Odevixibat (Bylvay®) is a step closer to becoming an option for treating patients with Alagille syndrome (ALGS), after the US Food and Drug Administration (FDA) accepted Albireo Pharma’s supplemental New Drug Application and issued a Prescription Drug User Fee Act action date of June 15, 2023.

“With the phase 3 randomized, placebo-controlled ASSERT data and Orphan Designation, we are planning for approval in ALGS in mid-2023, expanding access to Bylvay for more of the cholestatic liver community,” said Ron Cooper, president and chief executive officer of Albireo.

The results from the ASSERT phase 3 study showed that odevixibat provided early, rapid, clinically meaningful, and sustained improvements in pruritus. Moreover, it led to significant reductions in bile acids and improved patients’ sleep quality.

Continue Reading

The treatment was generally well tolerated, with the overall incidence of treatment-emergent adverse events being similar to that observed with placebo.

Read more about ALGS treatment

“It is great to know physicians may have another drug treatment option for the debilitating pruritus that affects so many Alagille patients,” said Roberta Smith, president of the Alagille Syndrome Alliance. “I personally know the devastation of pruritus and the terrible impact to a child’s quality of life and ability to sleep and thrive; this FDA acceptance and priority review paves the way to alleviating that burden for more patients.”

Albireo Pharma has requested approval for the use of odevixibat in patients with ALGS from both the FDA and the European Medicines Agency.


FDA grants June 15, 2023 PDUFA date to Albireo for Bylvay® in Alagille syndrome. News release. Albireo Pharma, Inc; February 14, 2023.