Ipsen has received US Food and Drug Administration (FDA) approval for Bylvay (odevixibat) to treat patients aged 12 months and above with cholestatic pruritus caused by Alagille syndrome (ALGS).
Bylvay is a once-a-day, non-systemic ileal bile acid transport inhibitor (IBATi) that acts locally in the small intestine and has minimal systemic exposure.
The latest approval marks the second rare cholestatic liver disease indication for Bylvay in the US, following the approval for progressive familial intrahepatic cholestasis-related pruritus in 2021.
Ipsen executive vice-president and research and development head Howard Mayer stated: “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.
“We are proud to have achieved FDA approval for Bylvay as a treatment for ALGS in the US and we are committed to making it available to many more eligible patients across the world.”
The FDA’s decision was based on the findings obtained from the Phase III ASSERT trial, which enrolled patients aged from early months to 17 years with a genetically confirmed diagnosis of ALGS.
The placebo-controlled, double-blind, randomised trial assessed the efficacy and safety of Bylvay 120µg/kg/day given for 24 weeks to relieve pruritus in patients with ALGS from 32 sites across the Middle East, Europe, North America and the Asia Pacific.
The findings showed that odevixibat provided statistically significant and clinically meaningful sustained improvement in pruritis, beginning soon after treatment commenced.
More than 90% of patients who received treatment with odevixibat were pruritus responders.
The therapy is being offered immediately through prescription for eligible ALGS patients.
Ipsen is also seeking marketing approval from the European Medicines Agency, with a final decision expected in the second half of 2023.
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