Sarepta Therapeutics reports positive 3-year topline data from EMBARK Phase 3 trial: ELEVIDYS gene therapy slows Duchenne muscular dystrophy progression by 73% in Time to Rise, 70% in 10MWR for ambulatory kids. FDA-approved details inside.
Written By: Pharmacally Medical News Desk
Sarepta Therapeutics announced positive topline three-year functional outcomes from Part 1-treated patients in EMBARK (Study SRP-9001-301), a global Phase 3 trial evaluating ELEVIDYS (delandistrogene moxeparvovec-rokl) in ambulatory children with Duchenne muscular dystrophy (DMD).
The study enrolled male patients aged four to seven years at the time of treatment. At the latest assessment, treated participants were on average older than nine years, a stage when Duchenne-related functional decline typically accelerates.
According to Sarepta, patients who received ELEVIDYS demonstrated statistically significant, clinically meaningful, and durable preservation of motor function across all major endpoints compared with a pre-specified propensity-weighted untreated external control group.
Louise Rodino-Klapac, Ph.D., president of research and development and technical operations at Sarepta, said ELEVIDYS is the first gene therapy in Duchenne to demonstrate a dramatic shift in disease progression out to three years in a controlled clinical dataset, consistent with earlier follow-up extending up to five years.
Three-Year Results
Three years after receiving a single one-time infusion of ELEVIDYS, patients maintained significantly higher levels of ambulatory motor performance, while the external control group continued to show the expected age-related decline below baseline.
Sarepta reported that the therapy slowed disease progression by 73% on Time to Rise (TTR) and 70% on the 10-meter walk/run (10MWR). The mean NSAA score remained above baseline at Year 3 in the treated group, contrasting with continued deterioration in untreated matched controls.
Topline efficacy findings included:
- North Star Ambulatory Assessment (NSAA): +4.39-point improvement (p=0.0002)
- Time to Rise (TTR): -6.05 seconds improvement (p<0.0001)
- 10-meter walk/run (10MWR): -2.70 seconds improvement (p=0.0039)
Crystal Proud, M.D., chief of Neurology and director of Neuromuscular Medicine at Children’s Hospital of The King’s Daughters and an EMBARK investigator, said the findings provide clearer evidence of how ELEVIDYS may help preserve everyday motor abilities such as standing, walking, and running.
Safety Profile
Sarepta reported no new treatment-related safety signals, reinforcing the manageable safety profile observed to date in ambulatory patients treated with ELEVIDYS.
EMBARK Trial Design
EMBARK (NCT05096221) was a multinational, randomized, placebo-controlled Phase 3 study conducted in two parts with a crossover design. In Part 1, 125 participants received either ELEVIDYS or placebo with 52-week follow-up. In Part 2, patients crossed over to the opposite treatment while remaining blinded.
The primary endpoint was change from baseline in NSAA at Week 52. Secondary measures included timed function tests, dystrophin production, stride velocity, and patient-reported mobility outcomes.
Further analysis is ongoing, including functional outcomes from crossover-treated participants two years after dosing. Sarepta plans to present additional data at upcoming medical meetings and in publication.
Two-year EMBARK results were published earlier this month in Neurology & Therapy and 1 year result was published in Nature Medicine.
Following trial completion in 2024, eligible participants could enroll in EXPEDITION (Study SRP-9001-305), Sarepta’s long-term follow-up program. Sarepta noted that 52 Part 1-treated patients continue to be followed three years after dosing.
ELEVIDYS Approval Status
ELEVIDYS is a single-dose AAV-based gene transfer therapy designed to address the underlying genetic cause of DMD by delivering a micro-dystrophin transgene to skeletal muscle.
It is currently indicated for ambulatory patients aged four years and older with a confirmed mutation in the DMD gene, following an updated FDA label announced at the end of 2025.
To date, Sarepta said ELEVIDYS has been administered to more than 1,200 patients globally.
Global Collaboration with Roche
Under a collaboration agreement signed in 2019, Sarepta and Roche are jointly working to expand global access to ELEVIDYS for the Duchenne community. Sarepta is responsible for regulatory approval, manufacturing, and commercialization in the United States, while Roche oversees regulatory approvals and commercialization outside the U.S. In Japan, commercialization is being handled by Chugai Pharmaceuticals, a member of the Roche Group.
References
Sarepta Announces Positive Topline Three-Year EMBARK Results Showing ELEVIDYS Significantly Slows Disease Progression on Key Functional Measures in Ambulatory Duchenne Patients, 26 January 2026, https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-announces-positive-topline-three-year-embark-results
Mendell, J.R., Muntoni, F., McDonald, C.M. et al. Two-Year Outcomes Following Delandistrogene Moxeparvovec Treatment in Ambulatory Patients with Duchenne Muscular Dystrophy: Phase 3 EMBARK Trial. Neurol Ther (2026). https://doi.org/10.1007/s40120-025-00879-8
A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Participants with Duchenne Muscular Dystrophy (DMD) (EMBARK), ClinicalTrials.gov ID NCT05096221, https://clinicaltrials.gov/study/NCT05096221
Mendell JR et al, AAV gene therapy for Duchenne muscular dystrophy: the EMBARK phase 3 randomized trial. Nat Med. 2025 Jan;31(1):332-34. Epub 2024 Oct 9. PMID: 39385046; PMCID: PMC11750718. https://doi.org/10.1038/s41591-024-03304-z
Sarepta Announces FDA’s Approval of Updated ELEVIDYS Prescribing Information, 14 November 2025, https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-announces-fdas-approval-updated-elevidys-prescribing