Albireo Pharma, Inc. announced a number of advances and new initiatives in the clinical program for odevixibat, an oral once-daily capsule in development for the treatment of progressive familial intrahepatic cholestasis (PFIC), biliary atresia and Alagille syndrome. The U.S. Food and Drug Administration (FDA) has cleared the company’s investigational new drug (IND) application to initiate a global pivotal trial in biliary atresia. Following scheduled FDA interactions in the first quarter of 2020, the company plans to commence an additional pivotal program in Alagille syndrome by the end of 2020. Management also continues to expect topline data from its Phase 3 trial in PFIC in mid-2020. The PEDFIC 1 trial is studying both high and low dose odevixibat in both PFIC type 1 and type 2 patients who are 6 months to 18 years of age. Patients randomized to odevixibat are being treated with high (120µg/kg) or low (40µg/kg) dose once-daily oral capsules or sprinkles. The trial uses Albireo’s planned commercial formulation of odevixibat, which does not require refrigeration. PEDFIC 1 has a target of 60 patients, 59 have been randomized and the randomization visit for the final patient has been scheduled in the coming days. Albireo expects top-line data from PEDFIC 1 in mid-2020 with potential first regulatory approval and odevixibat launch in the second half of 2021. Biliary atresia is a rare disease that impacts an estimated 15,000-20,000 people in the United States and European Union and is the leading cause of liver transplants among children. Albireo’s planned double-blind, placebo controlled pivotal trial in biliary atresia is designed to enroll approximately 200 patients at 70 sites globally. Patients will receive either placebo or high-dose (120µg/kg) odevixibat once daily. The primary endpoint is survival with native liver after 2 years of treatment. The FDA and European Commission have granted orphan designations for odevixibat in the treatment of biliary atresia. In addition, Albireo has been evaluating a proposed pivotal study design for odevixibat in Alagille syndrome in consultation with physicians and regulators, and plans to initiate the trial later this year. The FDA and European Commission have granted orphan designations for odevixibat in the treatment of Alagille syndrome.