Albireo Pharma has requested approval from the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for a second indication for odevixibat (Bylvay®)—treating patients with Alagille syndrome. The company said it expects approvals in the second half of 2023.
Odevixibat is a once-daily, nonsystemic ileal bile acid transport inhibitor. It acts locally in the small intestine and was designed to have minimal systemic exposure. It is approved in the United States for treating pruritus in patients with progressive familial intrahepatic cholestasis (PFIC) aged 3 years and above, as well as in Europe for the treatment of PFIC in patients aged 6 months and above.
Read more about Alagille syndrome etiology
Albireo Pharma recently concluded a phase 3, prospective, double-blind, randomized, placebo-controlled clinical trial across 32 sites around the globe to investigate the safety and efficacy of 120 µg/kg/day of odevixibat for the purpose of relieving pruritus in patients with Alagille syndrome. The trial recruited patients aged 17 years and under with a genetically confirmed diagnosis of Alagille syndrome.
The results of the study indicated that the use of the drug significantly relieved pruritus based on the PRUCISION Observer-Reported Outcome scratching score. Researchers also reported that the drug significantly reduced serum bile acid concentrations among participants in the study for an average of 20 to 24 weeks.
Participants also reported significant improvements in various sleep parameters, some noted as early as weeks 1 to 4. The overall incidence of treatment-emergent adverse events was similar to placebo; in addition, no patients discontinued this study.
“We are getting one step closer to expanding access to Bylvay beyond PFIC patients to the Alagille patient community and delivering on our mission of bringing hope to families with cholestatic liver diseases,” Ron Cooper, president and chief executive officer of Albireo Pharma, said.
Albireo submits Bylvay® Alagille syndrome regulatory filings to FDA and EMA. News release. Albireo Pharma, Inc; December 19, 2022.