Sarepta Therapeutics announced positive three-year topline results from the Phase 3 EMBARK study evaluating ELEVIDYS (delandistrogene moxeparvovec-rokl) in ambulatory patients with Duchenne muscular dystrophy (DMD).
Patients treated in Part 1 of the study, with a mean age of nine years at assessment, maintained motor function improvements three years after therapy.
Key functional outcomes showed sustained benefits compared with a propensity-weighted external control group: North Star Ambulatory Assessment (NSAA) scores remained above baseline, while Time to Rise (TTR) and 10-meter walk/run (10MWR) showed a 70% or greater slowing of disease progression. Treatment effects increased over time, with the gap between ELEVIDYS-treated patients and controls widening between Year 2 and Year 3.
No new safety concerns were observed, consistent with the therapy’s previously established manageable profile. ELEVIDYS remains the only approved gene therapy for Duchenne, having been administered to over 1,200 patients globally in clinical and real-world settings.
The EMBARK study included 125 patients aged 4–7 at enrollment, with follow-up through Part 1 and crossover in Part 2. Post-study, 52 patients continue to be monitored in the long-term EXPEDITION study. ELEVIDYS is approved for use in ambulatory patients aged 4 and above.
Sarepta, in collaboration with Roche, oversees regulatory approval and commercialization of ELEVIDYS in the U.S., while Roche manages international access. Commercialization in Japan is through Chugai Pharmaceuticals.
The three-year EMBARK results reinforce ELEVIDYS as a disease-modifying therapy for Duchenne, demonstrating durable functional benefits and a favorable safety profile, signaling a meaningful advancement in long-term care for patients living with this progressive condition.
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