Nitisinone

Nitisinone

Nitisinone is a hydroxyphenylpyruvate dioxygenase inhibitor used as an adjunct to dietary restrictions for the treatment of hereditary tyrosinemia type 1 (HT-1), which causes intolerance to tyrosine containing foods.

Systematic name

2-[2-nitro-4-(trifluoromethyl)benzoyl]cyclohexane-1,3-dione

Brand name(s)

Nityr, Orfadin

Therapeutic category

Hereditary tyrosinemia

Available formulations

Solid

Approvals

USDMF Exclusive for US market

EU-WC

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