Nitisinone(SC0735) is an inhibitor of the enzyme 4-hydroxyphenylpyruvate dioxygenase.Target: 4-Hydroxyphenylpyruvate DioxygenaseNitisinone is a drug used to slow the effects of hereditary tyrosinemia type 1. Nitisinone reduced urinary HGA levels from an average of 4.0 +/- 1.8 (SD) g/day to 0.2 +/- 0.2 g/day ( P < .001). Low-dose nitisinone effectively reduced urinary HGA levels in patients with alkaptonuria. Future long-term clinical trials are planned to determine the benefits of nitisinone in preventing joint deterioration and providing pain relief, and its long-term side effects [1]. Nitisinone can prevent the development of liver disease and significantly reduce the risk of developing hepatocellular carcinoma; however, vigorous surveillance for the development of HCC needs to be continued lifelong [2].
References:
[1]. Suwannarat, P., et al., Use of nitisinone in patients with alkaptonuria. Metabolism, 2005. 54(6): p. 719-28.
[2]. Santra, S. and U. Baumann, Experience of nitisinone for the pharmacological treatment of hereditary tyrosinaemia type 1. Expert Opin Pharmacother, 2008. 9(7): p. 1229-36.