CAS Number:
104206-65-7
Category:
4-hydroxyphenylpyruvate dioxygenase inhibitor
Regulatory Status:
DMF Active in US
regulatory_status
CEP/EDMF:
-
Innovator Brand:
NITYR, ORFADIN
Therapeutic Area:
Tyrosinemia
USDMF:
#035810
Others:
-
Mechanism of Action
Nitisinone works by inhibiting the enzyme 4-hydroxyphenylpyruvate dioxygenase, which is involved in the catabolic pathway of the amino acid tyrosine. This prevents the accumulation of toxic metabolites in the blood and tissues.
Uses
Nitisinone is used to treat hereditary tyrosinemia type 1, a rare genetic metabolic disorder.