Correction

Ipsen provides update on E.U. Marketing Authorization Application for Bylvay for cholestatic pruritus in patients with Alagille syndrome

• Positive opinion from Committee for Medicinal Products for Human Use (CHMP) based on Bylvay Phase III ASSERT clinical-trial data in Alagille syndrome (ALGS)
• Negative opinion from Committee for Orphan Medicinal Products (COMP) recommending not to maintain orphan drug designation for treatment of ALGS
• Ipsen to appeal negative COMP opinion, which might delay final European Commission decision
• Approval already granted by U.S. FDA in June 2023 for the treatment of cholestatic pruritus in patients with ALGS aged 12 months and older

PARIS, FRANCE, 21 July 2023 – Ipsen (Euronext: IPN: ADR: IPSEY) today announced that the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion recommending the approval of Bylvay (odevixibat) for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) aged six months or older.

“We are pleased with today’s CHMP positive opinion which is supported by the data from our Phase III ASSERT trial,” said Howard Mayer, Executive Vice President and Head of Research and Development for Ipsen. “These children endure a very poor quality of life. The severe itching, known as pruritus, caused by the back-up of bile acids in the liver and bloodstream of individuals living with Alagille syndrome, sometimes results in scratching so hard it leads to broken skin. We are committed to bringing a much-needed additional drug treatment option to Alagille patients and families in the E.U.”

The Committee for Orphan Medicinal Products (COMP), a scientific committee of the EMA, has concurrently issued a negative opinion for the maintenance of Bylvay’s orphan drug designation in ALGS.
This negative COMP opinion prevents the retention of orphan-drug status in Bylvay’s marketing authorization in ALGS and might delay a final European Commission decision. Ipsen plans to submit an appeal in respect of the COMP opinion.

Bylvay, is an orphan medicine already approved in the E.U. for the treatment of progressive familial intrahepatic cholestasis (PFIC) in patients aged six months or older. In November 2022, a variation application to the current market authorization was submitted. The application sought approval in the E.U. for a second orphan indication for Bylvay, the treatment of pruritus in patients with ALGS. In 2012, Bylvay received orphan designation for the treatment of ALGS in the E.U., which supported the development of Bylvay through preclinical and clinical stages.