CAMBRIDGE, Mass. — Sarepta Therapeutics has acknowledged a negative opinion from the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) regarding conditional marketing authorization for its gene therapy ELEVIDYS, intended for treating ambulatory children aged three to seven with Duchenne muscular dystrophy (DMD).
Despite the setback, Sarepta and its global partner Roche say they remain committed to pursuing regulatory pathways in Europe to make the therapy available to patients. Roche will continue discussions with the EMA to explore potential next steps.
“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, Ph.D., Sarepta’s president of R&D and technical operations.
ELEVIDYS, the first and only gene therapy designed to modify the progression of Duchenne, received initial approval from the U.S. Food and Drug Administration in June 2023 and has since gained regulatory clearance in several other countries. Sarepta is currently working with the FDA to address recent safety concerns raised about the treatment.
Although the primary endpoint of the EMBARK study was not met after one year, the therapy showed statistically significant improvements on key secondary functional outcomes. Sarepta submitted additional long-term efficacy data to the EMA, including two-year results from EMBARK and a three-year pooled analysis from other ELEVIDYS trials, which indicated durable improvements in motor function.
Notable data from the EMBARK study have been published in peer-reviewed journals, including Nature Medicine in October 2024 and JAMA Neurology in May 2025. Year-two results were also presented at the 2025 Muscular Dystrophy Association Clinical & Scientific Conference.
Sarepta leads regulatory and commercial efforts for ELEVIDYS in the United States and oversees manufacturing. Roche is responsible for the therapy’s approval and distribution outside the U.S., while Chugai Pharmaceuticals handles commercialization in Japan through its partnership with Roche.
