Sarepta Therapeutics Reports Positive Three-Year EMBARK Data for Elevidys in Duchenne Amid Past Safety Concerns

Sarepta released three-year topline data from the Phase 3 EMBARK study on January 26, 2026, showing Elevidys-treated ambulatory DMD patients (aged 4-7 at treatment) had statistically significant improvements in motor function compared to historical controls, with gaps widening over time123.

Key metrics included a 4-point difference on NSAA at three years (vs. 0.7 at 52 weeks), 73% slower disease progression in time-to-rise, and 70% in 10-meter walk-run234.

No new safety signals reported in year three, though four adverse events occurred; prior 2025 patient deaths led to FDA pause for non-ambulatory patients23.

Data aims to address slowing sales and safety concerns; analysts see it as positive but modest for sales impact, with shares rising2.

Sarepta plans Elevidys + rapamycin data by end-2026 to potentially resume non-ambulatory use34.

Sources:

1. https://marketchameleon.com/articles/b/2026/1/26/sarepta-embark-three-year-data-gene-therapy-duchenne

2. https://www.biopharmadive.com/news/sarepta-elevidys-duchenne-gene-therapy-embark-data/810461/

3. https://www.biospace.com/drug-development/sareptas-dmd-gene-therapy-staves-off-disease-three-years-after-treatment

4. https://www.clinicaltrialsarena.com/news/sarepta-elevidys-dmd-phase-iii-embark-three-year-data/