Roche will launch a global Phase III trial of its Duchenne muscular dystrophy (DMD) gene therapy Elevidys (delandistrogene moxeparvovec), after the drug was rejected for use in Europe.
The European Medicines Agency (EMA) said that patient improvement was not significant enough for approval in July 2025. Based on this feedback, Roche will generate new placebo-controlled data through a pivotal, global trial.
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The study will evaluate the efficacy and safety of Elevidys compared to placebo over 72 weeks in approximately 100 early ambulatory boys with DMD. The primary endpoint of the study is the change in “Time to Rise” (TTR) from the floor velocity, which is a critical prognostic factor of future disease progression.
Dr Levi Garraway, CMO of Roche, said, “The initiation of this new study reflects Roche’s commitment to the Duchenne community and our resolve to make this disease-modifying therapy available to ambulatory boys in Europe and around the world. Our confidence is rooted in robust long-term data showing the durable efficacy and safety of Elevidys, alongside the experience of treating more than a thousand ambulatory boys worldwide.”
Elevidys is approved for ambulatory DMD patients in the US, where it’s sold by Sarepta Therapeutics. The US approval was based on the Phase III EMBARK trial (NCT05096221), despite the study missing its primary endpoint of improvement in motor function after one year.
The EMA noted that the 0.65-point difference with placebo in the 34-point North Star Ambulatory Assessment scores was not statistically significant.
The US Food and Drug Administration (FDA) and Sarepta were embroiled in a regulatory disagreement in 2025 after several patients dosed with Elevidys and other Sarepta adeno-association virus (AAV) gene therapies suffered fatal adverse events related to acute liver injury (ALI).
Following the deaths, the FDA and Sarepta agreed to a pause on certain Elevidys trials, and the company temporarily stopped sales of the gene therapy after pressure from the agency. These pauses have since been lifted.
Elevidys remains available as appropriate to ambulatory individuals ages four and over, as per an updated label announced earlier in November 2025. The new label has added a boxed warning for the risk of ALI and acute liver failure (ALF), as well as the therapy no longer being approved for use in the non-ambulatory indication.
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