• 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

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.

“Physicians urgently need more options to treat patients with Alagille syndrome and this approval from the U.S. FDA spotlights the robustness of the Phase III ASSERT clinical study results,” said Nadia Ovchinsky, MD, Chief of the Division of Gastroenterology and Hepatology, Hassenfeld Children's Hospital at NYU Langone 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.”

Roberta Smith, President, Alagille Syndrome Alliance said: “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. 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.”

Ipsen has also submitted Bylvay to the European Medicines Agency (EMA), seeking authorization for ALGS, with Committee for Medicinal Products for Human Use opinion expected in Q2 2023 and final EMA regulatory decision anticipated in second half of 2023. Bylvay has received orphan exclusivity for the treatment of PFIC, and Orphan Drug Designations for the treatment of ALGS and biliary atresia, in the U.S. and Europe. Bylvay is already approved in the U.S. for the treatment of pruritus in patients aged three months and older with all types of PFIC, and in Europe for the treatment of all types of PFIC in patients aged six months or older. In a third indication, the rare pediatric cholestatic liver disease, biliary atresia, Bylvay is in late-stage development with the Phase III BOLD trial.