Nitisinone is a hydroxyphenylpyruvate dioxygenase inhibitor used as an adjunct to dietary restrictions for the treatment of hereditary tyrosinemia type 1 (HT-1) and Alkaptonuria, which cause intolerance to tyrosine containing foods. It is sold under the brand names Orfadin and Harliku.
- Mechanism
- Curator reviewed
Nitisinone is a competitive inhibitor of 4-hydroxyphenyl-pyruvate dioxygenase, an enzyme upstream of fumarylacetoacetate hydrolyase (FAH) in the tyrosine catabolic pathway. By inhibiting the normal catabolism of tyrosine in patients with hereditary tyrosinemia type 1 (HT-1), nitisinone prevents the accumulation of the catabolic intermediates maleylacetoacetate and fumarylacetoacetate.
- Primary indication
- Nitisinone under the brand name ORFADIN is indicated as an adjunct to dietary restriction of tyrosine and phenylalanine in the treatment of hereditary tyrosinemia type 1.Curator reviewed · 2 structured indications
- Formula / weight
- C14H10F3NO5 · 329.2281 g/mol (avg)
- First approval
- Canada, 2016 · United States, 2002 · European Union, 2021
- Code names
- SC-0735
- Brand names
Resolves to
OUBCNLGXQFSTLU-UHFFFAOYSA-NSMILES[O-][N+](=O)C1=C(C=CC(=C1)C(F)(F)F)C(=O)C1C(=O)CCCC1=OWhat you can answer from here — as of September 20, 2026
Which drugs share a target with Nitisinone, and which of those have an active Phase 3 trial?
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