U.S. FDA approves Bylvay for patients living with cholestatic pruritus due to Alagille syndrome

• Approval heralds the second rare cholestatic liver disease indication for Bylvay in the U.S. after progressive familial intrahepatic cholestasis related pruritus in 2021
• Immediate U.S. commercial launch and availability for eligible patients
• ASSERT clinical study demonstrated efficacy of Bylvay in improvement of pruritus. Also showed improvement in certain sleep disturbances and reduction in bile acids – which were secondary endpoints – with a low drug-related diarrhea rate in patients with Alagille syndrome
• Committee for Medicinal Products for Human Use opinion expected in Q2 2023 with final European Medicines Agency decision in second half of 2023

PARIS, FRANCE, 13 June 2023 – Ipsen (Euronext: IPN: ADR: IPSEY) today announced that the U.S. Food and Drug Administration (FDA) has approved Bylvay (odevixibat) for the treatment of cholestatic pruritus in patients from 12 months of age with Alagille syndrome (ALGS). Bylvay is a once-daily, non-systemic ileal bile acid transport inhibitor (IBATi) that acts locally in the small intestine and has minimal systemic exposure. Bylvay was approved as the first drug treatment option for patients living with cholestatic pruritus due to progressive familial intrahepatic cholestasis (PFIC) in the U.S., and for the treatment of PFIC in Europe, in 2021. Bylvay is immediately available via prescription for eligible ALGS patients.

“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. “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.”

Positive data from the Phase III ASSERT study, presented at the 2022 American Association for Study of Liver Diseases congress, demonstrated that Bylvay provided highly statistically significant and clinically meaningful sustained improvements in pruritus, starting early after initiation of treatment. More than 90% of patients were pruritus responders (≥ 1 point change at any time during 24 weeks). The overall incidence of treatment-emergent adverse events was similar to placebo. No patients discontinued the study and 96% of patients rolled over into the open-label extension study.