Orfadin(nitisinone)
Nitisinone, Nityr, Orfadin (nitisinone) is a small molecule pharmaceutical. Nitisinone was first approved as Orfadin on 2002-01-18. It is used to treat tyrosinemias in the USA. It has been approved in Europe to treat tyrosinemias. The pharmaceutical is active against 4-hydroxyphenylpyruvate dioxygenase.
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Commercial
Therapeutic Areas
Trade Name
FDA
EMA
Nitisinone, Nityr, Orfadin (discontinued: Nitisinone)
Drug Products
FDA
EMA
New Drug Application (NDA)
New Drug Application (NDA)
Abbreviated New Drug Application (ANDA)
Abbreviated New Drug Application (ANDA)
Nitisinone
Tradename | Company | Number | Date | Products |
|---|---|---|---|---|
| NITYR | Cycle Pharmaceuticals | N-209449 RX | 2017-07-26 | 3 products, RLD, RS |
| ORFADIN | Orphan Medical | N-021232 RX | 2002-01-18 | 4 products, RLD |
| ORFADIN | Orphan Medical | N-206356 RX | 2016-04-22 | 1 products, RLD, RS |
Labels
FDA
EMA
Brand Name | Status | Last Update |
|---|---|---|
| nitisinone | ANDA | 2025-03-31 |
| nityr | New Drug Application | 2024-05-24 |
| orfadin | New Drug Application | 2022-06-20 |
Indications
FDA
EMA
Indication | Ontology | MeSH | ICD-10 |
|---|---|---|---|
| tyrosinemias | — | D020176 | E70.21 |
Agency Specific
FDA
EMA
No data
HCPCS
No data
Clinical
Clinical Trials
19 clinical trials
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Indications Phases 4
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Indications Phases 3
Indication | MeSH | Ontology | ICD-10 | Ph 1 | Ph 2 | Ph 3 | Ph 4 | Other | Total |
|---|---|---|---|---|---|---|---|---|---|
| Tyrosinemias | D020176 | — | E70.21 | 4 | 1 | 1 | — | 4 | 10 |
| Alkaptonuria | D000474 | — | E70.29 | — | 3 | 2 | — | — | 4 |
| Ochronosis | D009794 | — | E70.29 | — | 3 | 2 | — | — | 4 |
Indications Phases 2
Indication | MeSH | Ontology | ICD-10 | Ph 1 | Ph 2 | Ph 3 | Ph 4 | Other | Total |
|---|---|---|---|---|---|---|---|---|---|
| Oculocutaneous albinism | D016115 | — | E70.32 | 1 | 1 | — | — | — | 1 |
| Low vision | D015354 | — | — | 1 | 1 | — | — | — | 1 |
| Albinism | D000417 | — | E70.3 | 1 | 1 | — | — | — | 1 |
Indications Phases 1
Indication | MeSH | Ontology | ICD-10 | Ph 1 | Ph 2 | Ph 3 | Ph 4 | Other | Total |
|---|---|---|---|---|---|---|---|---|---|
| Healthy volunteers/patients | — | — | — | 2 | — | — | — | — | 2 |
| Drug interactions | D004347 | — | — | 1 | — | — | — | — | 1 |
Indications Without Phase
Indication | MeSH | Ontology | ICD-10 | Ph 1 | Ph 2 | Ph 3 | Ph 4 | Other | Total |
|---|---|---|---|---|---|---|---|---|---|
| Acidosis | D000138 | EFO_1000014 | E87.2 | — | — | — | — | 1 | 1 |
| Chronic disease | D002908 | — | — | — | — | — | — | 1 | 1 |
| Lactic acidosis | D000140 | EFO_1000036 | E87.20 | — | — | — | — | 1 | 1 |
Epidemiology
Epidemiological information for investigational and approved indications
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Drug
General
| Drug common name | Nitisinone |
| INN | nitisinone |
| Description | Nitisinone is a cyclohexanone that is cyclohexane-1,3-dione substituted at position 2 by a 2-nitro-4-(trifluoromethyl)benzoyl group. It is used in the treatment of hereditary tyrosinemia type 1. It has a role as an EC 1.13.11.27 (4-hydroxyphenylpyruvate dioxygenase) inhibitor. It is a member of cyclohexanones, a C-nitro compound, a member of (trifluoromethyl)benzenes and a mesotrione. |
| Classification | Small molecule |
| Drug class | — |
| Image (chem structure or protein) |  |
| Structure (InChI/SMILES or Protein Sequence) | O=C1CCCC(=O)C1C(=O)c1ccc(C(F)(F)F)cc1[N+](=O)[O-] |
Identifiers
| PDB | — |
| CAS-ID | 104206-65-7 |
| RxCUI | — |
| ChEMBL ID | CHEMBL1337 |
| ChEBI ID | 50378 |
| PubChem CID | 115355 |
| DrugBank | DB00348 |
| UNII ID | K5BN214699 (ChemIDplus, GSRS) |
Target
Agency Approved
Alternate
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Variants
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Trends
PubMed Central
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Safety
Black-box Warning
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Adverse Events
Top Adverse Reactions
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1,042 adverse events reported
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