Albireo Pharma has submitted odevixibat approval for the treatment of patients with progressive familial intrahepatic cholestasis (PFIC) to the US Food and Drug Administration (FDA) and European Medicines Agency (EMA)
The US-based biopharmaceutical company has previously received means , rare paediatric disease and orphan drug designations for odevixibat within the US for PFIC.
The drug also has orphan drug designations for the treatment for Alagille Syndrome, biliary atresia and first biliary cholangitis.
Odevixibat may be a one-daily non-systemic ileal steroid transport inhibitor (IBATi) currently being developed for the treatment of variety of rare paediatric liver diseases, like PFIC, biliary atresia and Alagille syndrome.
In a phase III clinical trial trial evaluating the efficacy and safety of odevixibat in PFIC, the drug met two primary endpoints, demonstrating the power to scale back serum steroid responses and improve pruritus assessments with one 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 within the EU and means designation within the US, we’re on target for potential approval, launch and broad global access to odevixibat for PFIC patients within the last half of 2021,” said said Ron Cooper, president and chief military officer of Albireo.
PFIC may be a rare disorder that causes progressive, life-threatening disease and is characterised by pruritus (intense itching).
Other symptoms include jaundice, poor weight gain and slowed growth and in many cases, PFIC results in cirrhosis and liver failure within the primary ten years of life.