This reversible inhibitor of 4-hydroxyphenylpyruvate
dioxygenase was discovered by Swedish Orphan and is comarketed
by Apoteket AB and Rare Disease Therapeutics. It
is used as an adjunct to dietary restriction of tyrosine and
phenylalanine in the treatment of hereditary tyrosinemia type
1 (HT-1) disease.
Treatment of hereditary tyrosinemia.
Nitisinone is a herbicidal triketone that inhibits 4-hydroxyphenylpyruvate dioxygenase (HPPD), an enzyme involved in plastoquinone biosynthesis in plants and in tyrosine catabolism in mammals. It is used in treatment of inherited tyrosinemia type I.
. Orfadin (Swedish Orphan).
Nitisinone (17) was synthesized in
one step by reacting 2-nitro-4-trifluoromethylbenzoyl
chloride (153) with cyclohexane-1, 3-dione (154) in the
presence of TEA and trimethylsilylcyanide or 2-cyano-2-
(trimethylsilyloxy)propane.
