Nitisinone

Nitisinone is an inhibitor of 4-hydroxyphenylpyruvate dioxygenase (HPPD), which converts 4-hydroxyphenylpyruvate (HPPA) to homogentisate in the tyrosine catabolic pathway. Nitisinone increases urinary levels of HPPA and 4-hydroxyphenyllactate (HPLA) in rats when administered at a dose of 10 mg/kg. Nitisinone (3 mg/kg) prevents the neonatal lethality of fumarylacetoacetate hydrolase (FAH) deficiency in mice when administered to pregnant dams. It exhibits hepatoprotective effects in FAH-/- mice, such as prevention of increases in plasma levels of aspartate serine aminotransferase (AST) and conjugated bilirubin, when administration is continued following birth at a dose of 1 mg/kg. Nitisinone (100 µg) decreases urinary excretion of homogentisate and increases urinary excretion of HPPA, HPLA, and 4-hydroxyphenylacetate in a mouse model of alkaptonuria induced by ethylnitrosourea. Formulations containing nitisinone have been used in the treatment of hereditary tyrosinemia type 1 (HT-1).Formal Name: 2-[2-nitro-4-(trifluoromethyl)benzoyl]-1,3-cyclohexanedione. CAS Number: 104206-65-7. Synonyms: NTBC, SC-0735. Molecular Formula: C14H10F3NO5. Formula Weight: 329.2. Purity: >98%. Formulation: (Request formulation change), A crystalline solid. Solubility: DMF: 30 mg/ml, DMSO: 30 mg/ml, DMSO:PBS(pH7.2) (1:1): 0.5 mg/ml, Ethanol: 1 mg/ml. lambdamax: 216, 271 nm. SMILES: O=C(CCC1)C(C(C2=CC=C(C(F)(F)F)C=C2[N+]([O-])=O)=O)C1=O. InChi Code: InChI=1S/C14H10F3NO5/c15-14(16,17)7-4-5-8(9(6-7)18(22)23)13(21)12-10(19)2-1-3-11(12)20/h4-6,12H,1-3H2. InChi Key: OUBCNLGXQFSTLU-UHFFFAOYSA-N.