Sarepta shares new 3-year Elevidys data

Sarepta Therapeutics presented 3-year topline functional results from patients treated in part 1 of the phase 3 EMBARK study evaluating gene therapy Elevidys in ambulatory individuals with Duchenne muscular dystrophy who were aged four to seven at time of treatment.

Three years after treatment, patients who received Elevidys demonstrated statistically significant, clinically meaningful and durable efficacy across all key motor function measures, North Star Ambulatory Assessment (NSAA), Time to Rise (TTR) and 10-meter walk/run (10MWR), when compared to a pre-specified propensity-weighted untreated external control group.

The mean NSAA score remained above baseline at year 3 for the Elevidys-treated group (n=52) while the external control group (n=73) continued to show the expected age-related decline below their baseline score. The Elevidys group showed a 73% slowing of disease progression as measured by TTR and 70% slowing of disease progression as measured by 10MWR when compared to the control group.

Patients treated with Elevidys in Part 1 maintained significantly higher levels of motor function three years after treatment compared to the external control group.

"Elevidys is the first gene therapy for Duchenne to show a dramatic shift in disease trajectory out to three years consistent with earlier long-term data extending up to five years. This is long-term data in a robust, controlled clinical dataset that demonstrates the power of a disease-modifying therapy targeting the underlying cause of Duchenne," said Louise Rodino-Klapac, Ph.D., president of R&D and technical operations, Sarepta.

Importantly, new treatment-related safety signals were observed in ambulatory individuals. In November 2025, Sarepta announced an update to the prescribing information for Elevidys. Following the deaths of two non-ambulatory patients, the non-ambulatory indication was removed from the indication and usage section of the therapy’s prescribing information. A boxed warning indicating the risk of ALI and acute liver failure was added to the therapy’s label.