Delandistrogene moxeparvovec

After a series of safety issues, including deaths, nearly derailed Sarepta Therapeutics' Duchenne muscular dystrophy (DMD) treatment Elevidys (delandistrogene moxeparvovec-rokl) last year, the company shared some positive news with new three-year data for its beleaguered gene therapy. The update sent shares in the company up as much as 13% after markets opened Monday.Initially granted FDA accelerated approval in 2023 for use in ambulatory patients between the ages of four and five, Elevidys' label was expanded the following year to include both non-ambulatory boys and those aged four years and older. The expansion was based on data from the Phase III EMBARK study, which had missed its primary endpoint, but did achieve significance on several key secondary goals, including time-to-rise and 10-metre walk test.Two-year data from EMBARK, reported a year ago, showed sustained improvements and disease stabilisation in ambulatory patients treated with Elevidys. The study includes boys ages four through seven years old. In Part 1, participants were randomly assigned to Elevidys or placebo with a follow-up period to test the primary endpoint of North Star Ambulatory Assessment (NSAA) total score. That was followed by a crossover in Part 2 where groups switched treatments while remaining blinded. Each phase had a 52-week follow-up to assess effectiveness.Three-year improvementsIn the latest update, Sarepta said that three years after receiving Elevidys, the 52 patients from Part 1 of EMBARK continued to show meaningful improvements in physical function, scoring 4.39 points higher on the NSAA compared to matched external controls. They also performed better on key mobility tests, taking 6.05 seconds less to stand up from a seated position and completing a 10-metre walk/run test 2.7 seconds faster than the control group.Oppenheimer analysts said that although investors tend to be wary of studies using external controls, the update should nevertheless bolster physician and patient perceptions of Elevidys' benefit-risk profile ⁠and help accelerate commercial ⁠uptake.Sparse on new safety dataHowever, shortly after disclosing the two-year update last January, Sarepta said that a 16-year-old boy had died from acute liver failure following treatment with Elevidys. That was followed by a second fatality a few months later. The safety issues led to Sarepta voluntarily pausing shipments of Elevidys amid pressure from the FDA, although the regulator subsequently allowed the therapy back on the market, albeit with a more restrictive label and a boxed warning for acute serious liver injury and acute liver failure.Sarepta said little about the safety profile of Elevidys in Monday's update, other than that "no new treatment-related safety signals were observed, reinforcing the consistent and manageable safety profile seen in ambulatory patients…to date." The company indicated that further analysis of the three-year data is ongoing, and findings are expected to be presented at upcoming medical meetings.Earlier this month, Sarepta released preliminary financial results, with Elevidys generating sales in 2025 of $898.7 million, including $110.4 million in the fourth quarter, a period CEO Doug Ingram said "was impacted by the severe year-end flu season and the need in December to reschedule six patient infusions into 2026."Ingram also reconfirmed that sales of Elevidys should be at least $500 million in 2026. Although he stated the company's "intentions to exceed it," he said "we will stop short of providing more detailed guidance."

iStock, ktsimage After a spate of patient deaths in 2025 linked to the company’s Duchenne gene therapy, Sarepta shared new data showing benefits of the therapy three years after dosing. The benefits of taking Elevidys, Sarepta Therapeutics’ gene therapy for Duchenne muscular dystrophy, continue three years post treatment, the company said Monday. Sarepta announced year three data from the Phase III EMBARK trial testing Elevidys in ambulatory patients with DMD, marking a positive step after a discouraging 2025 marred by patient deaths and a plummeting stock price. Elevidys appeared to stabilize the symptoms of DMD by a number of measures, according to the data. Patients receiving the drug saw improvements in motor function as measured by a commons scale of the disease’s effect on walking and muscle-use ability. Patients also had a slowing of disease progression by 73% in time-to-rise and 70% in the 10-meter walk-run measurement, compared to an external control. The results in all three measurements were statistically significant, Sarepta said. “We’re encouraged by the concordance of these functional measures,” analysts at Jefferies wrote Monday morning after the data announcement, “especially as physical function deteriorates after age.” Sarepta’s shares rose 9% to $23.09 on Monday morning, compared to a previous closing price of $21.13 on Friday. Sarepta has been tracking patients in EMBARK every year for the past three years and will continue logging data out to five years. The first year of the trial was used to support Elevidys’ full approval in June 2024, according to Jefferies, after which patients in the treatment and placebo arms were switched, while keeping the trial blinded. Crystal Proud, a pediatric neurologist who specializes in DMD, said on Sarepta’s Monday call that the numerical benefits 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 year three of the trial experienced adverse events , though none of the safety signals were new. Sarepta did not say what the adverse events were. Monday’s readout comes after a tumultuous 2025 for Elevidys. In March, the gene therapy was implicated in the death of a young boy with DMD who developed acute liver injury leading to liver failure. This was followed by another patient death in June 2025 , likewise linked to liver damage. In an investor call to discuss the second mortality, CEO Doug Ingram insisted that “liver injury is a known risk of AAV therapies.” Still, the deaths ushered in months of regulatory difficulties and business realignment efforts for Sarepta. In July 2025, the biotech laid off around 500 employees in a pivot that included moving away from gene therapies and leaning into its siRNA pipeline. Sarepta at the time also added a black box warning to Elevidys for acute liver injury, liver failure and the potential for death. The FDA in November 2025 officially imposed the boxed warning on Elevidys and also narrowed its approval to only ambulatory patients 4 years and up. The FDA paused dosing of Elevidys for non-ambulatory patients after the deaths last year. Sarepta is planning on releasing data on a combination of Elevidys and the immunosuppressant medication rapamycin by the end of 2026, that, according to Jefferies, could push the FDA to resume Elevidys shipments in non-ambulatory patients, who make up half of the DMD patient population.