FDA Restricts Use of Sarepta's Elevidys Gene Therapy Following Patient Deaths

On November 14, 2025, the U.S. Food and Drug Administration (FDA) announced significant restrictions on the use of Sarepta Therapeutics’ gene therapy, Elevidys (delandistrogene moxeparvovec-rokl), following the deaths of two patients from acute liver failure. The FDA's updated label now limits Elevidys to ambulatory patients aged four years and older with Duchenne muscular dystrophy (DMD), effectively barring non-ambulatory patients from receiving the treatment.

FiercePharmaNew York TimesSarepta Announces FDA’s Approval of Updated ELEVIDYS Prescribing Information

The revised prescribing information for Elevidys includes a boxed warning, the FDA's most serious safety alert, highlighting the risks of acute serious liver injury and acute liver failure. The FDA's decision follows a safety review that confirmed the liver damage leading to the fatalities. Additionally, the updated label specifies that Elevidys is contraindicated for patients with preexisting liver impairment, recent vaccinations, or active infections. Patients must also undergo weekly liver function monitoring for at least three months post-infusion and remain near a medical facility for two months after treatment.

Sarepta Announces FDA’s Approval of Updated ELEVIDYS Prescribing InformationFiercePharma

Elevidys is a one-time gene therapy designed to address the genetic cause of DMD by delivering a transgene that produces micro-dystrophin in skeletal muscle. While it has been administered to over 1,100 patients globally, the therapy has faced scrutiny due to safety concerns, particularly regarding liver-related complications. The FDA's restrictions come after Sarepta voluntarily suspended treatment for non-ambulatory patients earlier in 2025, following reports of liver failure.

FiercePharma

The FDA's decision has been met with mixed reactions. Advocates for DMD patients have expressed concerns over the limited treatment options available for non-ambulatory patients, who typically lose mobility around age 12. Critics argue that the FDA's actions reflect a broader issue of regulatory oversight in the approval of gene therapies, particularly given the urgency expressed by families seeking effective treatments for this devastating disease.

New York Times

Louise Rodino-Klapac, Ph.D., Sarepta's president of research and development, stated, “Completion of the safety labeling change for ELEVIDYS will ensure that families and healthcare professionals have clear information... to help understand these updates and guide treatment decisions.” The FDA emphasized the need for ongoing monitoring and research, requiring Sarepta to conduct a post-marketing observational study involving approximately 200 DMD patients to further assess the risks associated with Elevidys.

Sarepta Announces FDA’s Approval of Updated ELEVIDYS Prescribing Information

While the FDA's decision is based on confirmed cases of liver injury and fatalities, there remains a lack of comprehensive data regarding the long-term effects of Elevidys and the overall efficacy of the treatment in the broader DMD patient population. The agency's requirement for further studies indicates ongoing concerns about the therapy's safety profile.

FiercePharma

Sarepta plans to initiate a study of an enhanced sirolimus immunosuppressive regimen aimed at mitigating the risks of liver injury, potentially allowing for the resumption of dosing in non-ambulatory patients, pending FDA agreement. The company continues to seek full FDA approval for its other DMD treatments despite recent setbacks in clinical trials.

FiercePharma

• “will ensure that families and health-care professionals have clear information,” — Louise Rodino-Klapac, Ph.D., Sarepta Therapeutics

New York Times

• “said it had decided to restrict the therapy to boys 4 years and older who were still able to walk.” — FDA Statement

New York Times

The FDA's decision marks a pivotal moment in the landscape of gene therapy for Duchenne muscular dystrophy, balancing the urgent need for effective treatments against the imperative of patient safety.

New York Times