Sarepta Elevidys Results Show Three-Year Benefit

Sarepta Therapeutics said in a Jan. 26, 2026, press release that the Sarepta Elevidys results at three years showed statistically significant slowing of Duchenne muscular dystrophy progression on three motor-function measures, renewing focus on the therapy's commercial outlook and regulatory path for non-ambulatory patients.

Three-Year EMBARK Trial Results and Outlook

In the Phase 3 EMBARK trial, ambulatory patients treated with Elevidys (n=52) maintained a mean North Star Ambulatory Assessment (NSAA) score above baseline at Year 3, while an external control group (n=73) showed the expected age-related decline below baseline. Elevidys slowed disease progression by 73% on Time to Rise and 70% on the 10-meter walk/run compared with the external control. The NSAA difference widened from a non-significant 0.7-point gap at 52 weeks to a statistically significant four-point advantage at three years.

The company said the pre-specified external-control analysis pooled data from three studies—one randomized trial and two natural-history cohorts—and applied propensity-score weighting to balance age, height, body-mass index, steroid use, baseline NSAA, and timed-function tests. Participants were 4–7 years old at treatment and averaged just over nine years at the final assessment, making the durability of the functional benefit central to Sarepta’s clinical case.

Sarepta reported no new treatment-related safety signals in the three-year data and is analyzing functional outcomes from patients who crossed over to receive Elevidys about two years after treatment. The update follows a turbulent 2025, when two patient deaths linked to liver injury led regulators to pause dosing for non-ambulatory patients. Elevidys’ approval was narrowed to ambulatory patients aged 4 and older in November 2025, and a black box warning was added.

On the commercial front, Elevidys generated $898.7 million in full-year 2025 sales, including $110.4 million in the fourth quarter. Quarterly revenue declined from $282 million in Q2 to $132 million in Q3 and then to the fourth-quarter level amid safety concerns and the restricted label. Sarepta has not updated sales forecasts or revenue guidance tied to the three-year results.

Analysts see a mixed outlook: the consistent functional improvements and widening treatment effect strengthen the efficacy case, but some expect only modest commercial growth given the largely treated initial patient pool and ongoing hesitancy among some physicians and families.

Sarepta plans to present data on Elevidys combined with rapamycin, an immunosuppressant, by the end of 2026. This combination data could support FDA resumption of Elevidys shipments for non-ambulatory patients. The EMBARK cohort will continue to be followed to a planned five-year endpoint, a milestone regulators and commercial stakeholders will watch as they assess durability against the drug’s safety record.