Bylvay Alagille Syndrome Submitted for Regulatory Filings to FDA, EMA

Albireo Pharma, Inc. announced on December 19, 2022 the submission of a supplemental New Drug Application (sNDA) to FDA and a variation application to the European Medicines Agency (EMA) for approval for a second Bylvay (odevixibat) indication for use in patients with Alagille syndrome (ALGS).

“The clear results from our ASSERT Phase III data allowed the Company to swiftly submit FDA and EMA regulatory filings,” said Ron Cooper, president and chief executive officer of Albireo, in a press release. “We are getting one step closer to expanding access to Bylvay beyond PFIC [progressive familial intrahepatic cholestasis] patients to the Alagille patient community and delivering on our mission of bringing hope to families with cholestatic liver diseases.”

Bylvay has minimal systemic exposure and acts locally in the small intestine as a potent, once-daily, non-systemic ileal bile acid transport inhibitor (IBATi). The drug is already approved in the United States for the treatment of pruritis in patients three months of age and older in all types of PFIC and in Europe for the treatment of all types of PFIC in patients six months of age and older.

New positive data from the Phase III ASSERT study showed that Bylvay provided early, rapid, clinically meaningful and sustained improvements in pruritus and significant reductions in bile acids and improvements in sleep quality across the two most prominent genetic types in Alagille syndrome: JAG1 and NOTCH2. The study showed that more than 90% of patients were pruritus responders and Bylvay was well tolerated. Further, the overall incidence of treatment emergent adverse events was like the placebo. There were no patients who discontinued the study, and 96% transitioned over to the open-label extension study.

Albireo Pharma, Inc. hopes to approve Bylvay in the second half of 2023, and it is being evaluated in the Phase III BOLD study in biliary atresia.

Source: Albireo