FDA Investigates Patient Deaths Following Sarepta's DMD Gene Therapy Elevidys
The U.S. Food and Drug Administration (FDA) has launched an investigation into Sarepta Therapeutics' Duchenne muscular dystrophy (DMD) gene therapy Elevidys following reports of two patient deaths. The agency is evaluating the need for further regulatory action, potentially reshaping the landscape for gene therapies in rare diseases.
Safety Concerns Emerge for Elevidys
Sarepta reported two fatalities in non-ambulatory DMD patients treated with Elevidys, both linked to acute liver failure occurring within two months of treatment. The first death was disclosed in March 2025, with the second following in June. These incidents have raised significant concerns about the therapy's safety profile, particularly in more advanced stages of the disease.
In response to these events, Sarepta has implemented several safety measures:
- Suspension of Elevidys shipments for non-ambulatory patients in the U.S.
- Pausing of dosing in the Phase III ENVISION study, which includes both ambulatory and non-ambulatory patients
- Assembly of an independent expert panel to consider enhanced immunosuppression regimens
Roche, Sarepta's ex-U.S. partner, has discontinued Elevidys use in commercial non-ambulatory patients outside the United States.
Regulatory Scrutiny and Industry Impact
The FDA's investigation comes at a critical juncture for gene therapies, particularly those utilizing adeno-associated virus (AAV) vectors. Liver toxicity is a known risk associated with AAV-based therapies, but the severity of these cases has amplified concerns.
The regulatory landscape for Elevidys has been complex, with former FDA Center for Biologics Evaluation and Research (CBER) director Peter Marks overruling internal review teams to grant accelerated approval in 2023 and expanded approval in 2024. The current CBER director, Vinay Prasad, has been critical of these decisions, adding another layer of scrutiny to the ongoing investigation.
Industry analysts suggest that this development could have broader implications for the gene therapy field, potentially raising the regulatory bar for investigational DMD gene therapies and other rare disease treatments.
Market Response and Future Outlook
Sarepta's stock price plummeted following the announcement of the second death, with shares down 41% in pre-market trading on June 16, 2025. The company has suspended its revenue guidance, with plans to provide an update in its second-quarter earnings report.
As the FDA continues its investigation, the pharmaceutical industry awaits clarity on the future of Elevidys and potential ripple effects across the gene therapy landscape. The outcome may provide insights into the new FDA leadership's approach to novel cell and gene technologies for rare diseases, balancing innovation with patient safety.
References
- FDA Reviews Sarepta's Elevidys After Second Patient Death
The FDA is assessing the need for “further regulatory action” on Sarepta's Duchenne muscular dystrophy gene therapy in the aftermath of two patient deaths, though the regulator has not yet specified what action this could be.
- FDA investigates patient deaths after treatment with Sarepta's Duchenne gene therapy Elevidys
In one of the first major tests of the new FDA leadership’s regulatory philosophy toward gene therapies for rare diseases, the agency is investigating two Duchenne muscular dystrophy patient deaths following treatment with Sarepta Therapeutics’ Elevidys.
- FDA investigates patient deaths after treatment with Sarepta's Duchenne gene therapy Elevidys
In one of the first major tests of the new FDA leadership’s regulatory philosophy toward gene therapies for rare diseases, the agency is investigating two Duchenne muscular dystrophy patient deaths following treatment with Sarepta Therapeutics’ Elevidys.
- UPDATE: Second Patient Dies After Receiving Sarepta’s DMD Gene Therapy Elevidys
Sarepta’s shares crashed 41% in premarket trading Monday morning to $21.01 after the biotech reported a second death from acute liver failure, a known side effect of adeno-associated virus-based gene therapies.
- Sarepta reports 2nd death after DMD gene therapy Elevidys, stops dosing in half of patients
A second patient has died following treatment with Sarepta Therapeutics’ Elevidys, raising more doubts about the Duchenne muscular dystrophy gene therapy’s safety profile.
- Sarepta reports 2nd death after DMD gene therapy Elevidys, stops dosing in half of patients
A second patient has died following treatment with Sarepta Therapeutics’ Elevidys, raising more doubts about the Duchenne muscular dystrophy gene therapy’s safety profile.
Explore Further
What is the competitive landscape for Duchenne muscular dystrophy gene therapies currently approved or in development?
What specific safety measures have other companies taken to mitigate liver toxicity associated with AAV-based gene therapies?
How will the FDA's investigation into Elevidys potentially impact the regulatory process for other gene therapies under review?
What are the historical sales and market performance figures for Elevidys prior to the recent safety concerns?
What alternative treatments are available for non-ambulatory Duchenne muscular dystrophy patients, and how effective are they compared to Elevidys?