CHMP Recommends Against Approval of Elevidys Gene Therapy in Europe, Dealing Blow to Sarepta and Roche
On July 25, 2025, the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) issued a negative opinion against approving Elevidys (delandistrogene moxeparvovec) for ambulatory patients aged 3–7 years with Duchenne muscular dystrophy (DMD) in Europe1235.
The CHMP based its recommendation on EMBARK trial results, in which Elevidys failed to show a significant effect on movement abilities after 12 months, despite demonstrating disease stabilization and a manageable safety profile13.
Sarepta Therapeutics has accepted the CHMP decision, while Roche (which owns ex-U.S. rights) announced it will continue working with the EMA to explore paths forward due to the high unmet medical need in DMD134.
The negative opinion causes another major regulatory and commercial setback for Sarepta and Roche, with the European Commission expected to issue a final, legally binding decision within 67 days2.
Analysts described the outcome as unfortunate but unsurprising, especially following recent safety issues and related scrutiny2.
Sources:
1. https://www.pharmaceutical-technology.com/news/chmp-rejects-elevidys-latest-setback-sarepta/
2. https://www.biospace.com/business/sarepta-fallout-continues-with-eus-negative-opinion-of-elevidys-in-ambulatory-patients
3. https://www.roche.com/media/releases/med-cor-2025-07-25
4. https://www.fiercepharma.com/pharma/roche-wont-throw-towel-after-dmd-gene-therapy-elevidys-rebuff-europe
5. https://www.biopharmadive.com/news/sarepta-elevidys-ema-europe-negative-opinion-duchenne/754042/