EMA award orphan status to A 4250 (Albireo) for the treatment of Primary Biliary Cirrhosis

The European Medicines Agency Committee for Orphan Medicinal Products (COMP) has issued a positive opinion on an application for orphan medicinal product status for A 4250, from Albireo, for the treatment of Primary Biliary Cirrhosis (PBC), Progressive Familial Intrahepatic Cholestasis (PFIC) and Alagille Syndrome. A 4250 belongs to a class of inhibitors of the ileal bile acid transporter (IBAT, syn. apical sodium-dependent bile acid transporter ASBT). Usually bile excreted into the small bowel is being reused by a transport mechanism in which bile acids are absorbed in the distal part of the small bowel. A 4250 decreases this re-absorption and will reduce the toxic levels of bile acids in the diseases described above. By using a specialized delivery technology, the bile acids will be neutralized in the large bowel.