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NITISINONE
SynDRA canonical compound SYN0005391 — structure-anchored drug synonym record
| Clinical phase | Launched |
|---|
| Approval status | Approved · Rx/OTC |
|---|
| Primary target(s) | HPD |
|---|
| Mechanism of action | Hydroxyphenylpyruvate dioxygenase inhibitor |
|---|
| Indication | tyrosinemia |
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| Disease area | metabolism |
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All known targets
Mechanisms of action (all sources)
- Hydroxyphenylpyruvate dioxygenase inhibitor
- HPD INHIBITOR
- 4-hydroxyphenylpyruvate dioxygenase inhibitor
Drug classes
- Enzyme Inhibitors
- Hydroxyphenylpyruvate Dioxygenase Inhibitors
- 4-Hydroxyphenyl-Pyruvate Dioxygenase Inhibitor
- Cytochrome P450 2C9 Inhibitors
- Organic Anion Transporter 1 Inhibitors
- Organic Anion Transporter 3 Inhibitors
- Cytochrome P450 2E1 Inducers
ATC classes
- A16A · OTHER ALIMENTARY TRACT AND METABOLISM PRODUCTS
Cross-references
Synonyms
- nitisone
- orfadin
- NTBC
- Nitisinona
- SC-0735
- Nitisinone mdk (previously nitisinone mendelikabs)
- Nityr
- Orfadin
- Nitisinonum
- Nitisone
- Nitisinone [INN]
- SC 0735
- FE-0200
- Nitisinone (NTBC)
- Nitisinone [USAN:INN]
- Orfadin (TN)
- Nitisinone (JAN/USAN/INN)
- 2-(2-Nitro-4-trifluoromethylbenzoyl)cyclohexane-1,3-dione
- 2-(2-nitro-4-trifluoromethylbenzoyl)-1,3-cyclohexanedione
- 2-(Alpha,alpha,alpha-trifluoro-2-nitro-p-tuluoyl)-1,3-cyclohexanedione
- 2-[2-nitro-4-(trifluoromethyl)benzoyl]cyclohexane-1,3-dione
- 2-{[2-nitro-4-(trifluoromethyl)phenyl]carbonyl}cyclohexane-1,3-dione
- 2-(2-nitro-4-(trifluoromethyl)benzoyl)cyclohexane-1,3-dione
- 1,3-Cyclohexanedione, 2-[2-nitro-4-(trifluoromethyl)benzoyl]-
- CHEMBL1337
- CHEBI:50378
- 1,3-Cyclohexanedione, 2-(2-nitro-4-(trifluoromethyl)benzoyl)-
- MFCD01752192
- SC0735
- K5BN214699
- nitisinona
- nitisinonum
- SMR002529592
- UNII-K5BN214699
- NITYR
- 2-[2-Nitro-4-(trifluoromethyl)benzoyl]-1,3-cyclohexanedione
- NITISINONE [MI]
- NITISINONE [JAN]
Reported adverse events
- Conjunctivitis
- Thrombocytopenia
- Blepharitis
- Pruritus
- Eye pain
- Leukopenia
- Granulocytopenia
- Photophobia
- Alopecia
- Cataract
- Corneal opacity
- Epistaxis
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