Albireo Pharma, Inc., a clinical-stage orphan pediatric liver disease company developing novel bile acid modulators, today announced the European Commission has granted orphan designation to lead product candidate A4250, an ileal bile acid transporter (IBAT) inhibitor, for the treatment of biliary atresia, a rare and life-threatening liver disease with no approved pharmacologic treatment option.
A4250 also holds orphan drug designations in both the U.S. and EU for the treatment of progressive familial intrahepatic cholestasis (PFIC), Alagille syndrome and primary biliary cholangitis (PBC).
The European Commission grants orphan designation to medicines intended for the treatment, prevention or diagnosis of a disease that is life-threatening or chronically disabling, and that has a prevalence of not more than 5 in 10,000 in the EU. Orphan designation allows for protocol assistance, and streamlined regulatory review processes and registration in addition to 10 years of market exclusivity upon regulatory approval. Albireo could be eligible for an additional 2 years of market exclusivity following completion of a pediatric investigation plan (PIP).
‚ÄúWe are pleased to receive orphan designation from the European Commission for A4250 for the treatment of biliary atresia,‚Äù said Ron Cooper, President and Chief Executive Officer of Albireo. ‚ÄúThis latest regulatory milestone builds on our recent fast track designation in PFIC and orphan drug designation in Alagille syndrome in the U.S., and underscores the widespread need for new therapies to treat rare cholestatic liver diseases and the potential of A4250. We plan to expand the development of A4250 in rare cholestatic liver diseases in 2019.‚Äù
Biliary atresia is a rare disease of the liver and bile ducts with symptoms developing about 2-8 weeks after birth. In biliary atresia, damaged or absent bile ducts result in bile and bile acids being trapped inside the liver, quickly resulting in damage and scarring of the liver cells (cirrhosis), and even liver failure. About 80 percent of patients with biliary atresia (with or without Kasai hepatoportoenterostomy) ultimately need a liver transplant within the first two decades after birth. Biliary atresia is estimated to affect between 4.5 and 8.5 in every 100,000 children born worldwide.