Albireo Pharma submits regulatory filings to FDA, EMA for Bylvay in ALGS

Albireo Pharma announced the submission of a supplemental New Drug Application to the U.S. Food and Drug Administration and a variation application to the European Medicines Agency seeking approval for a second Bylvay indication for use in patients with Alagille syndrome in the second half of 2023. A potent, once-daily, non-systemic ileal bile acid transport inhibitor, Bylvay has minimal systemic exposure and acts locally in the small intestine. Bylvay is already approved in the U.S. for the treatment of pruritus in patients 3 months of age and older in all types of progressive familial intrahepatic cholestasis, and in Europe for the treatment of all types of PFIC in patients aged 6 months or older. Positive data from the Phase 3 ASSERT study recently presented at the 2022 AASLD The Liver Meeting demonstrated that Bylvay provided early, rapid, clinically meaningful and sustained improvements in pruritus, as well as significant reductions in bile acids and improvements in sleep quality across the two most prominent genetic types in Alagille syndrome, JAG1 and NOTCH2, and in a wider age range of ALGS patients. Over 90% of patients were pruritus responders and Bylvay was generally well tolerated; the overall incidence of treatment emergent adverse events was similar to placebo. No patients discontinued the study and 96% of patients rolled over into the open-label extension study. In the U.S., Bylvay received orphan exclusivity for the treatment of pruritus in PFIC and Orphan Drug Designations for the treatment of ALGS, biliary atresia and primary biliary cholangitis. In Europe, Bylvay received orphan exclusivity for the treatment of PFIC and Orphan Drug Designations for the treatment of ALGS, biliary atresia and primary biliary cholangitis. With completed submissions for Bylvay in the U.S. and EU for use in patients with ALGS, the Company anticipates approvals in the second half of 2023. Bylvay is also being evaluated in the Phase 3 BOLD study in biliary atresia, a global gold standard pivotal trial that enrolled 205 patients. Topline results are expected to be available by the end of 2024.