FDA award orphan status to A 4250 (Albireo) for Primary Biliary Cirrhosis and PFIC

The FDA has awarded A 4250, from Albireo, orphan-drug designation for the treatment of Progressive Familial Intrahepatic Cholestasis (PFIC)and Primary Biliary Cirrhosis (PBC). Orphan designation was granted based on an extensive preclinical data package showing that A4250 has a high potency and a minimal systemic exposure. In addition, substantial benefits were shown in an animal model of cholestasis; the predominant feature of PFIC and PBC. The EMA awarded the drug orphan status in July 2012.