New data released Monday shows the benefits of Sarepta Therapeutics’ Elevidys gene therapy for Duchenne muscular dystrophy persist for at least three years after treatment, offering renewed hope for patients and a boost to the company after a challenging period.
three-Year Data Bolsters Elevidys’ Promise
The latest findings from the EMBARK trial demonstrate sustained improvements in motor function for patients receiving Elevidys.
- Elevidys stabilized symptoms of Duchenne muscular dystrophy (DMD) as measured by improvements in motor function.
- Patients receiving Elevidys experienced a 73% slowing of disease progression in time-to-rise and a 70% slowing in the 10-meter walk-run measurement.
- The data arrives after a arduous 2025 for Sarepta, marked by patient deaths and regulatory hurdles.
Elevidys appeared to stabilize the symptoms of DMD by a number of measures, according to the data from the Phase III EMBARK trial. Patients receiving the drug saw improvements in motor function as measured by a common scale of the disease’s effect on walking and muscle-use ability. Compared to an external control group, patients also had a slowing of disease progression by 73% in time-to-rise and 70% in the 10-meter walk-run measurement. Thes results were statistically significant, Sarepta announced.
“We’re encouraged by the concordance of these functional measures,” analysts at jefferies wrote Monday morning, “especially as physical function deteriorates after age.”
Shares of Sarepta rose 9% to $23.09 on Monday morning,compared to a previous closing price of $21.13 on Friday, signaling investor confidence in the long-term potential of Elevidys.
A Rocky Road to Recovery
Sarepta has been tracking patients in EMBARK annually for the past three years and will continue to log data out to five years.The first year of the trial supported Elevidys’ full approval in June 2024, according to Jefferies, after which patients in the treatment and placebo arms were switched, while maintaining the trial’s blinded nature.
Crystal Proud,a pediatric neurologist specializing in DMD,stated on Sarepta’s Monday call that the numerical benefits observed in the trial “mirror my observations” of her patients,and that the benefits of Elevidys have been “tangible and measurable.”
Sarepta reported that four patients in the third year of the trial experienced adverse events,though none of the safety signals were new. The company did not disclose the specific nature of these events.
Monday’s data release follows a turbulent 2025 for Elevidys. In March 2025, the gene therapy was implicated in the death of a young boy with DMD who developed acute liver injury leading to liver failure. A second patient death linked to liver damage followed in June 2025.
During an investor call discussing the second mortality, CEO doug Ingram emphasized that “liver injury is a known risk of AAV therapies.”
These deaths triggered months of regulatory scrutiny and prompted a business realignment for Sarepta. In July 2025, the biotech company laid off approximately 500 employees as part of a strategic shift away from gene therapies and toward its siRNA pipeline.
Sarepta added a black box warning to Elevidys regarding acute liver injury, liver failure, and the potential for death.The Food and Drug Administration (FDA) officially imposed the boxed warning in November 2025 and narrowed Elevidys’ approval to ambulatory patients aged 4 years and older.
The FDA paused dosing of Elevidys for non-ambulatory patients following the deaths last year. Sarepta plans to release data on a combination of Elevidys and the immunosuppressant medication rapamycin by the end of 2026, which, according to Jefferies, could lead the FDA to resume Elevidys shipments for non-ambulatory patients, who comprise half of the DMD patient population.
