BioCentury
ARTICLE | Clinical News

Oral suspension Orfadin nitisinone regulatory update

September 21, 2015 7:00 AM UTC

FDA accepted for review an NDA from Swedish Orphan for an oral suspension formulation of Orfadin nitisinone to treat hereditary tyrosinaemia type I, a rare genetic disorder that can cause liver failure, kidney dysfunction and neurological problems. The company said it expects a decision from FDA in 2Q16. The 4-hydroxyphenylpyruvate dioxygenase (HPPD) inhibitor is marketed as a capsule formulation. ...