The US Food and Drug Administration (FDA) has approved odevixibat (Bylvay®), a once-daily, nonsystemic ileal bile acid transport inhibitor that acts locally in the small intestine and has minimal systemic exposure, for the treatment of cholestatic pruritus in patients aged 12 months or older with Alagille syndrome (ALGS). 

A prescription for odevixibat is now immediately available for eligible patients.

“As an advocate for families impacted by Alagille syndrome, it is such a blessing to know physicians now have another drug treatment option for the debilitating pruritus that affects so many Alagille patients.” said Roberta Smith, president, Alagille Syndrome Alliance.

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“I know personally the terrible impact of this rare disease on a child; this approval will help to alleviate the pruritus burden for more patients.”

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Odevixibat showed sustained improvements in pruritus that were statistically significant and clinically meaningful starting early after initial treatment, as demonstrated by positive data from the phase 3 ASSERT clinical study. In the study, more than 90% of patients were pruritus responders (≥1 point change at any time during 24 weeks) and the overall frequency of treatment-emergent adverse events was similar to placebo. 

“Physicians urgently need more options to treat patients with Alagille syndrome and this approval from the US FDA spotlights the robustness of the phase 3 ASSERT clinical study results,” said Nadia Ovchinsky, MD, chief of the division of gastroenterology and hepatology, Hassenfeld Children’s Hospital at NYU Langone, in New York, and ASSERT principal investigator. “The ASSERT study showed that Bylvay reduced pruritus associated with ALGS, which is so common among this patient population and one of the leading indications for a liver transplant.”

The study also showed improvement in certain sleep disturbances and a reduction in bile acids with a low drug-related diarrhea rate in patients with ALGS. 

“Today’s approval of Bylvay in a second indication allows patients and physicians to access an additional treatment option that has the potential to improve the management of pruritus, or intense itch, in this distressing condition that tends to affect young children,” said Howard Mayer, executive vice president and head of research and development for Ipsen. 

The approval is the second rare cholestatic liver disease indication for odevixibat in the United States after it was approved in 2021 as the first available drug treatment option for patients living with cholestatic pruritus due to progressive familial intrahepatic cholestasis (PFIC). 


U.S. FDA approves Bylvay® for patients living with cholestatic pruritus due to Alagille syndrome. News release. Ipsen; June 13, 2023.