Nitisinone
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Nitisinone
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| Systematic (IUPAC) name | |
| 2-[2-nitro-4-(trifluoromethyl)benzoyl] cyclohexane-1,3-dione |
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| Identifiers | |
| CAS number | |
| ATC code | A16 |
| PubChem | |
| DrugBank | |
| Chemical data | |
| Formula | C14H10F3NO5 |
| Mol. mass | 329.228 g/mol |
| Pharmacokinetic data | |
| Bioavailability | ? |
| Metabolism | ? |
| Half life | Approximately 54 hours |
| Excretion | ? |
| Therapeutic considerations | |
| Pregnancy cat. |
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| Legal status | |
| Routes | Oral |
Nitisinone (also known as NTBC, an abbreviation of its full chemical name) is a drug originally developed as an herbicide now used in the treatment of hereditary tyrosinemia type 1. Since its first use for this indication in 1991 it has replaced liver transplantation as the first-line treatment for this rare condition.[1] It is also being studied in the less severe but related condition alkaptonuria.[citation needed] It is marketed under the brand name Orfadin.
The mechanism of action of nitrisinone involves reversibile inhibition of 4-hydroxyphenylpyruvate oxidase,[2] thus preventing the formation of maleylacetoacetic acid and fumarylacetoacetic acid, which have the potential to be converted to succinyl acetone, a toxin that damages the liver and kidneys.[1]
[edit] References
- ^ a b McKiernan PJ (2006). "Nitisinone in the treatment of hereditary tyrosinaemia type 1". Drugs 66 (6): 743-50. PMID 16706549.
- ^ Kavana M, Moran GR (2003). "Interaction of (4-hydroxyphenyl)pyruvate dioxygenase with the specific inhibitor 2-[2-nitro-4-(trifluoromethyl)benzoyl]-1,3-cyclohexanedione". Biochemistry 42 (34): 10238-45. doi:. PMID 12939152.
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