Sarepta Therapeutics has released updated findings from its Phase 3 EMBARK study, providing a three-year assessment of the gene therapy ELEVIDYS (delandistrogene moxeparvovec-rokl). These results, announced on January 26, 2026, focus on the functional impact of the treatment in ambulatory patients with Duchenne muscular dystrophy.
Breaking Down the Three-Year Results
The study compared ambulatory individuals treated with ELEVIDYS to an external control group (untreated individuals of similar age and health). The study tracked the functional impact of treatment on patients who were aged four to seven at time of treatment, and at time of last assessment were on average over nine years of age. Key findings include:
- Slowing Disease Progression: ELEVIDYS-treated patients showed a 70% or greater reduction in the rate of decline (as measured by Time to Rise (TTR) and 10-meter walk/run (10MWR)) compared to the control group.
- Maintaining Function: At a mean age of 9 years old, ELEVIDYS-treated patients achieved mean North Star Ambulatory Assessment (NSAA) scores above baseline three years after treatment.
- Improved Speed and Strength: There was a 73% slowing in the time it takes to rise from the floor (TTR) and a 70% slowing in the 10-meter walk/run test.
- Growing Impact: ELEVIDYS treated patients showed an increasing treatment effect over time, with the functional gap versus the external control group significantly widening between Year 2 and Year 3.
Safety and Monitoring
The long-term data showed no new treatment-related safety signals, which remains consistent with the manageable safety profile observed with ELEVIDYS in ambulatory patients to-date.
Access and Availability
- In the U.S.: ELEVIDYS It is approved for ambulatory individuals aged 4 and older, and is currently the only approved gene therapy for Duchenne.
- In the UK: Sarepta is collaborating with Roche, who are responsible for working with the relevant regulatory in the UK and Europe, but the treatment remains unavailable to patients in the UK.
About ELEVIDYS
ELEVIDYS (delandistrogene moxeparvovec-rokl) is a single-dose, adeno-associated virus (AAV)-based gene transfer therapy for intravenous infusion designed to address the underlying genetic cause of Duchenne muscular dystrophy – mutations or changes in the DMD gene that result in the lack of dystrophin protein – through the delivery of a transgene that codes for the targeted production of ELEVIDYS micro-dystrophin in skeletal muscle.
Training Plans and Injury Prevention
