Overview
- Roche said it has begun a late-stage study of Elevidys to generate data for a European re-submission and for other regulatory filings.
- The randomized trial will run for more than 72 weeks in about 100 early ambulatory boys with Duchenne muscular dystrophy and will track change in time-to-rise velocity as the main measure.
- The EMA declined to back the therapy last year, saying it had not shown lasting benefit, after regulators examined two deaths tied to treatment.
- Elevidys is cleared in nine countries for ambulatory boys with confirmed DMD mutations, and U.S. authorities briefly paused shipments in July.
- Roche controls rights outside the United States, while developer Sarepta sells the therapy in the U.S., and Roche’s medical chief says use in over a thousand boys supports durable benefit and safety.