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, Orphan Designations in both the US and EU, accelerated assessment and access to the PRIME scheme in the EU and fast track designation in the US, we’re on track for potential approval, launch and broad global access to odevixibat for PFIC patients in the second half of 2021,” said said Ron Cooper, president and chief executive officer of Albireo.

PFIC is a rare disorder that causes progressive, life-threatening liver disease and is characterised by pruritus (intense itching).

Other symptoms include jaundice, poor weight gain and slowed growth and in many cases, PFIC leads to cirrhosis and liver failure within the first ten years of life.