Albireo submits NDA to FDA and MAA to EMA seeking approval of odevixibat for the treatment of patients with progressive familial intrahepatic cholestasis .

Albireo Pharma, Inc. a clinical-stage rare liver disease company developing novel bile acid modulators, announced it has submitted a New Drug Application (NDA) to the FDA and a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) seeking approval of odevixibat for the treatment of patients with progressive familial intrahepatic cholestasis (PFIC). Odevixibat is a potent, once-daily, non-systemic ileal bile acid transport inhibitor (IBATi) being developed to treat rare pediatric cholestatic liver diseases, including PFIC, biliary atresia and Alagille syndrome. The EMA has validated the odevixibat MAA on the accelerated assessment timeline, which begins the formal review process. With FDA and EMA regulatory submissions complete, odevixibat has the potential to become the first approved drug treatment for patients with PFIC in the U.S and Europe. Odevixibat has previously received Fast Track, Rare Pediatric Disease and Orphan Drug Designations in the U.S. In addition to PFIC, odevixibat has Orphan Drug Designations for the treatment of Alagille Syndrome, biliary atresia and primary biliary cholangitis. The EMA has granted odevixibat accelerated assessment, Orphan Designation, as well as access to the Priority MEdicines (PRIME) scheme for the treatment of PFIC. The EMA’s Pediatric Committee has agreed to Albireo’s odevixibat Pediatric Investigation Plans for PFIC and biliary atresia. With U.S. and EU regulatory submissions for odevixibat in PFIC completed, the Company anticipates potential regulatory approvals, issuance of a rare pediatric disease priority review voucher and launch in the second half of 2021.