Rare Daily Staff

The U.S. Food and Drug Administration has granted approval to Endo International’s partner Novitium Pharma for a room temperature stable, AB-rated, generic equivalent of Swedish Orphan Biovitrum’s Orfadin (nitisinone). 

Endo’s operating company Par Pharmaceutical expects to distribute the product through specialty pharmacies beginning this month.

Nitisinone capsules are indicated for the treatment of adult and pediatric patients with hereditary tyrosinemia type 1 (HT-1) in combination with dietary restriction of tyrosine and phenylalanine.

Hereditary tyrosinemia type-1 (HT-1) is a rare and serious inherited metabolic disease caused by the inability to metabolize the amino acid tyrosine. Left untreated, HT-1 can cause hepatic, renal and peripheral nerve damage. HT-1 affects at least 1 in 100,000 patients worldwide, with approximately 150 patients in the United States.

“We are pleased to offer this rare patient population suffering from HT-1 the first and only bioequivalent and therapeutically equivalent option to Orfadin capsules,” said Domenic Ciarico, executive vice president and chief commercial officer, sterile and generics for Endo. “Additionally, the product can be stored at room temperature which is an added convenience to patients.”

According to Swedish Orphan Biovitrum, global sales for Orfadin were approximately $85 million over the last four quarters.


Photo: Domenic Ciarico, executive vice president and chief commercial officer, sterile and generics for Endo

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