Ipsen gets Bylvay FDA approval for cholestatic pruritus in Alagille syndrome

The US Food and Drug Administration (FDA) has granted approval to French biopharmaceutical company Ipsen for Bylvay (odevixibat) for the treatment of cholestatic pruritus in Alagille syndrome (ALGS) patients aged 12 months and older.

This once-daily, non-systemic ileal bile acid transport inhibitor (IBATi) primarily acts in the small intestine and has minimal systemic exposure. Already approved for treating progressive familial intrahepatic cholestasis (PFIC) in the US and Europe, Bylvay is now accessible via prescription for eligible Alagille syndrome patients.

Howard Mayer — Ipsen Executive Vice President and Head of Research and Development, commenting on Bylvay FDA approval, said: “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 U.S. and we are committed to making it available to many more eligible patients across the world.”

Ipsen bags Bylvay FDA approval for the treatment of cholestatic pruritus in Alagille syndrome. Photo courtesy of Ipsen Pharma.

Bylvay FDA approval follows the ASSERT phase 3 clinical trial’s positive results, presented at the 2022 American Association for Study of Liver Diseases Congress. The study found Bylvay led to significant and clinically meaningful sustained improvements in pruritus, with over 90% of patients responding positively. The incidence of treatment-emergent adverse events was comparable to the placebo group, and no patients discontinued the study.

Moreover, Ipsen has submitted Bylvay to the European Medicines Agency (EMA) for approval for ALGS treatment, with the Committee for Medicinal Products for Human Use’s opinion expected in Q2 2023, and the EMA’s final decision likely in the second half of 2023.

The drug has already received orphan exclusivity for progressive familial intrahepatic cholestasis treatment and orphan drug designations for Alagille syndrome and biliary atresia treatment in the US and Europe. It has been approved in the US for pruritus treatment in all types of PFIC patients aged three months and older, and in Europe for progressive familial intrahepatic cholestasis treatment in patients aged six months or older.

Additionally, Bylvay is in the late-stage of development for a third indication, biliary atresia, with the phase 3 BOLD trial underway.