FDA Approves New Generic for Rare HT-1 Disorder

The FDA has approved the first generic versions of nitisinone capsules (Orfadin, Sobi) for the treatment of adult and pediatric patients with hereditary tyrosinemia type 1 (HT-1) in combination with dietary restriction of tyrosine and phenylalanine. 

Nitisinone is a hydroxy-phenylpyruvate dioxygenase inhibitor originally approved in 2002 for the rare, inherited metabolic disease. The new capsules will be distributed by Endo International and Novitium Pharma’s operating company Par Pharmaceutical. If left untreated, HT-1 can lead to liver and kidney failure, softening and weakening of the bones, as well as neurological issues and an increased risk of liver cancer.

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Prescribing information for the reference product includes warnings of Elevated plasma tyrosine levels, ocular symptoms, developmental delay and hyperkeratotic plaques; leukopenia and severe thrombocytopenia; and a risk of adverse reactions due to glycerol content in the oral suspension.