Nitisinone Systematic (IUPAC) name 2-[2-nitro-4-(trifluoromethyl)benzoyl]
Clinical data AHFS/Drugs.com Licence data EMA: , US FDA: Pregnancy cat. ? Legal status ℞-only (US) Routes Oral Pharmacokinetic data Half-life Approximately 54 h Identifiers CAS number ATC code A16 PubChem DrugBank ChemSpider UNII KEGG ChEBI ChEMBL Chemical data Formula C14H10F3NO5 Mol. mass 329.228 g/mol SMILES & (what is this?)
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. It is also being studied in the less severe but related condition alkaptonuria. It is marketed under the brand name Orfadin.
The mechanism of action of nitrisinone involves reversibile inhibition of 4-hydroxyphenylpyruvate oxidase,  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.
- ^ a b McKiernan PJ (2006). "Nitisinone in the treatment of hereditary tyrosinaemia type 1". Drugs 66 (6): 743–50. doi:10.2165/00003495-200666060-00002. PMID 16706549.
- ^ Phornphutkul C, Introne WJ, Perry MB et al. (2002). "Natural history of alkaptonuria". New England Journal Medicine 347 (26): 2111–21. doi:10.1056/NEJMoa021736. PMID 12501223. http://content.nejm.org/cgi/content/full/347/26/2111.
- ^ Lock EA, Ellis MK, Gaskin P, Robinson M, Auton TR, Provan WM, Smith LL, Prisbylla MP, Mutter LC, Lee DL (1998). "From toxicological problem to therapeutic use: The discovery of the mode of action of 2-(2-nitro-4-trifluoromethylbenzoyl)-1,3-cyclohexanedione (NTBC), its toxicology and development as a drug". Journal of Inherited Metabolic Disease 42 (21): 498–506. doi:10.1023/A:1005458703363. PMID 9728330.
- ^ 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:10.1021/bi034658b. PMID 12939152.
Other alimentary tract and metabolism products (A16) Amino acids and derivatives EnzymesGlycosaminoglycan: iduronidase (Laronidase) • arylsulfatase B (Galsulfase) • iduronate-2-sulfatase (Idursulfase) Various alimentary tract
and metabolism products
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