Sarepta receives negative CHMP opinion on Elevidys marketing authorization

Sarepta (SRPT) acknowledged that the Committee for Medicinal Products for Human Use issued a negative opinion on the conditional marketing authorization for Elevidys in ambulatory individuals ages three to seven years for the treatment of Duchenne muscular dystrophy. “While we are disappointed by the CHMP’s negative opinion, we understand the urgent need for continued dialogue and collaboration to bring transformative therapies to people with Duchenne who live with a relentless disease that steals their mobility, independence and ultimately life – often by early adulthood,” said Louise Rodino-Klapac, president of research and development and technical operations, Sarepta. “Following the initial FDA approval of ELEVIDYS on June 22, 2023, the therapy has subsequently received regulatory approval in several other countries. In the U.S., we are actively working with the FDA to address recent safety questions. We remain committed to working with regulators to address outstanding questions on safety so that people living with Duchenne have access to this important therapy.”