Following feedback from the EMA, Roche will initiate a new, global, pivotal phase 3 study for Elevidys with the goal of expanding access to the gene therapy.
The study aims to generate additional placebo-controlled data required for a regulatory resubmission with the EMA and address the needs for ambulatory boys living with Duchenne muscular dystrophy in Europe and other global regions.
The EMA refused marketing authorization for Elevidys back in July 2025, claiming that they study provided failed to show that Elevidys had an effect on movement abilities after 12 months.
The new study will evaluate the efficacy and safety of Elevidys compared to placebo over 72 weeks in approximately 100 early ambulatory boys with DMD.
The U.S. FDA granted accelerated approval for Elevidys to Sarepta in June 2023 for ambulatory pediatric patients aged 4-5 years who have a confirmed mutation in the DMD gene. A year later, the agency approved a label expansion for Elevidys to include all individuals, regardless of ambulatory status, with DMD with a confirmed mutation in the DMD gene who are at least 4 years of age. Then, following the deaths of two non-ambulatory patients last year, the non-ambulatory indication was removed from the indication and usage section of the therapy’s prescribing information.
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