Albireo submits rare liver disease med odevixibat to FDA and EMA

Albireo Pharma has submitted odevixibat to the US Food and Drug Administration (FDA) and European Medicines Agency (EMA), seeking approval for the treatment of patients with progressive familial intrahepatic cholestasis (PFIC). The US-based biopharmaceutical company has previously received fast track, rare paediatric disease and orphan drug designations for odevixibat in the US for PFIC. The drug also has orphan drug designations for the treatment for Alagille Syndrome, biliary atresia and primary biliary cholangitis. Odevixibat is a one-daily non-systemic ileal bile acid transport inhibitor (IBATi) currently being developed for the treatment of a number of rare paediatric liver diseases, such as PFIC, biliary atresia and Alagille syndrome. In a phase III trial evaluating the efficacy and safety of odevixibat in PFIC, the drug met two primary endpoints, demonstrating the ability to reduce serum bile acid responses and improve pruritus assessments with a single digit diarrhoea rate. “With randomised, placebo-controlled PEDFIC data, ...

• Source: drugdu
• 191
• March 5, 2021