Sarepta's Elevidys Continues to be Prescribed Despite Safety Concerns

Sarepta Therapeutics' gene therapy for Duchenne muscular dystrophy (DMD), Elevidys, remains in use despite recent safety concerns, according to a new analyst survey. The therapy, which has been subject to regulatory scrutiny following patient deaths, still maintains support from prescribing physicians, particularly for ambulatory patients.

Physician Survey Reveals Continued Elevidys Use

A survey conducted by BMO Capital Markets found that approximately 90% of doctors are still prescribing Elevidys, focusing on ambulatory patients. This comes after a series of deaths in non-ambulatory patients led to increased regulatory oversight and a brief hold on shipments by the FDA.

The survey, which included 30 physicians, revealed that more than a quarter of respondents would prescribe Elevidys to younger and healthier patients. Doctors cited a favorable risk/benefit ratio in ambulatory patients, with over 760 treated and no reported deaths in this group.

Safety Concerns and Regulatory Actions

Elevidys faced significant challenges earlier this year when two non-ambulatory patients died after receiving the gene therapy. The first death, reported in March, was attributed to liver injury—a known side effect of gene therapies using AAV vectors for delivery. A second death in June led to the addition of a black box warning on the Elevidys package, specifically addressing risks for non-ambulatory patients.

In July, a third death occurred in a clinical trial for another of Sarepta's gene therapies for limb-girdle muscular dystrophy, which also utilized an AAV vector. This event raised broader concerns about the future of AAV gene therapies.

The FDA temporarily halted Elevidys shipments in response to these incidents. However, the hold was lifted at the end of July after discussions between Sarepta and regulators.

Market Impact and Future Outlook

The safety concerns have significantly impacted Sarepta's market position, with the company's stock price declining by 85% year to date. As of September 16, 2025, Sarepta's stock opened at $17.47, compared to $124.09 at the beginning of the year.

Despite these challenges, demand for Elevidys persists, albeit at a slightly reduced level. The BMO survey indicates that 70% of physicians are seeing similar to slightly lower demand compared to the period when the deaths were initially reported.

Since Elevidys returned to the market on July 28, surveyed doctors reported collectively infusing 27 patients, with 12 more scheduled between Q3 2025 and Q1 2026. This translates to approximately $95 million in sales for the one-time infusion therapy, which costs $3.2 million per treatment.

Sarepta has implemented new suggested dosing guidelines, including the use of the immunosuppressant sirolimus alongside Elevidys to mitigate elevated liver enzymes believed to have caused the deaths. Eighty percent of surveyed doctors expressed confidence that this change would improve Elevidys' safety profile.

However, the future of Elevidys remains uncertain. Half of the surveyed physicians indicated they would cease prescribing the therapy and await further safety updates if a death were to occur in the ambulatory patient population. As of now, no such incident has been reported in the market environment.