Sarepta Voluntarily Pauses All Shipments of DMD Gene Therapy Elevidys Several Days After Informal Request From FDA

Sarepta Therapeutics stated that it has voluntarily paused all shipments of delandistrogene moxeparvovec-rokl (Elevidys), its marketed adeno-associated virus (AAV) vector-based gene therapy for Duchenne muscular dystrophy (DMD), in the United States.¹

The company stated that it has informed the FDA about its decision, which will go into effect at close of business on July 22, 2025. Notably, the move comes several days after the FDA made an informal request for Sarepta to voluntarily pause US shipments of Elevidys on the afternoon of July 18, 2025.² Initially, the company made a public statement that it would not be complying with the request, acknowledging that it had previously paused shipments to patients who are nonambulant, but would continue to ship the product to patients who are ambulant. The company justified its decision on the basis of a “comprehensive scientific interpretation of the data” that did not indicate any new or changed safety signals for Elevidys in ambulant patients.²

The company now seems to have changed its tune, having paused US shipments of Elevidys for all patients.¹ The company referred to the pause as “temporary” and stated that it will enable it to respond to potential requests for information from the FDA, as well as allow the company and the agency to finish a safety labeling supplement process for the gene therapy product.

“As a patient-centric organization, the decision to voluntarily and temporarily pause shipments of ELEVIDYS was a painful one, as individuals with Duchenne are losing muscle daily and in need of disease-modifying options,” Doug Ingram, JD, the president and chief executive officer of Sarepta, said in a statement.¹ “It is important for the patients we serve that Sarepta maintains a productive and positive working relationship with FDA, and it became obvious that maintaining that productive working relationship required this temporary suspension while we address any questions that FDA may have and complete the Elevidys label supplement process.”

In June 2025, the FDA announced that it is investigating 2 deaths that Sarepta Therapeutics had reported in patients who received treatment with Elevidys.³ Both deaths occurred in patients who were nonambulatory at the time of treatment and both patients had been hospitalized within 2 months of treatment in relation to raised levels of transaminases. Both patients’ deaths were attributed to acute liver failure (ALF) and the FDA stated that it is evaluating the risk of ALF that results in hospitalization or death after treatment with the gene therapy and whether additional regulatory measures will be necessary. Sarepta had made the move to voluntarily pause shipments of Elevidys to nonambulatory patients after the second patient’s death was reported in June 2025.⁴ The company also announced that it had voluntarily paused dosing in the confirmatory phase 3 ENVISION clinical trial (NCT05881408, Study 303) evaluating Elevidys and that it is working with a panel of experts to create an enhanced immunosuppressive regimen intend to bolster safety in nonambulatory patients.

“Our paramount priority is the safety and well-being of the patients we serve,” Louise Rodino-Klapac, PhD, the chief scientific officer and head of research & development at Sarepta, said in a June 15, 2025, statement.⁴ “We are taking immediate, decisive steps to better understand and mitigate the risk of ALF, including enhancing the immunosuppressive regimen, for those with Duchenne who are nonambulatory. We are deeply saddened by the loss of a second patient and extend our heartfelt condolences to the patient's family and his care team during this incredibly difficult time. DMD is a devastating disease that profoundly affects lives and often cuts them far too short. With more than 900 individuals treated to-date, we know how much hope families place in new treatment options like Elevidys – and we are committed to honoring that hope by acting swiftly, guided by scientific rigor and the insights of leading experts, to strengthen safety for all future patients.”

REFERENCES
1. Sarepta Therapeutics announces voluntary pause of Elevidys shipments in the U.S.. News release. Sarepta Therapeutics, Inc. July 21, 2025. Accessed July 22, 2025. https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-announces-voluntary-pause-elevidys
2. Sarepta Therapeutics provides statement on Elevidys. News release. Sarepta Therapeutics, Inc. July 18, 2025. Accessed July 22, 2025. https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-provides-statement-elevidys
3. FDA investigating deaths due to acute liver failure in non-ambulatory Duchenne muscular dystrophy patients following ELEVIDYS. News release. FDA. June 24, 2025. Accessed June 25, 2025. https://www.fda.gov/vaccines-blood-biologics/safety-availability-biologics/fda-investigating-deaths-due-acute-liver-failure-non-ambulatory-duchenne-muscular-dystrophy-patients
4. Sarepta provides safety update for ELEVIDYS and initiates steps to strengthen safety in non-ambulatory individuals with Duchenne. News release. Sarepta Therapeutics, Inc. June 15, 2025. Accessed June 25, 2025. https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-provides-safety-update-elevidys-and-initiates-steps?_ga=2.73448155.831031676.1750179906-198354166.1749064502