Even before the deaths linked to Sarepta Therapeutics’ gene therapies in 2025, families of patients with Duchenne muscular dystrophy (DMD) expressed dissatisfaction with the company’s flagship product, Elevidys. The therapy, which is currently the only approved gene treatment for DMD, costs $3.2 million per dose. Despite its availability, both the patient community and investors have voiced concerns about a lack of data transparency and disappointing clinical results.
At a 2024 patient advocacy conference, Catherine Collins, whose son Dylan received Elevidys, said: “You don’t think you can do a little more effort and give us a little bit more, a little bit more information? We’re going to turn on you. We’re the people who give you the millions of dollars, but you’re not giving us anything back, which is facts, data and science. You’re just taking money.”
In 2025, two deaths were linked to Elevidys in the spring. Later that year, another death was associated with an investigational gene therapy for limb-girdle muscular dystrophy using the same AAV vector as Elevidys. Sarepta’s share price fell by 80% over the course of 2025—its lowest point in nearly ten years.
Sarepta recently released three-year data for Elevidys alongside its fourth-quarter earnings report. While William Blair analysts described this as “incremental positive for Sarepta,” they added: “we do not think the data will significantly impact Elevidys prescribing and uptake behavior in the near term.” The company’s stock dropped another 12% following these announcements.
Louise Rodino-Klapac, president of research and development and technical operations at Sarepta, defended Elevidys during an interview with BioSpace: “The data speaks for itself,” she said. “We’re seeing an over four-point difference on NSAA [North Star Ambulatory Assessment]. This effect has grown over time, and I think that’s what’s really important.”
However, some industry experts remain unconvinced about Elevidys’ effectiveness. Mitchell Kapoor, senior analyst at H.C. Wainwright, commented after reviewing recent data: “I don’t know if [Elevidys] works,” he told BioSpace.
Three years after its approval as the first gene therapy for DMD patients in the United States (FDA announcement), Sarepta now faces new competition. REGENXBIO plans to file for approval of its own gene therapy candidate RGX-202 in mid-2026; Solid Biosciences also reported promising early-stage results for SGT-003 last year.
Sarepta reported preliminary fourth-quarter 2025 sales totaling $369.6 million—above consensus estimates—but Elevidys sales missed expectations by about $10 million due to factors including a severe flu season and delayed patient infusions into 2026.
For full-year 2026 guidance, Sarepta set a sales floor of $500 million for Elevidys but analysts such as Kapoor believe actual performance may fall short unless sales improve markedly this year.
To address declining demand and intensifying competition from companies like REGENXBIO (REGENXBIO corporate site), Sarepta announced plans to expand its sales force and increase outreach efforts within the DMD community.
Doug Ingram, CEO of Sarepta Therapeutics (company leadership page), initially projected continued dominance in treating DMD patients through decade’s end but recently acknowledged emerging competitors during public remarks.
Industry observers note that all current AAV-based therapies—including those from REGENXBIO and Solid Biosciences—use similar delivery vectors as Elevidys. Courtney Rice from Acadia Strategy Partners explained: “You can only AAV once…if you pick Sarepta’s treatment you’re potentially passing up alternatives.”
Rice further stated: “There’s resentment there; parents are hungry for an alternative…They’ve been held captive by [Elevidys] being the only thing on the market.”
As new options approach regulatory review or reach later stages of clinical testing (ClinicalTrials.gov), families affected by DMD may soon face choices between multiple one-time treatments—a significant shift after years relying solely on Elevidys.
