Roche Phase III study shows benefits of Elevidys in DMD

27 January 2025

Swiss pharma giant Roche (ROG: SIX) today announced positive top-line results from year two of the EMBARK trial, a global, randomized, double-blind Phase III study of Elevidys (delandistrogene moxeparvovec), the first approved gene therapy for the treatment of individuals with Duchenne muscular dystrophy.

Roche noted that, two years after treatment with Elevidys, statistically-significant and clinically-meaningful improvements were observed across three key motor function measures of NSAA, TTR and 10MWR, when compared to a pre-specified propensity-weighted untreated external control group. Functional differences between individuals treated with Elevidys and those in the external control group increased between one and two years after dosing. Together, these results demonstrate consistent, sustained benefit in favor of Elevidys.

Detailed results from year two of the EMBARK study will be shared at an upcoming medical meeting and discussed with health authorities.

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