "EU national (Art57)": "authorised" }, "n_countries": 2, "countries": [ "Germany", "Luxembourg" ], "orpha": null }, { "unii": "J8GF8BXI8S", "name": "LERCANIDIPINE, (S)-", "moiety": "LERCANIDIPINE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Lercanidipine", "Lercanidipine Hydrochloride", "Lercanidipine Hydrochloride Hemihydrate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 30, "countries": [ "Austria", "Belgium", "Bulgaria", "Croatia", "Cyprus", "Czech Republic", "Denmark", "Estonia", "Finland", "France", "Germany", "Greece", "Hungary", "Iceland", "Ireland", "Italy", "Latvia", "Lithuania", "Luxembourg", "Malta", "Netherlands", "Norway", "Poland", "Portugal", "Romania", "Slovakia", "Slovenia", "Spain", "Sweden", "United Kingdom (Northern Ireland)" ], "orpha": null }, { "unii": "J7P3696BCQ", "name": "FUSIDATE SODIUM", "moiety": "SODIUM FUSIDATE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Sodium Fusidate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 29, "countries": [ "Austria", "Belgium", "Bulgaria", "Cyprus", "Czech Republic", "Denmark", "Estonia", "Finland", "France", "Germany", "Greece", "Hungary", "Iceland", "Ireland", "Italy", "Latvia", "Lithuania", "Luxembourg", "Malta", "Netherlands", "Norway", "Poland", "Portugal", "Romania", "Slovakia", "Slovenia", "Spain", "Sweden", "United Kingdom (Northern Ireland)" ], "orpha": null }, { "unii": "U5JDO2770Z", "name": "ALANYL GLUTAMINE", "moiety": "ALANYL GLUTAMINE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Alanyl Glutamine" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 27, "countries": [ "Austria", "Belgium", "Bulgaria", "Croatia", "Czech Republic", "Denmark", "Estonia", "Finland", "France", "Germany", "Greece", "Hungary", "Ireland", "Italy", "Latvia", "Lithuania", "Luxembourg", "Netherlands", "Norway", "Poland", "Portugal", "Romania", "Slovakia", "Slovenia", "Spain", "Sweden", "United Kingdom (Northern Ireland)" ], "orpha": null }, { "unii": "E1UOL152H7", "name": "IRON", "moiety": "IRON", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Iron" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['DRUG FOR FURTHER PROCESSING', 'EXPORT ONLY', 'UNAPPROVED DRUG OTHER', 'UNAPPROVED HOMEOPATHIC']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 26, "countries": [ "Belgium", "Bulgaria", "Croatia", "Czech Republic", "Denmark", "Estonia", "France", "Germany", "Greece", "Hungary", "Ireland", "Italy", "Latvia", "Lithuania", "Luxembourg", "Malta", "Netherlands", "Norway", "Poland", "Portugal", "Romania", "Slovakia", "Slovenia", "Spain", "Sweden", "United Kingdom (Northern Ireland)" ], "orpha": null }, { "unii": "4109R0VM5R", "name": "SULPIRIDE, (R)-", "moiety": "SULPIRIDE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Sulpiride" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 25, "countries": [ "Austria", "Belgium", "Croatia", "Cyprus", "Czech Republic", "Estonia", "Finland", "France", "Germany", "Greece", "Hungary", "Ireland", "Italy", "Latvia", "Lithuania", "Luxembourg", "Malta", "Netherlands", "Poland", "Portugal", "Romania", "Slovakia", "Slovenia", "Spain", "United Kingdom (Northern Ireland)" ], "orpha": null }, { "unii": "E7QC7V26B8", "name": "DEQUALINIUM", "moiety": "DEQUALINIUM", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Dequalinium Chloride" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 23, "countries": [ "Austria", "Belgium", "Bulgaria", "Croatia", "Czech Republic", "Estonia", "Finland", "France", "Germany", "Hungary", "Italy", "Latvia", "Lithuania", "Luxembourg", "Norway", "Poland", "Portugal", "Romania", "Slovakia", "Slovenia", "Spain", "Sweden", "United Kingdom (Northern Ireland)" ], "orpha": null }, { "unii": "XBO8UII5SV", "name": "HYDRAZINONICOTINYL-TYR3-OCTREOTIDE", "moiety": "HYDRAZINONICOTINYL TYR3 OCTREOTIDE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Hydrazinonicotinyl-Tyr3-Octreotide Trifluoroacetate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 23, "countries": [ "Austria", "Belgium", "Bulgaria", "Cyprus", "Czech Republic", "Denmark", "Estonia", "Finland", "France", "Germany", "Greece", "Hungary", "Italy", "Malta", "Netherlands", "Norway", "Poland", "Portugal", "Romania", "Slovakia", "Spain", "Sweden", "United Kingdom (Northern Ireland)" ], "orpha": null }, { "unii": "QN49A5911G", "name": "BIFONAZOLE, (R)-", "moiety": "BIFONAZOLE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Bifonazole" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 21, "countries": [ "Austria", "Belgium", "Bulgaria", "Croatia", "Czech Republic", "Finland", "France", "Germany", "Greece", "Hungary", "Ireland", "Italy", "Luxembourg", "Netherlands", "Poland", "Portugal", "Romania", "Slovakia", "Slovenia", "Spain", "United Kingdom (Northern Ireland)" ], "orpha": null }, { "unii": "E1W4N241FO", "name": "SODIUM PHOSPHATE, DIBASIC, DODECAHYDRATE", "moiety": "SODIUM PHOSPHATE DODECAHYDRATE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Disodium Phosphate Dodecahydrate, Sodium Dihydrogen Phosphat", "Sodium Phosphate Dodecahydrate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 21, "countries": [ "Belgium", "Cyprus", "Czech Republic", "Denmark", "Finland", "France", "Germany", "Greece", "Hungary", "Ireland", "Italy", "Luxembourg", "Malta", "Netherlands", "Norway", "Poland", "Portugal", "Slovakia", "Spain", "Sweden", "United Kingdom (Northern Ireland)" ], "orpha": null }, { "unii": "934T64RMNJ", "name": "METAMIZOLE", "moiety": "METAMIZOLE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Metamizole", "Metamizole Magnesium", "Metamizole Magnesium Hexahydrate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 19, "countries": [ "Austria", "Belgium", "Bulgaria", "Croatia", "Czech Republic", "Germany", "Greece", "Hungary", "Italy", "Latvia", "Lithuania", "Luxembourg", "Netherlands", "Poland", "Portugal", "Romania", "Slovakia", "Slovenia", "Spain" ], "orpha": null }, { "unii": "AKU7QFL9ZT", "name": "TIANEPTINE, (+)-", "moiety": "TIANEPTINE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Tianeptine", "Tianeptine Sodium" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 18, "countries": [ "Austria", "Bulgaria", "Croatia", "Czech Republic", "Estonia", "France", "Germany", "Greece", "Hungary", "Latvia", "Lithuania", "Luxembourg", "Poland", "Portugal", "Romania", "Slovakia", "Slovenia", "Spain" ], "orpha": null }, { "unii": "J2Y905FQ57", "name": "THIOCTIC ACID, (-)-", "moiety": "THIOCTIC", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Thioctic Acid" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT FOR HUMAN PRESCRIPTION COMPOUNDING', 'UNAPPROVED HOMEOPATHIC']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 16, "countries": [ "Austria", "Bulgaria", "Croatia", "Czech Republic", "Estonia", "Germany", "Hungary", "Italy", "Latvia", "Lithuania", "Netherlands", "Poland", "Romania", "Slovakia", "Slovenia", "Spain" ], "orpha": null }, { "unii": "7DKH3NEJ7F", "name": "FLUOROCHOLINE CHLORIDE F-18", "moiety": "FLUOROCHOLINE 18F", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Fluorocholine (18f)", "Fluorocholine (18f) Chloride" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 15, "countries": [ "Austria", "Belgium", "Czech Republic", "France", "Germany", "Greece", "Hungary", "Italy", "Lithuania", "Netherlands", "Poland", "Portugal", "Slovakia", "Slovenia", "Spain" ], "orpha": null }, { "unii": "6330179ARU", "name": "OTILONIUM", "moiety": "OTILONIUM", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Otilonium Bromide" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 15, "countries": [ "Belgium", "Bulgaria", "Croatia", "Cyprus", "Czech Republic", "Greece", "Hungary", "Italy", "Latvia", "Lithuania", "Luxembourg", "Malta", "Portugal", "Romania", "Spain" ], "orpha": null }, { "unii": "JRD7912W79", "name": "ACENOCOUMAROL, (R)-", "moiety": "ACENOCOUMAROL", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Acenocoumarol" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 14, "countries": [ "Austria", "Belgium", "Bulgaria", "France", "Greece", "Hungary", "Italy", "Luxembourg", "Netherlands", "Poland", "Portugal", "Romania", "Spain", "United Kingdom (Northern Ireland)" ], "orpha": null }, { "unii": "RJ253LM2KD", "name": "NEFOPAM, (S)-", "moiety": "NEFOPAM", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Nefopam", "Nefopam Hydrochloride" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 13, "countries": [ "Croatia", "Cyprus", "Estonia", "France", "Ireland", "Latvia", "Lithuania", "Malta", "Netherlands", "Poland", "Romania", "Spain", "United Kingdom (Northern Ireland)" ], "orpha": null }, { "unii": "A585MN1H2L", "name": "MONTMORILLONITE", "moiety": "DIOSMECTITE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Diosmectite" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 12, "countries": [ "Bulgaria", "Czech Republic", "Estonia", "France", "Greece", "Hungary", "Italy", "Latvia", "Lithuania", "Poland", "Romania", "Slovakia" ], "orpha": null }, { "unii": "7V4KUP966U", "name": "SODIUM MOLYBDATE MO-99", "moiety": "SODIUM MOLYBDATE 99MO", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Sodium Molybdate (99mo)" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['DRUG FOR FURTHER PROCESSING']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 12, "countries": [ "Austria", "Belgium", "Bulgaria", "Denmark", "Germany", "Greece", "Hungary", "Italy", "Netherlands", "Poland", "Portugal", "Romania" ], "orpha": null }, { "unii": "20VJ8H4MNI", "name": "NITRENDIPINE, (R)-", "moiety": "NITRENDIPINE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Nitrendipine" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 11, "countries": [ "Czech Republic", "Estonia", "France", "Germany", "Greece", "Italy", "Latvia", "Lithuania", "Poland", "Portugal", "Slovakia" ], "orpha": null }, { "unii": "88IQ3US5N5", "name": "TOLPERISONE, (S)-", "moiety": "TOLPERISONE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Tolperisone Hydrochloride" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 11, "countries": [ "Bulgaria", "Cyprus", "Czech Republic", "Germany", "Hungary", "Latvia", "Lithuania", "Netherlands", "Poland", "Romania", "Slovakia" ], "orpha": null }, { "unii": "GI96W46M5A", "name": "MAGNESIUM OROTATE", "moiety": "MAGNESIUM OROTATE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Magnesium Orotate Dihydrate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['UNAPPROVED HOMEOPATHIC']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 10, "countries": [ "Austria", "Bulgaria", "Czech Republic", "Estonia", "Germany", "Hungary", "Latvia", "Lithuania", "Romania", "Slovakia" ], "orpha": null }, { "unii": "2381769MQC", "name": "ORNITHINE, DL-", "moiety": "ORNITHINE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Ornithine Aspartate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "STEREO_NAME: name carries an enantiomer/racemate marker; check the counterpart's US status by hand", "US_NON_APPLICATION_MARKETING: sold in the US under ['DRUG FOR FURTHER PROCESSING', 'UNAPPROVED HOMEOPATHIC']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 10, "countries": [ "Austria", "Bulgaria", "Croatia", "Germany", "Hungary", "Latvia", "Lithuania", "Luxembourg", "Poland", "Romania" ], "orpha": null }, { "unii": "DAA13NKG2Q", "name": "PAPAVERINE", "moiety": "PAPAVERINE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Papaverine Hydrochloride" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT', 'BULK INGREDIENT FOR HUMAN PRESCRIPTION COMPOUNDING', 'DRUG FOR FURTHER PROCESSING', 'UNAPPROVED DRUG OTHER']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 10, "countries": [ "Belgium", "Bulgaria", "Denmark", "France", "Germany", "Italy", "Latvia", "Poland", "Portugal", "Romania" ], "orpha": null }, { "unii": "U2368VVE7O", "name": "PINAVERIUM", "moiety": "PINAVERIUM", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Pinaverium Bromide" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 9, "countries": [ "Croatia", "Cyprus", "France", "Greece", "Hungary", "Italy", "Portugal", "Slovakia", "Spain" ], "orpha": null }, { "unii": "A17BS39YWR", "name": "AGOMELATINE CITRIC ACID", "moiety": "AGOMELATINE CITRIC", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Agomelatine Citric Acid" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 8, "countries": [ "Czech Republic", "Estonia", "France", "Germany", "Latvia", "Lithuania", "Portugal", "United Kingdom (Northern Ireland)" ], "orpha": null }, { "unii": "4LX0U5342U", "name": "ETHYLENEDIAMINE-N,N'-DIACETIC ACID", "moiety": "ETHYLENEDIAMINE N N DIACETIC", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Ethylenediamine-N,n'-Diacetic Acid" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 8, "countries": [ "Austria", "France", "Germany", "Greece", "Italy", "Portugal", "Spain", "United Kingdom (Northern Ireland)" ], "orpha": null }, { "unii": "RV6J6604TK", "name": "EUCALYPTOL", "moiety": "CINEOLE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Cineole" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT', 'OTC MONOGRAPH DRUG', 'UNAPPROVED HOMEOPATHIC']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 8, "countries": [ "Austria", "Czech Republic", "Germany", "Hungary", "Poland", "Romania", "Slovakia", "Slovenia" ], "orpha": null }, { "unii": "A4C6WE7BZN", "name": "NOSCAPINE, (+/-)-", "moiety": "NOSCAPINE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Noscapine", "Noscapine Hydrochloride", "Noscapine Hydrochloride Hydrate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 8, "countries": [ "Belgium", "Denmark", "Germany", "Luxembourg", "Netherlands", "Norway", "Spain", "Sweden" ], "orpha": null }, { "unii": "8P8Y8ZXJ8Y", "name": "SODIUM IOXITHALAMATE", "moiety": "IOXITALAMATE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Ioxitalamate Meglumine", "Ioxitalamate Sodium" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 8, "countries": [ "Belgium", "Czech Republic", "France", "Germany", "Hungary", "Luxembourg", "Netherlands", "Portugal" ], "orpha": null }, { "unii": "9RKX7S7WFY", "name": "TRIMEBUTINE, (+)-", "moiety": "TRIMEBUTINE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Trimebutine", "Trimebutine Maleate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 8, "countries": [ "Bulgaria", "France", "Greece", "Italy", "Poland", "Portugal", "Romania", "Spain" ], "orpha": null }, { "unii": "M7P27195AG", "name": "DOCUSATE", "moiety": "DOCUSATE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Docusate Sodium", "Docusate Sodium Bp" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT', 'BULK INGREDIENT FOR HUMAN PRESCRIPTION COMPOUNDING', 'DRUG FOR FURTHER PROCESSING', 'OTC MONOGRAPH DRUG', 'OTC MONOGRAPH NOT FINAL', 'UNAPPROVED DRUG OTHER']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 7, "countries": [ "Belgium", "France", "Ireland", "Luxembourg", "Netherlands", "Poland", "United Kingdom (Northern Ireland)" ], "orpha": null }, { "unii": "X6T6YN87SU", "name": "POTASSIUM SODIUM HYDROGEN CITRATE", "moiety": "", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "direct", "examples": [ "Potassium Sodium Hydrogen Citrate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 7, "countries": [ "Austria", "Belgium", "Bulgaria", "Germany", "Italy", "Luxembourg", "Portugal" ], "orpha": null }, { "unii": "602HN5L69H", "name": "PROPYLENE GLYCOL, (R)-", "moiety": "PROPYLENE GLYCOL", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Propylene Glycol" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT', 'OTC MONOGRAPH DRUG', 'OTC MONOGRAPH FINAL', 'UNAPPROVED HOMEOPATHIC']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 7, "countries": [ "Finland", "Germany", "Iceland", "Norway", "Poland", "Sweden", "United Kingdom (Northern Ireland)" ], "orpha": null }, { "unii": "73Y7P0K73Y", "name": "ALPHA LIPOIC ACID", "moiety": "ALPHA LIPOIC", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Alpha-Lipoic Acid Meglumine", "Alpha-Lipoic Acid, Meglumine Salt" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT', 'BULK INGREDIENT FOR HUMAN PRESCRIPTION COMPOUNDING', 'DRUG FOR FURTHER PROCESSING', 'UNAPPROVED DRUG OTHER', 'UNAPPROVED HOMEOPATHIC']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 6, "countries": [ "Austria", "Bulgaria", "Germany", "Latvia", "Slovakia", "Spain" ], "orpha": null }, { "unii": "R57ZHV85D4", "name": "BORIC ACID", "moiety": "BORIC", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Boric Acid", "Boric Acid, Sodium Borate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT', 'OTC MONOGRAPH DRUG', 'UNAPPROVED HOMEOPATHIC']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 6, "countries": [ "France", "Hungary", "Italy", "Latvia", "Poland", "Slovakia" ], "orpha": null }, { "unii": "V5PC588N7G", "name": "MAGNESIUM PIDOLATE", "moiety": "MAGNESIUM PIDOLATE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Magnesium Pidolate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 6, "countries": [ "France", "Greece", "Italy", "Luxembourg", "Portugal", "Spain" ], "orpha": null }, { "unii": "I5M8YK41GK", "name": "MYRISTALKONIUM ION", "moiety": "MYRISTALKONIUM", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Myristalkonium, Chloride" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 6, "countries": [ "Bulgaria", "France", "Hungary", "Latvia", "Lithuania", "Slovakia" ], "orpha": null }, { "unii": "DHD7FFG6YS", "name": "PHLOROGLUCINOL", "moiety": "PHLOROGLUCINOL", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Phloroglucinol", "Phloroglucinol Dihydrate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 6, "countries": [ "Bulgaria", "France", "Greece", "Italy", "Malta", "Poland" ], "orpha": null }, { "unii": "CIX3U01VAU", "name": ".ALPHA.-GLUCOSE-1-PHOSPHATE", "moiety": "GLUCOSE 1", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Glucose 1-Phosphate Disodium Tetrahydrate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 5, "countries": [ "Austria", "Czech Republic", "France", "Hungary", "Slovakia" ], "orpha": null }, { "unii": "19MLB4EQ88", "name": "ALUMINIUM ACETOTARTRATE", "moiety": "ALUMINIUM ACETOTARTRATE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Aluminium Acetotartrate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 5, "countries": [ "Germany", "Hungary", "Poland", "Slovakia", "Sweden" ], "orpha": null }, { "unii": "3JXF0Z0XOI", "name": "BICISATE", "moiety": "BICISATE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Bicisate Dihydrochloride" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT', 'DRUG FOR FURTHER PROCESSING']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 5, "countries": [ "Belgium", "Denmark", "France", "Luxembourg", "Spain" ], "orpha": null }, { "unii": "5TJD82A1ET", "name": "CAMPHOR (SYNTHETIC)", "moiety": "CAMPHOR RACEMIC", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Camphor, Racemic" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT', 'DRUG FOR FURTHER PROCESSING', 'OTC MONOGRAPH DRUG', 'OTC MONOGRAPH FINAL', 'OTC MONOGRAPH NOT FINAL', 'UNAPPROVED DRUG OTHER', 'UNAPPROVED HOMEOPATHIC']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 5, "countries": [ "Bulgaria", "France", "Germany", "Latvia", "Lithuania" ], "orpha": null }, { "unii": "2M105305SU", "name": "CLOPERASTINE FENDIZOATE", "moiety": "CLOPERASTINE FENDIZOATE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Cloperastine Fendizoate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 5, "countries": [ "Belgium", "Italy", "Luxembourg", "Portugal", "Spain" ], "orpha": null }, { "unii": "T940307O7B", "name": "DISTIGMINE", "moiety": "DISTIGMINE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Distigmine Bromide" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 5, "countries": [ "Czech Republic", "Germany", "Netherlands", "Poland", "Slovakia" ], "orpha": null }, { "unii": "FS5K818KA7", "name": "DROPROPIZINE, (R)-", "moiety": "DROPROPIZINE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Dropropizine" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 5, "countries": [ "Czech Republic", "Germany", "Italy", "Portugal", "Slovakia" ], "orpha": null }, { "unii": "700YTQ6F2B", "name": "FLUOROCHOLINE F-18", "moiety": "FLUOROMETHYL 18F DIMETHYL 2 HYDROXYETHYL", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Fluoromethyl-(18f)-Dimethyl-2-Hydroxyethyl-Ammonium" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 5, "countries": [ "Belgium", "France", "Italy", "Luxembourg", "Malta" ], "orpha": null }, { "unii": "M40WCZ5X76", "name": "MANIDIPINE, (S)-", "moiety": "MANIDIPINE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Manidipine", "Manidipine Dihydrochloride" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 5, "countries": [ "France", "Greece", "Italy", "Portugal", "Spain" ], "orpha": null }, { "unii": "020IUV4N33", "name": "PHOSPHOCREATINE", "moiety": "PHOSPHOCREATINE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Phosphocreatine Sodium" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 5, "countries": [ "Czech Republic", "Italy", "Poland", "Romania", "Slovakia" ], "orpha": null }, { "unii": "K72K37878A", "name": "RHENIUM HEPTASULFIDE", "moiety": "RHENIUM SULFIDE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Rhenium Sulfide" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 5, "countries": [ "France", "Malta", "Portugal", "Spain", "Sweden" ], "orpha": null }, { "unii": "XK88N6ZJHP", "name": "TAUROSELCHOLIC ACID SE-75", "moiety": "TAUROSELCHOLIC ACID SE 75", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Tauroselcholic Acid Se-75" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 5, "countries": [ "Cyprus", "Czech Republic", "Germany", "Greece", "Spain" ], "orpha": null }, { "unii": "2Y49VWD90Q", "name": "YOHIMBINE", "moiety": "YOHIMBINE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Yohimbine Hydrochloride" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['UNAPPROVED HOMEOPATHIC']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 5, "countries": [ "Austria", "Belgium", "France", "Germany", "Luxembourg" ], "orpha": null }, { "unii": "75UI7QUZ5J", "name": "AMINOBENZOATE SODIUM", "moiety": "AMINOBENZOATE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Aminobenzoate Potassium" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT', 'UNAPPROVED HOMEOPATHIC']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 4, "countries": [ "Austria", "Germany", "Hungary", "United Kingdom (Northern Ireland)" ], "orpha": null }, { "unii": "E96303N5PG", "name": "BAMBUTEROL, (S)-", "moiety": "BAMBUTEROL", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Bambuterol Hydrochloride" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 4, "countries": [ "Austria", "France", "Germany", "United Kingdom (Northern Ireland)" ], "orpha": null }, { "unii": "29VT07BGDA", "name": "CITRULLINE", "moiety": "CITRULLINE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Citrulline Malate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT', 'UNAPPROVED HOMEOPATHIC']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 4, "countries": [ "France", "Malta", "Portugal", "Spain" ], "orpha": null }, { "unii": "5V7PT38BJU", "name": "DIETHYLAMINE SALICYLATE", "moiety": "", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "direct", "examples": [ "Diethylamine Salicylate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 4, "countries": [ "Austria", "Hungary", "Portugal", "United Kingdom (Northern Ireland)" ], "orpha": null }, { "unii": "RWO67D87EU", "name": "ETHYLMORPHINE", "moiety": "ETHYLMORPHINE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Ethylmorphine Hydrochloride", "Ethylmorphine Hydrochloride Dihydrate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 4, "countries": [ "Belgium", "Finland", "Luxembourg", "Sweden" ], "orpha": null }, { "unii": "7OV03QG267", "name": "NICKEL", "moiety": "NICKEL", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Nickel Sulfate Hexahydrate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['UNAPPROVED HOMEOPATHIC']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 4, "countries": [ "Germany", "Italy", "Netherlands", "Spain" ], "orpha": null }, { "unii": "SU420W1S6N", "name": "PENTAERYTHRITOL", "moiety": "PENTAERITHRITYL", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Pentaerithrityl Tetranitrate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 4, "countries": [ "Bulgaria", "Czech Republic", "Germany", "Poland" ], "orpha": null }, { "unii": "00OT1QX5U4", "name": "POTASSIUM PERMANGANATE", "moiety": "POTASSIUM PERMANGANATE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Potassium Permanganate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['UNAPPROVED HOMEOPATHIC']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 4, "countries": [ "Germany", "Latvia", "Lithuania", "Poland" ], "orpha": null }, { "unii": "3M4G523W1G", "name": "SILVER", "moiety": "SILVER", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Silver Nitrate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT', 'DRUG FOR FURTHER PROCESSING', 'OTC MONOGRAPH DRUG', 'UNAPPROVED DRUG OTHER', 'UNAPPROVED HOMEOPATHIC']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 4, "countries": [ "France", "Malta", "Spain", "United Kingdom (Northern Ireland)" ], "orpha": null }, { "unii": "1K8M7UR6O1", "name": "YTTRIUM Y-90", "moiety": "YTTRIUM 90", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Yttrium-90" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 4, "countries": [ "Iceland", "Liechtenstein", "Norway", "Poland" ], "orpha": null }, { "unii": "5RLR54Z22K", "name": "2-MERCAPTOBENZOTHIAZOLE", "moiety": "MERCAPTOBENZOTHIAZOLE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Mercaptobenzothiazole" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 3, "countries": [ "Germany", "Italy", "Netherlands" ], "orpha": null }, { "unii": "2X1K91UT6N", "name": "ALOVUDINE F-18", "moiety": "FLUOROTHYMIDINE F 18", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Fluorothymidine F-18" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 3, "countries": [ "Czech Republic", "Greece", "Poland" ], "orpha": null }, { "unii": "QUY32964DJ", "name": "ANETHOLTRITHION", "moiety": "ANETHOLE TRITHIONE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Anethole Trithione" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 3, "countries": [ "Belgium", "France", "Luxembourg" ], "orpha": null }, { "unii": "U015TT5I8H", "name": "BISMUTH", "moiety": "BISMUTH", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Bismuth", "Bismuth Gluconate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT', 'BULK INGREDIENT FOR HUMAN PRESCRIPTION COMPOUNDING', 'OTC MONOGRAPH DRUG', 'UNAPPROVED HOMEOPATHIC']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 3, "countries": [ "France", "Greece", "Hungary" ], "orpha": null }, { "unii": "G0L543D370", "name": "BRILLIANT GREEN", "moiety": "BRILLIANT GREEN", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Brilliant Green" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 3, "countries": [ "Estonia", "Latvia", "Lithuania" ], "orpha": null }, { "unii": "32SXB5VH2O", "name": "CLENBUTEROL, (S)-", "moiety": "CLENBUTEROL", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Clenbuterol Hydrochloride" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT', 'BULK INGREDIENT FOR HUMAN PRESCRIPTION COMPOUNDING']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 3, "countries": [ "Bulgaria", "Germany", "Italy" ], "orpha": null }, { "unii": "5G1JDI469J", "name": "DIHYDROXYALUMINUM GLUCONATE", "moiety": "DIHYDROXYALUMINUM", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Dihydroxyaluminum Sodium Carbonate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 3, "countries": [ "Denmark", "Poland", "Portugal" ], "orpha": null }, { "unii": "TMH99LTT6R", "name": "ERBIUM CITRATE ER-169", "moiety": "ERBIUM ER169", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Erbium (Er169) Citrate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 3, "countries": [ "Czech Republic", "France", "Germany" ], "orpha": null }, { "unii": "4I5K8199OQ", "name": "HIDROSMIN", "moiety": "HIDROSMIN", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Hidrosmin" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 3, "countries": [ "Bulgaria", "Portugal", "Spain" ], "orpha": null }, { "unii": "8P68XPY4HG", "name": "HYDROQUINIDINE", "moiety": "HYDROQUINIDINE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Hydroquinidine Hydrochloride" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 3, "countries": [ "France", "Italy", "Spain" ], "orpha": null }, { "unii": "424J5HGB8E", "name": "KETAZOLAM, (+)-", "moiety": "KETAZOLAM", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Ketazolam" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 3, "countries": [ "Italy", "Portugal", "Spain" ], "orpha": null }, { "unii": "Y4F5ODM66L", "name": "MAGNESIUM ASPARTATE DIHYDRATE", "moiety": "", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "direct", "examples": [ "Magnesium Aspartate Dihydrate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 3, "countries": [ "Germany", "Greece", "United Kingdom (Northern Ireland)" ], "orpha": null }, { "unii": "6JD4830MKN", "name": "MEPTAZINOL, (R)-", "moiety": "MEPTAZINOL", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Meptazinol", "Meptazinol Hydrochloride" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 3, "countries": [ "Germany", "Ireland", "United Kingdom (Northern Ireland)" ], "orpha": null }, { "unii": "0M7PSL4100", "name": "N-ISOPROPYL-N'-PHENYL-1,4-PHENYLENEDIAMINE", "moiety": "N ISOPROPYL N PHENYL 1 4 PHENYLENEDIAMINE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "N-Isopropyl-N'-Phenyl-1,4-Phenylenediamine" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 3, "countries": [ "Germany", "Italy", "Netherlands" ], "orpha": null }, { "unii": "HFE9M7UF0L", "name": "NORCHOLESTENOL IODOMETHYL I-131", "moiety": "IODINE NORCHOLESTEROL 131I", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Iodine Norcholesterol (131i)" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 3, "countries": [ "France", "Italy", "Spain" ], "orpha": null }, { "unii": "U770QIT64J", "name": "P-PHENYLENEDIAMINE", "moiety": "P PHENYLENEDIAMINE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "P-Phenylenediamine" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 3, "countries": [ "Germany", "Netherlands", "United Kingdom (Northern Ireland)" ], "orpha": null }, { "unii": "75934UD4GJ", "name": "PRAJMALIUM", "moiety": "PRAJMALIUM", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Prajmalium Bitartrate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 3, "countries": [ "Czech Republic", "Hungary", "Slovakia" ], "orpha": null }, { "unii": "91MBZ8H3QO", "name": "SODIUM BORATE", "moiety": "BORAX", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Borax", "Sodium Borate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['DRUG FOR FURTHER PROCESSING', 'UNAPPROVED HOMEOPATHIC']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 3, "countries": [ "Czech Republic", "Hungary", "Poland" ], "orpha": null }, { "unii": "24JO8Z0RJU", "name": "TOLUENE-2,5-DIAMINE", "moiety": "2 5 DIAMINOTOLUENE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "2,5-Diaminotoluene" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 3, "countries": [ "Germany", "Spain", "Sweden" ], "orpha": null }, { "unii": "K3D86KJ24N", "name": "UNDECYLENIC ACID", "moiety": "UNDECYLENIC", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Undecylenic Acid, Zinc Undecylenate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT', 'DRUG FOR FURTHER PROCESSING', 'OTC MONOGRAPH DRUG', 'OTC MONOGRAPH FINAL', 'UNAPPROVED DRUG OTHER', 'UNAPPROVED HOMEOPATHIC']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 3, "countries": [ "Ireland", "Poland", "United Kingdom (Northern Ireland)" ], "orpha": null }, { "unii": "11P2JDE17B", "name": ".BETA.-ALANINE", "moiety": "BETA ALANINE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Beta-Alanine" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 2, "countries": [ "France", "Luxembourg" ], "orpha": null }, { "unii": "2730CL9N7C", "name": "2',4',5',7'- TETRABROMOFLUORESCEIN", "moiety": "EOSINE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Eosine", "Eosine Disodium" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 2, "countries": [ "France", "Italy" ], "orpha": null }, { "unii": "FT898U5C9H", "name": "ALUMINUM PHOSPHATE GEL", "moiety": "ALUMINIUM PHOSPHATE GEL", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Aluminium Phosphate Gel" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 2, "countries": [ "France", "Poland" ], "orpha": null }, { "unii": "03JLX11PE9", "name": "AMINAPHTHONE", "moiety": "AMINAPHTONE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Aminaphtone" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 2, "countries": [ "Italy", "Portugal" ], "orpha": null }, { "unii": "CL7T957EGC", "name": "BENDAZAC LYSINE", "moiety": "BENDAZAC LYSINATE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Bendazac Lysinate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 2, "countries": [ "Italy", "Portugal" ], "orpha": null }, { "unii": "595726R32U", "name": "BISULEPIN", "moiety": "BISULEPIN", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Bisulepin Hydrochloride" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 2, "countries": [ "Czech Republic", "Slovakia" ], "orpha": null }, { "unii": "1D898P42YW", "name": "CALCIUM CHLORIDE HEXAHYDRATE", "moiety": "", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "direct", "examples": [ "Calcium Chloride Hexahydrate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 2, "countries": [ "France", "Spain" ], "orpha": null }, { "unii": "77YW1RTU8V", "name": "CALCIUM UNDECYLENATE", "moiety": "", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "direct", "examples": [ "Calcium Undecylenate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 2, "countries": [ "Ireland", "Malta" ], "orpha": null }, { "unii": "0T45P766JT", "name": "CARBAETHOPENDECINE BROMIDE", "moiety": "CARBAETHOPENDECINIUM", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Carbaethopendecinium Bromide" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 2, "countries": [ "Czech Republic", "Slovakia" ], "orpha": null }, { "unii": "06PW4M190R", "name": "CLONIXIN LYSINE", "moiety": "CLONIXIN LYSINATE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Clonixin Lysinate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 2, "countries": [ "Portugal", "Spain" ], "orpha": null }, { "unii": "3G0H8C9362", "name": "COBALT", "moiety": "COBALT", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Cobalt Dichloride Hexahydrate", "Cobalt Gluconate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['UNAPPROVED HOMEOPATHIC']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 2, "countries": [ "France", "Netherlands" ], "orpha": null }, { "unii": "6770B58N0V", "name": "DIHYDROABIETYL ALCOHOL", "moiety": "DIHYDROABIETYL ALCOHOL", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Dihydroabietyl Alcohol" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 2, "countries": [ "Germany", "Italy" ], "orpha": null }, { "unii": "3G98348KML", "name": "ETHYLENEDIAMINE THIOCTATE", "moiety": "ETHYLENEDIAMINE THIOCTATE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Ethylenediamine Thioctate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 2, "countries": [ "Croatia", "Germany" ], "orpha": null }, { "unii": "1HG84L3525", "name": "FORMALDEHYDE", "moiety": "FORMALDEHYDE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Formaldehyde" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['UNAPPROVED HOMEOPATHIC']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 2, "countries": [ "Germany", "Netherlands" ], "orpha": null }, { "unii": "K2R4N34Y9I", "name": "FURAZIDINE", "moiety": "FURAZIDINE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Furazidine" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 2, "countries": [ "Latvia", "Poland" ], "orpha": null }, { "unii": "75G4TW236W", "name": "MEGLUMINE ANTIMONIATE", "moiety": "MEGLUMINE ANTIMONATE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Meglumine Antimonate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 2, "countries": [ "France", "Spain" ], "orpha": null }, { "unii": "ED857BWP29", "name": "METHIONINE C-11", "moiety": "L METHIONINE 11C METHYL", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "L-Methionine ([11c]methyl)" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 2, "countries": [ "Czech Republic", "Hungary" ], "orpha": null }, { "unii": "M1V54MNO1P", "name": "METHYLPHENOBARBITAL, (R)-", "moiety": "METHYLPHENOBARBITAL", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Methylphenobarbital" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 2, "countries": [ "Croatia", "Slovenia" ], "orpha": null }, { "unii": "N6KNE1QA9O", "name": "OBIDOXIME", "moiety": "OBIDOXIME", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Obidoxime Chloride" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 2, "countries": [ "Germany", "Netherlands" ], "orpha": null }, { "unii": "Q824SGH3RN", "name": "OXOMEMAZINE, (+)-", "moiety": "OXOMEMAZINE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Oxomemazine" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 2, "countries": [ "Bulgaria", "France" ], "orpha": null }, { "unii": "8915147A2B", "name": "PERAZINE", "moiety": "PERAZINE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Perazine Dimaleate", "Perazine Dimalonate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 2, "countries": [ "Germany", "Poland" ], "orpha": null }, { "unii": "9U1VM840SP", "name": "PHYSOSTIGMINE", "moiety": "PHYSOSTIGMINE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Physostigmine Salicylate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT', 'UNAPPROVED DRUG FOR USE IN DRUG SHORTAGE', 'UNAPPROVED DRUG OTHER']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 2, "countries": [ "Austria", "Germany" ], "orpha": null }, { "unii": "88496G1ERL", "name": "PHYTATE SODIUM", "moiety": "SODIUM PHYTATE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Sodium Phytate Anhydrous" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 2, "countries": [ "France", "Hungary" ], "orpha": null }, { "unii": "T4423S18FM", "name": "POTASSIUM DICHROMATE", "moiety": "POTASSIUM DICHROMATE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Potassium Dichromate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT', 'DRUG FOR FURTHER PROCESSING', 'UNAPPROVED DRUG OTHER', 'UNAPPROVED HOMEOPATHIC']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 2, "countries": [ "Germany", "Netherlands" ], "orpha": null }, { "unii": "WZH3C48M4T", "name": "POTASSIUM HYDROXIDE", "moiety": "POTASSIUM HYDROXIDE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Potassium Hydroxide, Dipotassium Hydrogen Phosphate Trihydra" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['DRUG FOR FURTHER PROCESSING']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 2, "countries": [ "Finland", "Sweden" ], "orpha": null }, { "unii": "F887D1637W", "name": "SENNOSIDE B", "moiety": "SENNOSIDE B", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Sennoside B" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 2, "countries": [ "Austria", "United Kingdom (Northern Ireland)" ], "orpha": null }, { "unii": "AI0NX02O35", "name": "SOBREROL, TRANS-, (+/-)-", "moiety": "SOBREROL", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Sobrerol" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 2, "countries": [ "Italy", "Malta" ], "orpha": null }, { "unii": "DY38VHM5OD", "name": "SODIUM HYPOCHLORITE", "moiety": "SODIUM HYPOCHLORITE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Sodium Hypochlorite" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['UNAPPROVED DRUG OTHER', 'UNAPPROVED HOMEOPATHIC']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 2, "countries": [ "Germany", "Italy" ], "orpha": null, "eu_orphan_designation": { "use": "Treatment of partial deep dermal and full-thickness burns", "status": "Positive", "number": "EU/3/16/1709" }, "eu_orphan_designations": [ { "use": "Treatment of partial deep dermal and full-thickness burns", "status": "Positive", "number": "EU/3/16/1709" } ] }, { "unii": "368GB5141J", "name": "SODIUM LAURYL SULFATE", "moiety": "SODIUM LAURILSULFATE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Sodium Laurilsulfate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['UNAPPROVED DRUG OTHER', 'UNAPPROVED HOMEOPATHIC']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 2, "countries": [ "Germany", "Spain" ], "orpha": null }, { "unii": "D0Y70F2B9J", "name": "SODIUM LAURYL SULFOACETATE", "moiety": "SODIUM LAURYL SULFOACETATE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Sodium Lauryl Sulfoacetate, Sodium Citrate Dihydrate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 2, "countries": [ "Denmark", "Iceland" ], "orpha": null }, { "unii": "WIQ1H85SYP", "name": "SODIUM SALICYLATE", "moiety": "", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "direct", "examples": [ "Sodium Salicylate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT', 'BULK INGREDIENT FOR HUMAN PRESCRIPTION COMPOUNDING', 'DRUG FOR FURTHER PROCESSING', 'OTC MONOGRAPH DRUG', 'UNAPPROVED DRUG OTHER', 'UNAPPROVED HOMEOPATHIC']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 2, "countries": [ "Czech Republic", "Slovakia" ], "orpha": null }, { "unii": "8A3Q9VKO7U", "name": "SPAGLUMATE SODIUM", "moiety": "SPAGLUMATE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Spaglumate Sodium" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 2, "countries": [ "France", "Italy" ], "orpha": null }, { "unii": "UF17KA8AC5", "name": "TOSYLCHLORAMIDE", "moiety": "TOSYLCHLORAMIDE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Tosylchloramide Sodium" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 2, "countries": [ "Belgium", "Italy" ], "orpha": null }, { "unii": "91D9GV0Z28", "name": "TRIBASIC CALCIUM PHOSPHATE", "moiety": "CALCIUM PHOSPHATE TRIBASIC", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Calcium Phosphate, Tribasic" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT', 'DRUG FOR FURTHER PROCESSING', 'OTC MONOGRAPH DRUG', 'UNAPPROVED DRUG OTHER', 'UNAPPROVED HOMEOPATHIC']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 2, "countries": [ "Greece", "Spain" ], "orpha": null }, { "unii": "P7K44M64CW", "name": ".BETA.-ACETYLDIGOXIN", "moiety": "BETA ACETYLDIGOXIN", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Beta-Acetyldigoxin" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Germany" ], "orpha": null }, { "unii": "P01OUT964K", "name": ".BETA.-CITRONELLOL, (R)-", "moiety": "CITRONELLOL", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Citronellol" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['OTC MONOGRAPH FINAL', 'UNAPPROVED DRUG OTHER']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Germany" ], "orpha": null }, { "unii": "QYK0D6H79O", "name": ".BETA.-ESCIN", "moiety": "BETA AESCIN", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Beta-Aescin" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Poland" ], "orpha": null }, { "unii": "S347WMO6M4", "name": ".BETA.-SITOSTEROL", "moiety": "PHYTOSTEROL", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Phytosterol" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['OTC MONOGRAPH FINAL', 'UNAPPROVED DRUG OTHER']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Germany" ], "orpha": null }, { "unii": "CM46XGM78M", "name": ".GAMMA.-AMINO-.BETA.-HYDROXYBUTYRIC ACID, (+)-", "moiety": "GAMMA AMINO BETA HYDROXYBUTYRIC", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Gamma-Amino-Beta-Hydroxybutyric Acid" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Italy" ], "orpha": null }, { "unii": "3154M2Q6GD", "name": "1,3-DIBUTYL-2-THIOUREA", "moiety": "1 3 DIBUTYL 2 THIOUREA", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "1,3-Dibutyl-2-Thiourea" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Germany" ], "orpha": null }, { "unii": "E9MU425FIB", "name": "1,4-BUTANEDIOL DIGLYCIDYL ETHER", "moiety": "1 4 BIS 2 3 EPOXYPROPOXY BUTANE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "1,4-Bis(2,3-Epoxypropoxy)butane" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Germany" ], "orpha": null }, { "unii": "6R5Y84T8W9", "name": "2-(2-AMINOETHOXY)ETHANOL", "moiety": "2 2 AMINOETHOXY ETHANOL", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "2-(2-Aminoethoxy)ethanol" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Germany" ], "orpha": null }, { "unii": "6E1I4IV47V", "name": "2-HYDROXYETHYL METHACRYLATE", "moiety": "2 HYDROXYETHYL METHACRYLATE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "2-Hydroxyethyl Methacrylate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Germany" ], "orpha": null }, { "unii": "V3ZU6E76NB", "name": "3-HYDROXY-2-NAPHTHANILIDE", "moiety": "3 HYDROXY 2 NAPHTHANILIDE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "3-Hydroxy-2-Naphthanilide" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Germany" ], "orpha": null }, { "unii": "GG5LL7OBZC", "name": "4,4'-METHYLENEDIANILINE", "moiety": "4 4 METHYLENEDIANILINE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "4,4-Methylenedianiline" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Germany" ], "orpha": null }, { "unii": "X387L5559O", "name": "4-DIMETHYLAMINOPHENOL", "moiety": "4 DIMETHYLAMINOPHENOL", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "4-Dimethylaminophenol Hydrochloride" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Germany" ], "orpha": null }, { "unii": "9A069144KR", "name": "4-TERT-BUTYLCATECHOL", "moiety": "4 TERT BUTYLCATECHOL", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "4-Tert-Butylcatechol" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Germany" ], "orpha": null }, { "unii": "O81VMW36CV", "name": "4-TERT-BUTYLPHENOL", "moiety": "4 TERT BUTYLPHENOL", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "4-Tert-Butylphenol" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Germany" ], "orpha": null }, { "unii": "20E75SVO7A", "name": "7-ETHYLBICYCLOOXAZOLIDINE", "moiety": "7 ETHYLBICYCLOOXAZOLIDINE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "7-Ethylbicyclooxazolidine" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Germany" ], "orpha": null }, { "unii": "V3DHX33184", "name": "ABIETIC ACID", "moiety": "ABIETIC", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Abietic Acid" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Germany" ], "orpha": null }, { "unii": "C5BTS0548U", "name": "ADEMETIONINE BUTANEDISULFONATE", "moiety": "ADEMETIONINE 1 4 BUTANEDISULFONATE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Ademetionine 1,4-Butanedisulfonate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Bulgaria" ], "orpha": null }, { "unii": "8L70Q75FXE", "name": "ADENOSINE TRIPHOSPHATE", "moiety": "ADENOSINE 5", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Adenosine 5' Triphosphate, Disodium Salt" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['UNAPPROVED HOMEOPATHIC']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "France" ], "orpha": null }, { "unii": "XDK458E1J9", "name": "ALLANTOIN, (S)-", "moiety": "ALLANTOIN", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Allantoin" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT', 'OTC MONOGRAPH DRUG', 'OTC MONOGRAPH FINAL', 'UNAPPROVED DRUG OTHER']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Poland" ], "orpha": null, "eu_orphan_designation": { "use": "Treatment of epidermolysis bullosa", "status": "Withdrawn", "number": "EU/3/13/1232" }, "eu_orphan_designations": [ { "use": "Treatment of epidermolysis bullosa", "status": "Withdrawn", "number": "EU/3/13/1232" } ] }, { "unii": "0D34IG724V", "name": "ALUMINUM FORMATE", "moiety": "", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "direct", "examples": [ "Aluminium Formate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Germany" ], "orpha": null }, { "unii": "NZO8Q0FP2E", "name": "ALUMINUM HYDROXIDE OXIDE", "moiety": "BOEHMITE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Boehmite" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "France" ], "orpha": null }, { "unii": "F92V3S521O", "name": "ALUMINUM PHOSPHATE", "moiety": "", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "direct", "examples": [ "Aluminium Phosphate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT', 'DRUG FOR FURTHER PROCESSING', 'OTC MONOGRAPH DRUG', 'UNAPPROVED HOMEOPATHIC']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Poland" ], "orpha": null }, { "unii": "0HM1E174TN", "name": "AMBROXOL ACEFYLLINATE", "moiety": "AMBROXOL ACEFYLLINATE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Ambroxol Acefyllinate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Italy" ], "orpha": null }, { "unii": "7E2P22546V", "name": "AMIDEPHRINE", "moiety": "AMIDEFRINE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Amidefrine Mesilate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Austria" ], "orpha": null }, { "unii": "5138Q19F1X", "name": "AMMONIA", "moiety": "AMMONIA", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Ammonia" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT', 'OTC MONOGRAPH DRUG', 'OTC MONOGRAPH FINAL', 'UNAPPROVED DRUG OTHER', 'UNAPPROVED HOMEOPATHIC']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Lithuania" ], "orpha": null }, { "unii": "22QF6L357F", "name": "AMMONIUM PERSULFATE", "moiety": "AMMONIUM PERSULFATE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Ammonium Persulfate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Germany" ], "orpha": null }, { "unii": "SU46BAM238", "name": "AMMONIUM SULFATE", "moiety": "", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "direct", "examples": [ "Ammonium Sulfate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Poland" ], "orpha": null }, { "unii": "6P9E788VZZ", "name": "AMMONIUM THIOGLYCOLATE", "moiety": "AMMONIUM THIOGLYCOLATE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Ammonium Thioglycolate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Germany" ], "orpha": null }, { "unii": "QRW683O56T", "name": "AMYLOCAINE", "moiety": "AMYLOCAINE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Amylocaine Hydrochloride" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "France" ], "orpha": null }, { "unii": "N0BN0O8CSL", "name": "BENZODODECINIUM ION", "moiety": "BENZODODECINIUM", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Benzododecinium Bromide" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "France" ], "orpha": null }, { "unii": "D95EHT6Y2L", "name": "BENZOXONIUM", "moiety": "BENZOXONIUM", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Benzoxonium Chloride" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Italy" ], "orpha": null }, { "unii": "PZ35LUM333", "name": "BENZYLTHIOURACIL", "moiety": "BENZYLTHIOURACIL", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Benzylthiouracil" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "France" ], "orpha": null }, { "unii": "F069Y6XCZ6", "name": "BETAMETHASONE VALEROACETATE", "moiety": "BETAMETHASONE 17 VALERATE 21", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Betamethasone 17-Valerate 21-Acetate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Italy" ], "orpha": null }, { "unii": "Y02A7LBH82", "name": "BUTYL GLYCIDYL ETHER, (+)-", "moiety": "BUTYL GLYCIDYL ETHER", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Butyl Glycidyl Ether" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Germany" ], "orpha": null }, { "unii": "1P9D0Z171K", "name": "BUTYLATED HYDROXYTOLUENE", "moiety": "BUTYLHYDROXYTOLUENE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Butylhydroxytoluene" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['OTC MONOGRAPH FINAL', 'UNAPPROVED HOMEOPATHIC']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Germany" ], "orpha": null }, { "unii": "2Z3E1OF81V", "name": "BUTYLSCOPOLAMINE", "moiety": "BUTYLSCOPOLAMINE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Butylscopolamine" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT', 'BULK INGREDIENT FOR HUMAN PRESCRIPTION COMPOUNDING']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Italy" ], "orpha": null }, { "unii": "FHG725432Z", "name": "CETALKONIUM", "moiety": "CETALKONIUM", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Cetalkonium Chloride" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Germany" ], "orpha": null }, { "unii": "1V984I1L1M", "name": "CETHEXONIUM", "moiety": "CETHEXONIUM", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Cethexonium Bromide" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "France" ], "orpha": null }, { "unii": "KW8K411A1P", "name": "CHLORAMINE", "moiety": "CHLORAMINE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Chloramine" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Italy" ], "orpha": null }, { "unii": "4R7X1O2820", "name": "CHLORINE", "moiety": "CHLORINE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Chlorine" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['UNAPPROVED DRUG OTHER', 'UNAPPROVED HOMEOPATHIC']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "France" ], "orpha": null }, { "unii": "2R97846T1L", "name": "CHLOROACETAMIDE", "moiety": "CHLOROACETAMIDE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Chloroacetamide" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Germany" ], "orpha": null }, { "unii": "SR60A3XG0F", "name": "CINNAMALDEHYDE", "moiety": "CINNAMALDEHYDE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Cinnamaldehyde" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Germany" ], "orpha": null }, { "unii": "8406EY2OQA", "name": "COBALAMIN", "moiety": "COBALAMIN", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Cobalamin" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['UNAPPROVED DRUG OTHER', 'UNAPPROVED HOMEOPATHIC']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Germany" ], "orpha": null }, { "unii": "RTJ1W9DF34", "name": "COBALTOUS IODIDE", "moiety": "COBALT II", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Cobalt(Ii) Chloride" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['UNAPPROVED HOMEOPATHIC']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Germany" ], "orpha": null }, { "unii": "A4VZ22K1WT", "name": "COUMARIN", "moiety": "COUMARIN", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Coumarin" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['OTC MONOGRAPH FINAL', 'UNAPPROVED HOMEOPATHIC']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Germany" ], "orpha": null }, { "unii": "Z7F472XQPA", "name": "DIDECYLDIMONIUM", "moiety": "DIDECYLDIMETHYLAMMONIUM", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Didecyldimethylammonium Chloride" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['OTC MONOGRAPH DRUG']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Germany" ], "orpha": null }, { "unii": "3RYP614N6L", "name": "DIHYDRALAZINE SULFATE, HYDRATED", "moiety": "DIHYDRALAZINE SULFATE HYDRATED", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Dihydralazine Sulphate, Hydrated" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Germany" ], "orpha": null }, { "unii": "05D48LUM4Z", "name": "DIHYDROERGOCRISTINE", "moiety": "DIHYDROERGOCRISTINE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Dihydroergocristine Mesilate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Spain" ], "orpha": null }, { "unii": "S8XC5939VU", "name": "DIMETHYLTOLYLAMINE", "moiety": "DIMETHYL 4 TOLUIDINE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Dimethyl-4-Toluidine" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Germany" ], "orpha": null }, { "unii": "CR113982E5", "name": "DIPENTAMETHYLENETHIURAM DISULFIDE", "moiety": "DIPENTAMETHYLENETHIURAM DISULFIDE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Dipentamethylenethiuram Disulfide" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Germany" ], "orpha": null }, { "unii": "6MRZ85RNHQ", "name": "DIPHENYLGUANIDINE", "moiety": "DIPHENYLGUANIDINE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Diphenylguanidine" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Germany" ], "orpha": null }, { "unii": "0890872E2E", "name": "DISPERSE YELLOW 3", "moiety": "DISPERSE YELLOW 3", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Disperse Yellow 3" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Germany" ], "orpha": null }, { "unii": "4AZL56CU0F", "name": "DOMIPHEN", "moiety": "DOMIPHEN", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Domiphen Bromide, Sodium Chloride, Sodium Fluoride" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "France" ], "orpha": null }, { "unii": "22IHM5PF20", "name": "EPERISONE, (S)-", "moiety": "EPERISONE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Eperisone Hydrochloride" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Italy" ], "orpha": null }, { "unii": "FE5SPV1Z6G", "name": "ETHACIZINE", "moiety": "ETHACIZINE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Ethacizine" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Latvia" ], "orpha": null }, { "unii": "V14XF1PJUF", "name": "ETHANOLAMINE THENOATE", "moiety": "OLAMINE TENOATE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Ethanolamine Tenoate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "France" ], "orpha": null }, { "unii": "0F5N573A2Y", "name": "ETHER", "moiety": "DIETHYL ETHER", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Diethyl Ether" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['UNAPPROVED HOMEOPATHIC']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "France" ], "orpha": null }, { "unii": "46U771ERWK", "name": "ETHYL CHLORIDE", "moiety": "", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "direct", "examples": [ "Ethyl Chloride" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Poland" ], "orpha": null }, { "unii": "M0942C52XH", "name": "ETHYLENEDICYSTEINE", "moiety": "ETHYLENEDICYSTEINE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Ethylenedicysteine" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Hungary" ], "orpha": null }, { "unii": "PN2ZH5LOQY", "name": "FD&C RED NO. 3", "moiety": "ERYTHROSINE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Erythrosine" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "United Kingdom (Northern Ireland)" ], "orpha": null }, { "unii": "DI3477D0PI", "name": "FERROUS ASCORBATE", "moiety": "FERROUS ASCORBATE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Ferrous Ascorbate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "France" ], "orpha": null }, { "unii": "ZO2JV75L55", "name": "FLUDEOXYGLUCOSE", "moiety": "FLUDEOXYGLUCOSE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Fludeoxyglucose" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "United Kingdom (Northern Ireland)" ], "orpha": null }, { "unii": "284SYP0193", "name": "FLUORINE", "moiety": "FLUORINE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Fluorine" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['UNAPPROVED HOMEOPATHIC']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Sweden" ], "orpha": null }, { "unii": "L837108USY", "name": "GERANIOL", "moiety": "GERANIOL", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Geraniol" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['OTC MONOGRAPH FINAL', 'UNAPPROVED DRUG OTHER']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Germany" ], "orpha": null }, { "unii": "GAN16C9B8O", "name": "GLUTATHIONE", "moiety": "GLUTATHIONE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Glutathione", "Glutathione Sodium" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT', 'BULK INGREDIENT FOR HUMAN PRESCRIPTION COMPOUNDING', 'OTC MONOGRAPH DRUG', 'UNAPPROVED HOMEOPATHIC']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Italy" ], "orpha": null, "eu_orphan_designation": { "use": "Treatment of cystic fibrosis", "status": "Withdrawn", "number": "EU/3/06/361" }, "eu_orphan_designations": [ { "use": "Treatment of cystic fibrosis", "status": "Withdrawn", "number": "EU/3/06/361" } ] }, { "unii": "7BK5G69305", "name": "GLYCOL DIMETHACRYLATE", "moiety": "ETHYLENE GLYCOL DIMETHACRYLATE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Ethylene Glycol Dimethacrylate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Germany" ], "orpha": null }, { "unii": "R6GJS0F9NZ", "name": "GLYOXAL TRIMER DIHYDRATE", "moiety": "GLYOXAL TRIMER", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Glyoxal Trimer Dihydrate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['UNAPPROVED HOMEOPATHIC']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Germany" ], "orpha": null }, { "unii": "79Y1949PYO", "name": "GOLD", "moiety": "GOLD", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Gold" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['UNAPPROVED HOMEOPATHIC']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "France" ], "orpha": null, "eu_orphan_designation": { "use": "Treatment of amyotrophic lateral sclerosis", "status": "Positive", "number": "EU/3/22/2686" }, "eu_orphan_designations": [ { "use": "Treatment of amyotrophic lateral sclerosis", "status": "Positive", "number": "EU/3/22/2686" } ] }, { "unii": "31J3Q51T6L", "name": "HYDROQUININE", "moiety": "HYDROQUININE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Hydroquinine Hydrobromide" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Netherlands" ], "orpha": null }, { "unii": "8SQ0VA4YUR", "name": "HYDROXYCITRONELLAL", "moiety": "HYDROXYCITRONELLAL", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Hydroxycitronellal" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['OTC MONOGRAPH FINAL']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Germany" ], "orpha": null }, { "unii": "QUE43B9Z2Q", "name": "HYDROXYISOHEXYL 3-CYCLOHEXENE CARBOXALDEHYDE", "moiety": "4 4 HYDROXY 4 METHYLPENTYL CYCLOHEX 3 ENE 1 CARBALDEHYDE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "4-(4-Hydroxy-4-Methylpentyl)cyclohex-3-Ene-1-Carbaldehyde" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Germany" ], "orpha": null }, { "unii": "02X2TO1S2D", "name": "IBUPROFEN ISOBUTANOLAMMONIUM", "moiety": "IBUPROFEN ISOBUTANOLAMMONIUM", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Ibuprofen Isobutanolammonium" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Italy" ], "orpha": null }, { "unii": "M629807ATL", "name": "IMIDUREA", "moiety": "IMIDUREA", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Imidurea" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['OTC MONOGRAPH FINAL']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Germany" ], "orpha": null }, { "unii": "KXI2J76489", "name": "IODOFORM", "moiety": "IODOFORM", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Iodoform" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Germany" ], "orpha": null }, { "unii": "454I75YXY0", "name": "ISOPROPYLIDENEDIPHENYL BISOXYHYDROXYPROPYL METHACRYLATE", "moiety": "BISPHENOL A GLYCIDYL METHACRYLATE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Bisphenol A-Glycidyl Methacrylate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Germany" ], "orpha": null }, { "unii": "Z7Y1U20IBU", "name": "ISOSPAGLUMATE SODIUM", "moiety": "SODIUM ISOSPAGLUMATE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Sodium Isospaglumate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Romania" ], "orpha": null }, { "unii": "09837K287S", "name": "LEVOCLOPERASTINE FENDIZOATE", "moiety": "LEVOCLOPERASTINE FENDIZOATE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Levocloperastine Fendizoate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "STEREO_NAME: name carries an enantiomer/racemate marker; check the counterpart's US status by hand" ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Italy" ], "orpha": null }, { "unii": "BRH40Y9V1Q", "name": "LUTETIUM LU-177", "moiety": "LUTETIUM LU 177", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Lutetium Lu-177" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['DRUG FOR FURTHER PROCESSING']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Poland" ], "orpha": null }, { "unii": "L3WTS6QT82", "name": "M-AMINOPHENOL", "moiety": "3 AMINOPHENOL", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "3-Aminophenol" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Germany" ], "orpha": null }, { "unii": "5Q5ZYJ43UH", "name": "MAGNESIUM GLUTAMATE HYDROBROMIDE", "moiety": "", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "direct", "examples": [ "Magnesium Glutamate Hydrobromide" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Spain" ], "orpha": null }, { "unii": "70097M6I30", "name": "MAGNESIUM STEARATE", "moiety": "", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "direct", "examples": [ "Magnesium Stearate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Portugal" ], "orpha": null }, { "unii": "05VP796JC6", "name": "MEQUITAZINE, (R)-", "moiety": "MEQUITAZINE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Mequitazine" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "France" ], "orpha": null }, { "unii": "JD546Z56F0", "name": "MERCURIC CHLORIDE, AMMONIATED", "moiety": "MERCURIC CHLORIDE AMMONIATED", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Mercuric Chloride, Ammoniated" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Germany" ], "orpha": null }, { "unii": "7SPF5O5BW8", "name": "METANIL YELLOW", "moiety": "ACID YELLOW 36", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Acid Yellow 36" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Germany" ], "orpha": null }, { "unii": "196OC77688", "name": "METHYL METHACRYLATE", "moiety": "METHYL METHACRYLATE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Methyl Methacrylate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['UNAPPROVED HOMEOPATHIC']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Germany" ], "orpha": null }, { "unii": "YX089CPS05", "name": "METHYLDIBROMOGLUTARONITRILE", "moiety": "METHYLDIBROMO GLUTARONITRILE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Methyldibromo Glutaronitrile" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Germany" ], "orpha": null }, { "unii": "229D0E1QFA", "name": "METHYLISOTHIAZOLINONE", "moiety": "METHYLISOTHIAZOLINONE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Methylisothiazolinone" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['OTC MONOGRAPH FINAL']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Germany" ], "orpha": null }, { "unii": "37X3OTM55N", "name": "N,S-DIACETYLCYSTEINE METHYL ESTER", "moiety": "N S DIACETYL METHYL CYSTEINATE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "N,s-Diacetyl Methyl Cysteinate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "France" ], "orpha": null }, { "unii": "50Z9596NJ3", "name": "N-(CYCLOHEXYLTHIO)PHTHALIMIDE", "moiety": "N CYCLOHEXYLTHIO PHTHALIMIDE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "N-(Cyclohexylthio)phthalimide" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Germany" ], "orpha": null }, { "unii": "4LFS24GD0V", "name": "OCTHILINONE", "moiety": "OCTHILINONE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Octhilinone" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Germany" ], "orpha": null }, { "unii": "079IIA2811", "name": "OCTYL GALLATE", "moiety": "OCTYL GALLATE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Octyl Gallate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Germany" ], "orpha": null }, { "unii": "JA08T3B97O", "name": "ORNITHINE ASPARTATE", "moiety": "ORNITHINE L FORM", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Ornithine L-Form Aspartate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Poland" ], "orpha": null }, { "unii": "R7P8FRP05V", "name": "P-AMINOPHENOL", "moiety": "4 AMINOPHENOL", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "4-Aminophenol" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Germany" ], "orpha": null }, { "unii": "5TWQ1V240M", "name": "PALLADIUM", "moiety": "PALLADIUM", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Palladium Chloride" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['UNAPPROVED DRUG OTHER', 'UNAPPROVED HOMEOPATHIC']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Germany" ], "orpha": null }, { "unii": "7K81IL792L", "name": "PANTETHINE", "moiety": "PANTETHINE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Pantethine" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Italy" ], "orpha": null }, { "unii": "2RDB26I5ZB", "name": "PARTHENOLIDE", "moiety": "PARTHENOLIDE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Parthenolide" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Germany" ], "orpha": null }, { "unii": "HIE492ZZ3T", "name": "PHENOXYETHANOL", "moiety": "PHENOXYETHANOL", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Phenoxyethanol" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['OTC MONOGRAPH DRUG', 'OTC MONOGRAPH FINAL', 'UNAPPROVED DRUG OTHER']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Germany" ], "orpha": null }, { "unii": "27YLU75U4W", "name": "PHOSPHORUS", "moiety": "PHOSPHORUS", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Phosphorus" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['DRUG FOR FURTHER PROCESSING', 'UNAPPROVED DRUG OTHER', 'UNAPPROVED HOMEOPATHIC']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Germany" ], "orpha": null }, { "unii": "654G2VCI4Q", "name": "PICOTAMIDE", "moiety": "PICOTAMIDE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Picotamide Monohydrate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Italy" ], "orpha": null }, { "unii": "ZA38L5WD1K", "name": "PIPETHANATE ETHOBROMIDE", "moiety": "PIPETHANATE ETHOBROMIDE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Pipethanate Ethobromide" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Italy" ], "orpha": null }, { "unii": "RWP5GA015D", "name": "POTASSIUM", "moiety": "", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "direct", "examples": [ "Potassium" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['UNAPPROVED DRUG OTHER', 'UNAPPROVED HOMEOPATHIC']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "France" ], "orpha": null }, { "unii": "OC4598NZEQ", "name": "POTASSIUM ASPARTATE", "moiety": "", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "direct", "examples": [ "Potassium Aspartate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['UNAPPROVED HOMEOPATHIC']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Italy" ], "orpha": null }, { "unii": "96F264O9SV", "name": "PROPYL ALCOHOL", "moiety": "1 PROPANOL", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "1-Propanol" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Germany" ], "orpha": null }, { "unii": "YUL4LO94HK", "name": "RESORCINOL", "moiety": "RESORCINOL", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Resorcinol" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['OTC MONOGRAPH DRUG', 'UNAPPROVED HOMEOPATHIC']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Germany" ], "orpha": null }, { "unii": "MLT4718TJW", "name": "RUBIDIUM", "moiety": "RUBIDIUM", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Rubidium Chloride" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['UNAPPROVED HOMEOPATHIC']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Netherlands" ], "orpha": null }, { "unii": "FST467XS7D", "name": "SACCHARIN", "moiety": "1 2 BENZISOTHIAZOLIN 3 ONE 1 1 DIOXIDE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "1,2-Benzisothiazolin-3-One 1,1-Dioxide Sodium Salt" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT FOR HUMAN PRESCRIPTION COMPOUNDING', 'UNAPPROVED HOMEOPATHIC']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Germany" ], "orpha": null }, { "unii": "690VN2L7TK", "name": "SODIUM 2,3-DIMERCAPTO-1-PROPANESULFONATE", "moiety": "UNITHIOL", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Unithiol", "Unithiol Monohydrate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT', 'BULK INGREDIENT FOR HUMAN PRESCRIPTION COMPOUNDING']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Germany" ], "orpha": null }, { "unii": "X75ZKL134L", "name": "SODIUM BENZYLPENILLOATE", "moiety": "SODIUM BENZYLPENILLOATE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Sodium Benzylpenilloate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Germany" ], "orpha": null }, { "unii": "LC1V549NOM", "name": "SODIUM BROMIDE", "moiety": "", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "direct", "examples": [ "Sodium Bromide" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['UNAPPROVED HOMEOPATHIC']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Bulgaria" ], "orpha": null }, { "unii": "4VON5FNS3C", "name": "SODIUM METABISULFITE", "moiety": "SODIUM METABISULFITE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Sodium Metabisulfite" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Germany" ], "orpha": null }, { "unii": "94255I6E2T", "name": "SODIUM PHOSPHATE, DIBASIC, DIHYDRATE", "moiety": "", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "direct", "examples": [ "Disodium Phosphate Dihydrate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT', 'DRUG FOR FURTHER PROCESSING']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Belgium" ], "orpha": null }, { "unii": "X045WJ989B", "name": "SORBIC ACID", "moiety": "SORBIC", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Sorbic Acid" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT FOR HUMAN PRESCRIPTION COMPOUNDING', 'OTC MONOGRAPH FINAL', 'UNAPPROVED HOMEOPATHIC']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Germany" ], "orpha": null }, { "unii": "C151H8M554", "name": "SUCROSE", "moiety": "SUCROSE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Sucrose" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['DRUG FOR FURTHER PROCESSING', 'UNAPPROVED HOMEOPATHIC']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Netherlands" ], "orpha": null }, { "unii": "6032D45BEM", "name": "SURAMIN", "moiety": "SURAMIN", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Suramin Sodium" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Germany" ], "orpha": null }, { "unii": "S3V868548T", "name": "TERPIN HYDRATE", "moiety": "TERPINENE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Terpinene" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "France" ], "orpha": null }, { "unii": "C12674942B", "name": "TERT-BUTYLHYDROQUINONE", "moiety": "TERT BUTYLHYDROQUINONE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Tert-Butylhydroquinone" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Germany" ], "orpha": null }, { "unii": "H0KNQ242J5", "name": "TESTOSTERONE ISOBUTYRATE", "moiety": "TESTOSTERONE ISOBUTYRATE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Testosterone Isobutyrate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Slovakia" ], "orpha": null }, { "unii": "549BT7IE1Q", "name": "TETRACHLORODECAOXIDE", "moiety": "TETRACHLORODECAOXIDE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Tetrachlorodecaoxide" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Portugal" ], "orpha": null }, { "unii": "9YCB5VR86Z", "name": "THIOCARBANILIDE", "moiety": "DIPHENYLTHIOUREA", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Diphenylthiourea" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Germany" ], "orpha": null }, { "unii": "KL096K0KXL", "name": "TIMONACIC, (R)-", "moiety": "TIMONACIC", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Timonacic" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Poland" ], "orpha": null }, { "unii": "387GMG9FH5", "name": "TIN", "moiety": "TIN", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Tin" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['UNAPPROVED HOMEOPATHIC']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Germany" ], "orpha": null }, { "unii": "5V2JDO056X", "name": "TRICHLOROACETIC ACID", "moiety": "TRICHLOROACETIC", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Trichloroacetic Acid" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Italy" ], "orpha": null }, { "unii": "14I47YJ5EY", "name": "TRIETHYLENE GLYCOLDIMETHACRYLATE", "moiety": "TRIETHYLENE GLYCOL DIMETHACRYLATE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Triethylene Glycol Dimethacrylate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Germany" ], "orpha": null }, { "unii": "OU2JEB22IE", "name": "TRIS(N-HYDROXYETHYL) HEXAHYDROTRIAZINE", "moiety": "TRIS N HYDROXYETHYL HEXAHYDROTRIAZINE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Tris(N-Hydroxyethyl) Hexahydrotriazine" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Germany" ], "orpha": null }, { "unii": "9870N09I68", "name": "UNDECYLENIC ACID MONOETHANOLAMIDE", "moiety": "UNDECYLENIC ACID MONOETHANOLAMIDE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Undecylenic Acid Monoethanolamide" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Latvia" ], "orpha": null }, { "unii": "CHI530446X", "name": "VANILLIN", "moiety": "VANILLIN", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Vanillin" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Germany" ], "orpha": null }, { "unii": "3H3U766W84", "name": "XENON", "moiety": "XENON", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Xenon" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Germany" ], "orpha": null, "eu_orphan_designation": { "use": "Treatment of ischaemia/reperfusion injury associated with cardiac arrest", "status": "Positive", "number": "EU/3/16/1768" }, "eu_orphan_designations": [ { "use": "Treatment of ischaemia/reperfusion injury associated with cardiac arrest", "status": "Positive", "number": "EU/3/16/1768" }, { "use": "Treatment of perinatal asphyxia", "status": "Positive", "number": "EU/3/15/1483" } ] }, { "unii": "T967Q579KA", "name": "ZINC DL-ASPARTATE", "moiety": "ZINC BIS HYDROGEN DL", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Zinc-Bis(Hydrogen-Dl-Aspartate)" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "STEREO_NAME: name carries an enantiomer/racemate marker; check the counterpart's US status by hand" ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Germany" ], "orpha": null }, { "unii": "R5Y8O51589", "name": "ANECORTAVE", "moiety": "ANECORTAVE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EMA" ], "orphan": false, "tier": "moiety-base", "examples": [ "anecortave acetate" ], "area": "Wet Macular Degeneration", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "never_authorised", "status_by_source": { "EMA": "never_authorised" }, "n_countries": 0, "countries": [], "orpha": null }, { "unii": "0R0008Q3JB", "name": "CHROMIUM", "moiety": "CHROMIUM", "inn": false, "usan": false, "type": "chemical", "sources": [ "Swissmedic" ], "orphan": false, "tier": "exact-moiety", "examples": [ "chromium" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT FOR HUMAN PRESCRIPTION COMPOUNDING', 'UNAPPROVED DRUG OTHER', 'UNAPPROVED HOMEOPATHIC']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "Swissmedic": "authorised" }, "n_countries": 0, "countries": [], "orpha": null }, { "unii": "N0KCY1368Z", "name": "CUPRIC ACETATE CU-64", "moiety": "COPPER 64CU", "inn": false, "usan": false, "type": "chemical", "sources": [ "EMA" ], "orphan": false, "tier": "moiety-base", "examples": [ "copper (64Cu) chloride" ], "area": "Radionuclide Imaging", "atc": "Not yet assigned", "indication": "Cuprymina is a radiopharmaceutical precursor. It is not intended for direct use in patients. This medicinal product must be used only for the radiolabelling of carrier molecules, which have been specifically developed and authorised for radiolabelling with this radionuclide.", "approved": "23/08/2012", "flags": [], "foreign_status": "authorised", "status_by_source": { "EMA": "authorised" }, "n_countries": 0, "countries": [], "orpha": null }, { "unii": "1NKX3648J9", "name": "DICHLOROBENZYL ALCOHOL", "moiety": "DICHLOROBENZYL ALCOHOL", "inn": false, "usan": false, "type": "chemical", "sources": [ "Health Canada" ], "orphan": false, "tier": "exact-moiety", "examples": [ "DICHLOROBENZYL ALCOHOL" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "Health Canada": "authorised" }, "n_countries": 0, "countries": [], "orpha": null }, { "unii": "FC72KVT52F", "name": "ETHYLENE GLYCOL", "moiety": "GLYCOL", "inn": false, "usan": false, "type": "chemical", "sources": [ "Health Canada" ], "orphan": false, "tier": "moiety-base", "examples": [ "GLYCOL SALICYLATE" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['OTC MONOGRAPH DRUG']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "Health Canada": "authorised" }, "n_countries": 0, "countries": [], "orpha": null }, { "unii": "14255EXE39", "name": "ETHYLPARABEN", "moiety": "E 214", "inn": false, "usan": false, "type": "chemical", "sources": [ "Swissmedic" ], "orphan": false, "tier": "exact-moiety", "examples": [ "E 214" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['OTC MONOGRAPH FINAL']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "Swissmedic": "authorised" }, "n_countries": 0, "countries": [], "orpha": null }, { "unii": "6JKA7MAH9C", "name": "GUAIACOL", "moiety": "GUAIACOL", "inn": false, "usan": false, "type": "chemical", "sources": [ "Health Canada" ], "orphan": false, "tier": "moiety-base", "examples": [ "GUAIACOL CARBONATE" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "Health Canada": "authorised" }, "n_countries": 0, "countries": [], "orpha": null }, { "unii": "6634O61GOI", "name": "MOLYBDENUM MO-99", "moiety": "MOLYBDENUM 99 MO", "inn": false, "usan": false, "type": "chemical", "sources": [ "Swissmedic" ], "orphan": false, "tier": "exact-moiety", "examples": [ "molybdenum(99-Mo)" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['DRUG FOR FURTHER PROCESSING']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "Swissmedic": "authorised" }, "n_countries": 0, "countries": [], "orpha": null }, { "unii": "E775ICF82G", "name": "OCTANOIC ACID 1-C-13", "moiety": "1 13C CAPRYLIC", "inn": false, "usan": false, "type": "chemical", "sources": [ "EMA" ], "orphan": false, "tier": "moiety-base", "examples": [ "1-13C-caprylic acid" ], "area": "Gastric Emptying;Breath Tests", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "never_authorised", "status_by_source": { "EMA": "never_authorised" }, "n_countries": 0, "countries": [], "orpha": null }, { "unii": "UA8U0KJM72", "name": "PAMABROM", "moiety": "PAMABROM", "inn": false, "usan": false, "type": "chemical", "sources": [ "Health Canada" ], "orphan": false, "tier": "exact-moiety", "examples": [ "PAMABROM" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT', 'DRUG FOR FURTHER PROCESSING', 'OTC MONOGRAPH DRUG', 'UNAPPROVED DRUG OTHER']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "Health Canada": "authorised" }, "n_countries": 0, "countries": [], "orpha": null }, { "unii": "F1CB00L2RH", "name": "PSMA-1007 F-18", "moiety": "18F PSMA 1007", "inn": false, "usan": false, "type": "chemical", "sources": [ "Swissmedic" ], "orphan": false, "tier": "exact-moiety", "examples": [ "(18F)-PSMA-1007" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "authorised", "status_by_source": { "Swissmedic": "authorised" }, "n_countries": 0, "countries": [], "orpha": null }, { "unii": "3J50XA376E", "name": "THYMOL", "moiety": "THYMOLUM", "inn": false, "usan": false, "type": "chemical", "sources": [ "Swissmedic" ], "orphan": false, "tier": "exact-moiety", "examples": [ "thymolum" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT', 'OTC MONOGRAPH DRUG', 'UNAPPROVED HOMEOPATHIC']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "Swissmedic": "authorised" }, "n_countries": 0, "countries": [], "orpha": null } ], "review": [ { "unii": "7A997Q53NR", "name": "ZOPICLONE, (R)-", "moiety": "ZOPICLONE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)", "Health Canada" ], "orphan": false, "tier": "exact-moiety", "examples": [ "ZOPICLONE", "Zopiclone" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "STEREO_US_PARTNER: ESZOPICLONE is US-approved (stereo-unii:UZX80K71OE); s.529 treatment of enantiomer/isomer pairs is unsettled and GSRS carries no stereo edge for this pair", "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised", "Health Canada": "authorised" }, "n_countries": 28, "countries": [ "Austria", "Belgium", "Bulgaria", "Croatia", "Czech Republic", "Denmark", "Estonia", "Finland", "France", "Germany", "Greece", "Hungary", "Iceland", "Ireland", "Italy", "Latvia", "Lithuania", "Luxembourg", "Netherlands", "Norway", "Poland", "Portugal", "Romania", "Slovakia", "Slovenia", "Spain", "Sweden", "United Kingdom (Northern Ireland)" ], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "HUMAN", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "screen could not settle it (see funnelA_review.csv flags)" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "BZ1R15MTK7", "name": "LEVOMENTHOL", "moiety": "LEVOMENTHOL", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)", "Health Canada" ], "orphan": false, "tier": "exact-moiety", "examples": [ "LEVOMENTHOL", "Levomenthol", "Levomenthol Ph. Eur." ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "STEREO_US_PARTNER: MENTHOL is US-approved (stereo-unii:C6B1OE8P3W); s.529 treatment of enantiomer/isomer pairs is unsettled and GSRS carries no stereo edge for this pair", "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT', 'OTC MONOGRAPH DRUG', 'OTC MONOGRAPH NOT FINAL', 'UNAPPROVED DRUG OTHER']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised", "Health Canada": "authorised" }, "n_countries": 2, "countries": [ "Bulgaria", "United Kingdom (Northern Ireland)" ], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "HUMAN", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "screen could not settle it (see funnelA_review.csv flags)" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "671DKG7P5S", "name": "DEXIBUPROFEN", "moiety": "DEXIBUPROFEN", "inn": true, "usan": true, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Dexibuprofen" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "STEREO_US_PARTNER: IBUPROFEN is US-approved (stereo-unii:99W8H60N62); s.529 treatment of enantiomer/isomer pairs is unsettled and GSRS carries no stereo edge for this pair", "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 14, "countries": [ "Austria", "Czech Republic", "Denmark", "Germany", "Greece", "Hungary", "Italy", "Poland", "Portugal", "Romania", "Slovakia", "Spain", "Sweden", "United Kingdom (Northern Ireland)" ], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "HUMAN", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "screen could not settle it (see funnelA_review.csv flags)" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "6Y75Z4E8NS", "name": "LEVOMETHADONE", "moiety": "LEVOMETHADONE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Levomethadone Hydrochloride" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "STEREO_US_RELATIVE: RACEMATE->ACTIVE ENANTIOMER -> Methadone is US-approved (gsrs-unii:S95RZH8AMH); s.529 treatment of enantiomer/racemate pairs is unsettled", "STEREO_US_RELATIVE: RACEMATE->ACTIVE ENANTIOMER -> Methadone is US-approved (gsrs-unii:S95RZH8AMH); s.529 treatment of enantiomer/racemate pairs is unsettled", "STEREO_US_RELATIVE: ENANTIOMER->ENANTIOMER -> Esmethadone is US-approved (gsrs-unii:S95RZH8AMH); s.529 treatment of enantiomer/racemate pairs is unsettled", "STEREO_US_RELATIVE: ENANTIOMER->ENANTIOMER -> Esmethadone is US-approved (gsrs-unii:S95RZH8AMH); s.529 treatment of enantiomer/racemate pairs is unsettled", "STEREO_US_RELATIVE: ACTIVE ENANTIOMER->RACEMATE -> Methadone is US-approved (gsrs-unii:S95RZH8AMH); s.529 treatment of enantiomer/racemate pairs is unsettled", "STEREO_US_RELATIVE: ACTIVE ENANTIOMER->RACEMATE -> Methadone is US-approved (gsrs-unii:S95RZH8AMH); s.529 treatment of enantiomer/racemate pairs is unsettled", "STEREO_US_PARTNER: METHADONE is US-approved (stereo-unii:S95RZH8AMH); s.529 treatment of enantiomer/isomer pairs is unsettled and GSRS carries no stereo edge for this pair" ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 14, "countries": [ "Austria", "Bulgaria", "Croatia", "Denmark", "Finland", "Germany", "Hungary", "Italy", "Norway", "Poland", "Romania", "Slovenia", "Spain", "Sweden" ], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "HUMAN", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "screen could not settle it (see funnelA_review.csv flags)" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "4MXU9LJS4Q", "name": "LEUCOVORIN SODIUM", "moiety": "SODIUM FOLINATE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Disodium Folinate" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "STEREO_US_PARTNER: LEVOLEUCOVORIN SODIUM is US-approved (stereo-key:LEVOLEUCOVORIN SODIUM); s.529 treatment of enantiomer/isomer pairs is unsettled and GSRS carries no stereo edge for this pair" ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 3, "countries": [ "Germany", "Luxembourg", "United Kingdom (Northern Ireland)" ], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "HUMAN", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "screen could not settle it (see funnelA_review.csv flags)" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "K76S41V71X", "name": "ACETYLLEUCINE", "moiety": "ACETYLLEUCINE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Acetylleucine" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "STEREO_US_PARTNER: LEVACETYLLEUCINE is US-approved (stereo-unii:E915HL7K2O); s.529 treatment of enantiomer/isomer pairs is unsettled and GSRS carries no stereo edge for this pair" ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "France" ], "orpha": null, "eu_orphan_designation": { "use": "Treatment of CACNA1A disorders", "status": "Positive", "number": "EU/3/26/3214" }, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "HUMAN", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "screen could not settle it (see funnelA_review.csv flags)" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] }, "eu_orphan_designations": [ { "use": "Treatment of CACNA1A disorders", "status": "Positive", "number": "EU/3/26/3214" }, { "use": "Treatment of ataxia-oculomotor apraxia", "status": "Positive", "number": "EU/3/24/2913" }, { "use": "Treatment of GM1 gangliosidosis", "status": "Positive", "number": "EU/3/22/2708" }, { "use": "Treatment of spinocerebellar ataxia", "status": "Positive", "number": "EU/3/18/2059" }, { "use": "Treatment of ataxia telangiectasia", "status": "Positive", "number": "EU/3/18/2124" }, { "use": "Treatment of Niemann-Pick disease", "status": "Positive", "number": "EU/3/17/1848" }, { "use": "Treatment of GM2 gangliosidosis", "status": "Positive", "number": "EU/3/17/1949" } ] }, { "unii": "U9VY0ZOK7A", "name": "CARNITINE, (+)-", "moiety": "CARNITINE", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "exact-moiety", "examples": [ "Carnitine" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "STEREO_US_PARTNER: LEVOCARNITINE is US-approved (stereo-unii:0G389FZZ9M); s.529 treatment of enantiomer/isomer pairs is unsettled and GSRS carries no stereo edge for this pair", "US_NON_APPLICATION_MARKETING: sold in the US under ['UNAPPROVED HOMEOPATHIC']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Greece" ], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "HUMAN", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "screen could not settle it (see funnelA_review.csv flags)" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "01Q0586BG1", "name": "SORBITOL, L-", "moiety": "SORBITOL L", "inn": false, "usan": false, "type": "chemical", "sources": [ "EU national (Art57)" ], "orphan": false, "tier": "moiety-base", "examples": [ "Sorbitol, L-Arginine Hydrochloride" ], "area": "", "atc": "", "indication": "", "approved": "", "flags": [ "STEREO_US_PARTNER: SORBITOL is US-approved (stereo-unii:506T60A25R); s.529 treatment of enantiomer/isomer pairs is unsettled and GSRS carries no stereo edge for this pair" ], "foreign_status": "authorised", "status_by_source": { "EU national (Art57)": "authorised" }, "n_countries": 1, "countries": [ "Romania" ], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "HUMAN", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "screen could not settle it (see funnelA_review.csv flags)" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "R6D2UI4FLS", "name": "ENCLOMIPHENE", "moiety": "ENCLOMIFENE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EMA" ], "orphan": false, "tier": "moiety-base", "examples": [ "enclomifene citrate" ], "area": "Hypogonadism", "atc": "G03X", "indication": "Treatment of hypogonadotropic hypogonadism in men", "approved": "", "flags": [ "STEREO_US_PARTNER: CLOMIPHENE is US-approved (stereo-unii:1HRS458QU2); s.529 treatment of enantiomer/isomer pairs is unsettled and GSRS carries no stereo edge for this pair", "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT', 'BULK INGREDIENT FOR HUMAN PRESCRIPTION COMPOUNDING']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "never_authorised", "status_by_source": { "EMA": "never_authorised" }, "n_countries": 0, "countries": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "HUMAN", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "screen could not settle it (see funnelA_review.csv flags)" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } } ], "dossier_only": [ { "unii": "CEG1Q6OGU1", "name": "BARDOXOLONE METHYL", "moiety": "BARDOXOLONE METHYL", "inn": false, "usan": true, "type": "chemical", "sources": [ "EMA" ], "orphan": true, "tier": "exact-moiety", "examples": [ "Bardoxolone methyl" ], "area": "", "atc": "", "indication": "Treatment of chronic kidney disease", "approved": "", "flags": [], "foreign_status": "never_authorised", "status_by_source": { "EMA": "never_authorised" }, "n_countries": 0, "countries": [], "orpha": null, "eu_orphan_designation": { "use": "Treatment of autosomal dominant polycystic kidney disease", "status": "Positive", "number": "EU/3/21/2480" }, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] }, "eu_orphan_designations": [ { "use": "Treatment of autosomal dominant polycystic kidney disease", "status": "Positive", "number": "EU/3/21/2480" }, { "use": "Treatment of Alport syndrome", "status": "Positive", "number": "EU/3/18/2019" } ] }, { "unii": "GKF8S4C432", "name": "CATEQUENTINIB", "moiety": "CATEQUENTINIB", "inn": true, "usan": true, "type": "chemical", "sources": [ "EMA" ], "orphan": true, "tier": "moiety-base", "examples": [ "catequentinib dihydrochloride" ], "area": "Sarcoma;Leiomyosarcoma", "atc": "L01EX", "indication": "Treatment of synovial sarcoma or leiomyosarcoma.", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "never_authorised", "status_by_source": { "EMA": "never_authorised" }, "n_countries": 0, "countries": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "6QFP76V7S7", "name": "EPRODISATE", "moiety": "EPRODISATE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EMA" ], "orphan": true, "tier": "moiety-base", "examples": [ "eprodisate disodium" ], "area": "Amyloidosis", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "never_authorised", "status_by_source": { "EMA": "never_authorised" }, "n_countries": 0, "countries": [], "orpha": { "code": "69", "name": "Amyloidosis", "rarity": "rare", "rarity_evidence": "Orphanet 1-9 / 100 000 (Korea, Republic of) \u2248 16,750 in the US, under 200,000", "us_prevalence_est": 16750, "onset": "mixed", "onset_evidence": "Orphanet age of onset: All ages \u2014 spans pediatric and adult" }, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "PASS", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "Orphanet 1-9 / 100 000 (Korea, Republic of) \u2248 16,750 in the US, under 200,000" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "Orphanet age of onset: All ages \u2014 spans pediatric and adult" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "6JLQ8K35KK", "name": "GOVORESTAT", "moiety": "GOVORESTAT", "inn": true, "usan": true, "type": "chemical", "sources": [ "EMA" ], "orphan": true, "tier": "exact-moiety", "examples": [ "govorestat" ], "area": "Galactosemias", "atc": "A16AX24", "indication": "Treatment of adults and children aged 2 years and older with a confirmed diagnosis of classic galactosemia.", "approved": "", "flags": [ "SPONSOR_CHANGED: Applied Therapeutics acquired by Cycle Group Holdings 2026-02-03 and deregistered (Form 15) \u2014 EDGAR monitoring will no longer see this sponsor; holds FDA orphan + RPD designations (galactosemia) and a 2024-11-27 CRL" ], "foreign_status": "never_authorised", "status_by_source": { "EMA": "never_authorised" }, "n_countries": 0, "countries": [], "orpha": null, "eu_orphan_designation": { "use": "Treatment of galactosaemia", "status": "Positive", "number": "EU/3/22/2642" }, "us_crl": { "application": "NDA 219195", "date": "11/27/2024", "kind": "COMPLETE RESPONSE", "approval_status": "Unapproved", "company": "Applied Therapeutics, Inc.", "letters": 1, "source": "openFDA CRL dataset" }, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] }, "eu_orphan_designations": [ { "use": "Treatment of Charcot-Marie-Tooth disease", "status": "Positive", "number": "EU/3/23/2783" }, { "use": "Treatment of galactosaemia", "status": "Positive", "number": "EU/3/22/2642" } ] }, { "unii": "UJ0ZJ76DO9", "name": "GUSPERIMUS", "moiety": "GUSPERIMUS", "inn": true, "usan": false, "type": "chemical", "sources": [ "EMA" ], "orphan": true, "tier": "moiety-base", "examples": [ "gusperimus trihydrochloride" ], "area": "Wegener Granulomatosis", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "never_authorised", "status_by_source": { "EMA": "never_authorised" }, "n_countries": 0, "countries": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "M59NC4E26P", "name": "MASITINIB", "moiety": "MASITINIB", "inn": true, "usan": false, "type": "chemical", "sources": [ "EMA" ], "orphan": true, "tier": "moiety-base", "examples": [ "Masitinib mesilate", "masitinib" ], "area": "Amyotrophic Lateral Sclerosis", "atc": "L01EX06", "indication": "In combination with riluzole for the treatment of adult patients with amyotrophic lateral sclerosis (ALS)", "approved": "", "flags": [], "foreign_status": "never_authorised", "status_by_source": { "EMA": "never_authorised" }, "n_countries": 0, "countries": [], "orpha": { "code": "803", "name": "Amyotrophic lateral sclerosis", "rarity": "rare", "rarity_evidence": "Orphanet 1-9 / 100 000 (Worldwide) \u2248 16,750 in the US, under 200,000", "us_prevalence_est": 16750, "onset": "adult", "onset_evidence": "Orphanet age of onset: Adult \u2014 adult/elderly" }, "eligibility": { "verdict": "INELIGIBLE", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "PASS", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "Orphanet 1-9 / 100 000 (Worldwide) \u2248 16,750 in the US, under 200,000" }, { "key": "primarily_pediatric", "status": "FAIL", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "Orphanet age of onset: Adult \u2014 adult/elderly" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "Y76ID234HW", "name": "PLITIDEPSIN", "moiety": "PLITIDEPSIN", "inn": true, "usan": false, "type": "chemical", "sources": [ "EMA" ], "orphan": true, "tier": "exact-moiety", "examples": [ "plitidepsin" ], "area": "Multiple Myeloma", "atc": "L01", "indication": "Treatment of multiple myeloma ", "approved": "", "flags": [ "AUTHORISED_ELSEWHERE (TGA Australia): approved Dec 2018 (Aplidin, multiple myeloma); EMA row is a withdrawn application. Not visible to the registers loaded here." ], "foreign_status": "never_authorised", "status_by_source": { "EMA": "never_authorised" }, "n_countries": 0, "countries": [], "orpha": { "code": "29073", "name": "Multiple myeloma", "rarity": "rare", "rarity_evidence": "Orphanet 1-5 / 10 000 (Europe) \u2248 100,499 in the US, under 200,000", "us_prevalence_est": 100499, "onset": "adult", "onset_evidence": "Orphanet age of onset: Adult \u2014 adult/elderly" }, "eu_orphan_designation": { "use": "Treatment of post-essential thrombocythaemia myelofibrosis", "status": "Withdrawn", "number": "EU/3/10/838" }, "eligibility": { "verdict": "INELIGIBLE", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "PASS", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "Orphanet 1-5 / 10 000 (Europe) \u2248 100,499 in the US, under 200,000" }, { "key": "primarily_pediatric", "status": "FAIL", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "Orphanet age of onset: Adult \u2014 adult/elderly" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] }, "eu_orphan_designations": [ { "use": "Treatment of post-essential thrombocythaemia myelofibrosis", "status": "Withdrawn", "number": "EU/3/10/838" }, { "use": "Treatment of post-polycythaemia vera myelofibrosis", "status": "Withdrawn", "number": "EU/3/10/839" }, { "use": "Treatment of primary myelofibrosis", "status": "Withdrawn", "number": "EU/3/10/837" } ] }, { "unii": "HD4TW8S2VK", "name": "PRIDOPIDINE", "moiety": "PRIDOPIDINE", "inn": true, "usan": true, "type": "chemical", "sources": [ "EMA" ], "orphan": true, "tier": "moiety-base", "examples": [ "pridopidine hydrochloride" ], "area": "Huntington Disease", "atc": "N07", "indication": "Treatment of Huntington's disease ", "approved": "", "flags": [], "foreign_status": "never_authorised", "status_by_source": { "EMA": "never_authorised" }, "n_countries": 0, "countries": [], "orpha": { "code": "399", "name": "Huntington disease", "rarity": "rare", "rarity_evidence": "Orphanet 1-9 / 100 000 (United States) \u2248 16,750 in the US, under 200,000", "us_prevalence_est": 16750, "onset": "mixed", "onset_evidence": "Orphanet age of onset: Adolescent, Adult, Childhood, Elderly \u2014 spans pediatric and adult" }, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "PASS", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "Orphanet 1-9 / 100 000 (United States) \u2248 16,750 in the US, under 200,000" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "Orphanet age of onset: Adolescent, Adult, Childhood, Elderly \u2014 spans pediatric and adult" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "1578EUB98L", "name": "RESMINOSTAT", "moiety": "RESMINOSTAT", "inn": true, "usan": false, "type": "chemical", "sources": [ "EMA" ], "orphan": true, "tier": "exact-moiety", "examples": [ "resminostat" ], "area": "Mycosis Fungoides;Sezary Syndrome", "atc": "L01X", "indication": "Treatment of patients with advanced stage mycosis fungoides (MF) and S\u00e9zary syndrome (SS)", "approved": "", "flags": [], "foreign_status": "never_authorised", "status_by_source": { "EMA": "never_authorised" }, "n_countries": 0, "countries": [], "orpha": null, "eu_orphan_designation": { "use": "Treatment of cutaneous T-cell lymphoma", "status": "Positive", "number": "EU/3/23/2835" }, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] }, "eu_orphan_designations": [ { "use": "Treatment of Hodgkin's lymphoma", "status": "Withdrawn", "number": "EU/3/11/930" }, { "use": "Treatment of hepatocellular carcinoma", "status": "Withdrawn", "number": "EU/3/11/913" }, { "use": "Treatment of cutaneous T-cell lymphoma", "status": "Positive", "number": "EU/3/23/2835" } ] }, { "unii": "H19C446XXB", "name": "RUBITECAN", "moiety": "RUBITECAN", "inn": true, "usan": true, "type": "chemical", "sources": [ "EMA" ], "orphan": true, "tier": "exact-moiety", "examples": [ "rubitecan" ], "area": "Pancreatic Neoplasms", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "never_authorised", "status_by_source": { "EMA": "never_authorised" }, "n_countries": 0, "countries": [], "orpha": null, "eu_orphan_designation": { "use": "Treatment of pancreatic cancer", "status": "Positive", "number": "EU/3/03/145" }, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] }, "eu_orphan_designations": [ { "use": "Treatment of pancreatic cancer", "status": "Positive", "number": "EU/3/03/145" } ] }, { "unii": "DD5RBA1NKF", "name": "ANAMORELIN", "moiety": "ANAMORELIN", "inn": true, "usan": false, "type": "chemical", "sources": [ "EMA" ], "orphan": false, "tier": "moiety-base", "examples": [ "anamorelin hydrochloride" ], "area": "Cachexia;Anorexia;Carcinoma, Non-Small-Cell Lung", "atc": "V03", "indication": "Treatment of anorexia, cachexia or unintended weight loss in adult patients with non-small cell lung cancer (NSCLC)", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT', 'BULK INGREDIENT FOR HUMAN PRESCRIPTION COMPOUNDING']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs.", "AUTHORISED_ELSEWHERE (PMDA Japan): approved Jan 2021 (Adlumiz, cancer cachexia); EMA row is a refusal. Not visible to the registers loaded here." ], "foreign_status": "never_authorised", "status_by_source": { "EMA": "never_authorised" }, "n_countries": 0, "countries": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "9T210MMZ3F", "name": "BLARCAMESINE", "moiety": "BLARCAMESINE", "inn": true, "usan": true, "type": "chemical", "sources": [ "EMA" ], "orphan": false, "tier": "moiety-base", "examples": [ "blarcamesine hydrochloride" ], "area": "", "atc": "N06D", "indication": "Treatment of Alzheimer\u2019s disease and dementia.", "approved": "", "flags": [], "foreign_status": "never_authorised", "status_by_source": { "EMA": "never_authorised" }, "n_countries": 0, "countries": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "P7725I9V3Z", "name": "CARISBAMATE", "moiety": "CARISBAMATE", "inn": true, "usan": true, "type": "chemical", "sources": [ "EMA" ], "orphan": false, "tier": "exact-moiety", "examples": [ "carisbamate" ], "area": "Epilepsy", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "never_authorised", "status_by_source": { "EMA": "never_authorised" }, "n_countries": 0, "countries": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "3B03KPM27L", "name": "CASOPITANT", "moiety": "CASOPITANT", "inn": true, "usan": false, "type": "chemical", "sources": [ "EMA" ], "orphan": false, "tier": "exact-moiety", "examples": [ "casopitant" ], "area": "Vomiting;Postoperative Nausea and Vomiting;Cancer", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "never_authorised", "status_by_source": { "EMA": "never_authorised" }, "n_countries": 0, "countries": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "NQU9IPY4K9", "name": "CEDIRANIB", "moiety": "CEDIRANIB", "inn": true, "usan": true, "type": "chemical", "sources": [ "EMA" ], "orphan": false, "tier": "moiety-base", "examples": [ "cediranib maleate" ], "area": "Ovarian Neoplasms", "atc": "L01XE32", "indication": "Treatment of platinum sensitive relapsed (PSR) ovarian cancer", "approved": "", "flags": [], "foreign_status": "never_authorised", "status_by_source": { "EMA": "never_authorised" }, "n_countries": 0, "countries": [], "orpha": null, "eu_orphan_designation": { "use": "Treatment of ovarian cancer", "status": "Positive", "number": "EU/3/14/1303" }, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] }, "eu_orphan_designations": [ { "use": "Treatment of ovarian cancer", "status": "Positive", "number": "EU/3/14/1303" } ] }, { "unii": "958X4J301A", "name": "FALDAPREVIR", "moiety": "FALDAPREVIR", "inn": true, "usan": true, "type": "chemical", "sources": [ "EMA" ], "orphan": false, "tier": "exact-moiety", "examples": [ "faldaprevir" ], "area": "Hepatitis C, Chronic", "atc": "J05AE", "indication": "Treatment of chronic genotype-1 hepatitis C virus (HCV) infection.", "approved": "", "flags": [], "foreign_status": "never_authorised", "status_by_source": { "EMA": "never_authorised" }, "n_countries": 0, "countries": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "V72H9867WB", "name": "GARENOXACIN", "moiety": "GARENOXACIN", "inn": true, "usan": false, "type": "chemical", "sources": [ "EMA" ], "orphan": false, "tier": "moiety-base", "examples": [ "garenoxacin mesylate" ], "area": "Surgical Wound Infection;Sinusitis;Skin Diseases, Infectious;Pneumonia;Soft Tissue Infections;Bronchitis, Chronic", "atc": "", "indication": "", "approved": "", "flags": [ "AUTHORISED_ELSEWHERE (PMDA Japan): approved Jul 2007 (Geninax); EMA row is an withdrawn application. Not visible to the registers loaded here." ], "foreign_status": "never_authorised", "status_by_source": { "EMA": "never_authorised" }, "n_countries": 0, "countries": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "908SY76S4G", "name": "LAQUINIMOD", "moiety": "LAQUINIMOD", "inn": true, "usan": false, "type": "chemical", "sources": [ "EMA" ], "orphan": false, "tier": "exact-moiety", "examples": [ "laquinimod" ], "area": "Multiple Sclerosis", "atc": "N07", "indication": "Treatment of multiple sclerosis", "approved": "", "flags": [], "foreign_status": "never_authorised", "status_by_source": { "EMA": "never_authorised" }, "n_countries": 0, "countries": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "E0QN3D755O", "name": "LISURIDE", "moiety": "LISURIDE", "inn": true, "usan": false, "type": "chemical", "sources": [ "EMA" ], "orphan": false, "tier": "exact-moiety", "examples": [ "lisuride" ], "area": "Restless Legs Syndrome", "atc": "", "indication": "", "approved": "", "flags": [ "AUTHORISED_ELSEWHERE (EU national): nationally authorised for Parkinson's disease and hyperprolactinaemia; the EMA row is a withdrawn RLS application. Not visible to the registers loaded here." ], "foreign_status": "never_authorised", "status_by_source": { "EMA": "never_authorised" }, "n_countries": 0, "countries": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "YA84KI1VEW", "name": "MOLNUPIRAVIR", "moiety": "MOLNUPIRAVIR", "inn": true, "usan": true, "type": "chemical", "sources": [ "EMA" ], "orphan": false, "tier": "exact-moiety", "examples": [ "molnupiravir" ], "area": "COVID-19 virus infection", "atc": "J05A", "indication": "Treatment of coronavirus disease 2019 (COVID-19).", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT', 'DRUG FOR FURTHER PROCESSING']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "never_authorised", "status_by_source": { "EMA": "never_authorised" }, "n_countries": 0, "countries": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "8O74K609HN", "name": "OBICETRAPIB", "moiety": "OBICETRAPIB", "inn": true, "usan": true, "type": "chemical", "sources": [ "EMA" ], "orphan": false, "tier": "moiety-base", "examples": [ "obicetrapib hemicalcium" ], "area": "Hypercholesterolemia;Dyslipidemias", "atc": "C10A", "indication": "Treatment of primary hypercholesterolaemia or mixed dyslipidaemia.", "approved": "", "flags": [], "foreign_status": "never_authorised", "status_by_source": { "EMA": "never_authorised" }, "n_countries": 0, "countries": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "2M19539ERK", "name": "OMECAMTIV MECARBIL", "moiety": "OMECAMTIV MECARBIL", "inn": true, "usan": true, "type": "chemical", "sources": [ "EMA" ], "orphan": false, "tier": "moiety-base", "examples": [ "omecamtiv mecarbil dihydrochloride hydrate" ], "area": "Heart Failure, Systolic", "atc": "C01CX10", "indication": "Treatment of adult patients with symptomatic chronic heart failure and reduced ejection fraction less than 30%", "approved": "", "flags": [ "US_NON_APPLICATION_MARKETING: sold in the US under ['BULK INGREDIENT']. Does NOT disqualify (s.529 bars a prior approval in a 505 application, which these are not) but there is no exclusivity runway and supply already exists \u2014 and rows like this are often excipients or antiseptics rather than drugs." ], "foreign_status": "never_authorised", "status_by_source": { "EMA": "never_authorised" }, "n_countries": 0, "countries": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "OS7097575K", "name": "PARSACLISIB", "moiety": "PARSACLISIB", "inn": true, "usan": true, "type": "chemical", "sources": [ "EMA" ], "orphan": false, "tier": "moiety-base", "examples": [ "Parsaclisib hydrochloride" ], "area": "", "atc": "L01EM04", "indication": "Treatment of marginal zone lymphoma", "approved": "", "flags": [], "foreign_status": "never_authorised", "status_by_source": { "EMA": "never_authorised" }, "n_countries": 0, "countries": [], "orpha": { "code": "300912", "name": "Marginal zone lymphoma", "rarity": "rare", "rarity_evidence": "Orphanet 1-9 / 100 000 (Europe) \u2248 16,750 in the US, under 200,000", "us_prevalence_est": 16750, "onset": "adult", "onset_evidence": "Orphanet age of onset: Adult \u2014 adult/elderly" }, "eu_orphan_designation": { "use": "Treatment of marginal zone lymphoma", "status": "Withdrawn", "number": "EU/3/19/2185" }, "eligibility": { "verdict": "INELIGIBLE", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "PASS", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "Orphanet 1-9 / 100 000 (Europe) \u2248 16,750 in the US, under 200,000" }, { "key": "primarily_pediatric", "status": "FAIL", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "Orphanet age of onset: Adult \u2014 adult/elderly" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] }, "eu_orphan_designations": [ { "use": "Treatment of autoimmune haemolytic anaemia", "status": "Positive", "number": "EU/3/22/2659" }, { "use": "Treatment of marginal zone lymphoma", "status": "Withdrawn", "number": "EU/3/19/2185" } ] }, { "unii": "N0JWC7RO00", "name": "POVORCITINIB", "moiety": "POVORCITINIB", "inn": true, "usan": true, "type": "chemical", "sources": [ "EMA" ], "orphan": false, "tier": "moiety-base", "examples": [ "povorcitinib phosphate" ], "area": "Hidradenitis Suppurativa", "atc": "", "indication": "Treatment of active moderate to severe hidradenitis suppurativa (acne inversa) in adults", "approved": "", "flags": [], "foreign_status": "never_authorised", "status_by_source": { "EMA": "never_authorised" }, "n_countries": 0, "countries": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "721809WQCP", "name": "RUBOXISTAURIN", "moiety": "RUBOXISTAURIN", "inn": true, "usan": false, "type": "chemical", "sources": [ "EMA" ], "orphan": false, "tier": "exact-moiety", "examples": [ "ruboxistaurin" ], "area": "Diabetic Retinopathy", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "never_authorised", "status_by_source": { "EMA": "never_authorised" }, "n_countries": 0, "countries": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "AJS8S3P31H", "name": "SATAVAPTAN", "moiety": "SATAVAPTAN", "inn": true, "usan": false, "type": "chemical", "sources": [ "EMA" ], "orphan": false, "tier": "exact-moiety", "examples": [ "satavaptan" ], "area": "Hyponatremia", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "never_authorised", "status_by_source": { "EMA": "never_authorised" }, "n_countries": 0, "countries": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "8D7B37T28G", "name": "SATRAPLATIN", "moiety": "SATRAPLATIN", "inn": true, "usan": true, "type": "chemical", "sources": [ "EMA" ], "orphan": false, "tier": "exact-moiety", "examples": [ "satraplatin" ], "area": "Prostatic Neoplasms", "atc": "", "indication": "", "approved": "", "flags": [], "foreign_status": "never_authorised", "status_by_source": { "EMA": "never_authorised" }, "n_countries": 0, "countries": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "MNZ4OP95CF", "name": "SENAPARIB", "moiety": "SENAPARIB", "inn": true, "usan": false, "type": "chemical", "sources": [ "EMA" ], "orphan": false, "tier": "exact-moiety", "examples": [ "senaparib" ], "area": "Fallopian Tube Neoplasms;Peritoneal Neoplasms;Carcinoma, Ovarian Epithelial", "atc": "L01XK07", "indication": "Maintenance treatment of advanced epithelial high-grade ovarian, fallopian tube or primary peritoneal cancer", "approved": "", "flags": [], "foreign_status": "never_authorised", "status_by_source": { "EMA": "never_authorised" }, "n_countries": 0, "countries": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "9U1ETH79CK", "name": "SOLITHROMYCIN", "moiety": "SOLITHROMYCIN", "inn": true, "usan": true, "type": "chemical", "sources": [ "EMA" ], "orphan": false, "tier": "exact-moiety", "examples": [ "solithromycin" ], "area": "Tularemia;Pneumonia;Anthrax", "atc": "J01FA", "indication": "Treatment of bacterial infections", "approved": "", "flags": [], "foreign_status": "never_authorised", "status_by_source": { "EMA": "never_authorised" }, "n_countries": 0, "countries": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "B2K5L1L8S9", "name": "SURUFATINIB", "moiety": "SURUFATINIB", "inn": true, "usan": true, "type": "chemical", "sources": [ "EMA" ], "orphan": false, "tier": "exact-moiety", "examples": [ "surufatinib" ], "area": "Neuroendocrine Tumors", "atc": "L01EX01", "indication": "Treatment of progressive neuroendocrine tumours", "approved": "", "flags": [], "foreign_status": "never_authorised", "status_by_source": { "EMA": "never_authorised" }, "n_countries": 0, "countries": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } }, { "unii": "K6A90IIZ19", "name": "VOSAROXIN", "moiety": "VOSAROXIN", "inn": true, "usan": true, "type": "chemical", "sources": [ "EMA" ], "orphan": false, "tier": "exact-moiety", "examples": [ "vosaroxin" ], "area": "Leukemia, Myeloid, Acute", "atc": "L01", "indication": "Treatment acute myeloid leukaemia", "approved": "", "flags": [], "foreign_status": "never_authorised", "status_by_source": { "EMA": "never_authorised" }, "n_countries": 0, "countries": [], "orpha": { "code": "519", "name": "Acute myeloid leukemia", "rarity": "rare", "rarity_evidence": "Orphanet 1-5 / 10 000 (Europe) \u2248 100,499 in the US, under 200,000", "us_prevalence_est": 100499, "onset": "mixed", "onset_evidence": "Orphanet age of onset: All ages \u2014 spans pediatric and adult" }, "eu_orphan_designation": { "use": "Treatment of acute myeloid leukaemia", "status": "Positive", "number": "EU/3/12/990" }, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "PASS", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "Orphanet 1-5 / 10 000 (Europe) \u2248 100,499 in the US, under 200,000" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "Orphanet age of onset: All ages \u2014 spans pediatric and adult" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] }, "eu_orphan_designations": [ { "use": "Treatment of acute myeloid leukaemia", "status": "Positive", "number": "EU/3/12/990" } ] }, { "unii": "49HFB70472", "name": "XIMELAGATRAN", "moiety": "XIMELAGATRAN", "inn": true, "usan": true, "type": "chemical", "sources": [ "EMA" ], "orphan": false, "tier": "exact-moiety", "examples": [ "ximelagatran" ], "area": "Atrial Fibrillation", "atc": "", "indication": "", "approved": "", "flags": [ "AUTHORISED_ELSEWHERE (EU mutual recognition): authorised via MRP before 2006 withdrawal; the EMA centralised row is a withdrawn application. Not visible to the registers loaded here." ], "foreign_status": "never_authorised", "status_by_source": { "EMA": "never_authorised" }, "n_countries": 0, "countries": [], "orpha": null, "eligibility": { "verdict": "LIKELY_ELIGIBLE_PENDING_REVIEW", "criteria": [ { "key": "no_prior_us_active_moiety", "status": "PASS", "decidability": "machine", "citation": "s.529(a)(4)(B)(i)(I) / 360ff(a)(4)(B)(i)(I)", "note": "no US approval found across UNII variants, string keys and GSRS relatives" }, { "key": "rare_under_200k", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(3)(B)", "note": "US prevalence not supplied; no Orphanet match (a common-disease drug will not match)" }, { "key": "primarily_pediatric", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "no age-of-onset data resolved; must be read from Orphadata (if the disease is listed) or the literature" }, { "key": "serious_or_life_threatening", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(3)(A)", "note": "seriousness not assessed" }, { "key": "qualifies_for_priority_review", "status": "HUMAN", "decidability": "judgment", "citation": "s.529(a)(4)(C)", "note": "requires the US treatment landscape for this exact indication and a view on whether the drug is a significant improvement" }, { "key": "human_evidence_exists", "status": "UNKNOWN", "decidability": "data", "citation": "505(b)(2) reliance", "note": "no published human evidence located" }, { "key": "filed_as_505b1", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(B)(i)(II)", "note": "sponsor's filing decision; note Pub. L. 117-9 (2021) narrowed this to 505(b)(1) only, so a 505(b)(2) repurposing filing forfeits the voucher" }, { "key": "relies_on_pediatric_data", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(D)", "note": "sponsor's filing decision, not a drug property" }, { "key": "no_adult_indication_sought", "status": "HUMAN", "decidability": "design", "citation": "s.529(a)(4)(E)", "note": "sponsor's filing decision, not a drug property. Per FDA's 2019 draft guidance (Q5) a label covering adults AND children with the SAME rare pediatric disease is fine (cf. ZANVASTRO 2026-09, Emcitate 2026-09); what (E) bars is a separate adult indication" }, { "key": "voucher_awardable_before_sunset", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(b)(5)", "note": "no projected approval date supplied" }, { "key": "approved_after_30_sep_2016", "status": "UNKNOWN", "decidability": "data", "citation": "s.529(a)(4)(F)", "note": "no projected approval date supplied" } ] } } ], "orphadata": { "disorders_loaded": 7888, "candidates_matched": 11 }, "monitor": { "fedreg": { "count": 157, "recent": [ { "title": "Issuance of Priority Review Voucher; Rare Pediatric Disease Product; ZANVASTRO (Zilganersen)", "date": "2026-10-09" }, { "title": "Issuance of Priority Review Voucher; Rare Pediatric Disease Product; FAYUVI (rebisufligene etisparvovec-hopf)", "date": "2026-10-06" }, { "title": "Fee Rate for Using a Priority Review Voucher in Fiscal Year 2027", "date": "2026-09-10" }, { "title": "Issuance of Priority Review Voucher; Rare Pediatric Disease Product; GENGLYCOS (parisglasgene brecaparvovec-opnr)", "date": "2026-09-02" }, { "title": "Fee Rate for Using a Priority Review Voucher in Fiscal Year 2026; Correction", "date": "2026-07-28" } ] }, "edgar": { "total": 631, "filers": [ { "name": "Cadrenal Therapeutics, Inc. ", "form": "EX-99.1", "date": "2026-06-18" }, { "name": "Quoin Pharmaceuticals, Ltd. ", "form": "EX-99.1", "date": "2025-08-07" }, { "name": "Quoin Pharmaceuticals Ltd. ", "form": "EX-99.1", "date": "2026-09-10" }, { "name": "Longeveron Inc. ", "form": "EX-99.1", "date": "2026-05-08" }, { "name": "Jasper Therapeutics, Inc. ", "form": "EX-99.1", "date": "2026-07-17" }, { "name": "OS Therapies Inc ", "form": "EX-99.1", "date": "2026-04-30" }, { "name": "Monopar Therapeutics ", "form": "EX-99.1", "date": "2026-06-30" }, { "name": "ATOSSA THERAPEUTICS, INC. ", "form": "EX-99.1", "date": "2026-10-09" }, { "name": "CUMBERLAND PHARMACEUTICALS INC ", "form": "EX-99.1", "date": "2025-03-19" }, { "name": "Cartesian Therapeutics, Inc. ", "form": "EX-99.2", "date": "2025-01-13" }, { "name": "Camp4 Therapeutics Corp ", "form": "EX-99.2", "date": "2025-01-07" }, { "name": "Inmune Bio, Inc. ", "form": "EX-99.1", "date": "2025-02-10" }, { "name": "PRECISION BIOSCIENCES INC ", "form": "EX-99.1", "date": "2025-08-07" }, { "name": "Cabaletta Bio, Inc. ", "form": "8-K", "date": "2026-06-03" } ] }, "as_of": "2026-10-12 15:54" } }