FDA Recommends Sarepta Resume DMD Gene Therapy Shipments for Ambulatory Patients

The FDA notified Sarepta Therapeutics that the voluntary pause placed on shipments of delandistrogene moxeparvovec (Elevidys; Sarepta) to ambulatory patients with Duchenne muscular dystrophy (DMD) can be lifted, according to a press release from the company.¹ Shipping to sites of care for ambulatory patients with DMD will resume imminently, the company stated.

“Last week, at the suggestion of FDA, Sarepta made the difficult decision to pause shipments of [delandistrogene moxeparvovec] to provide the FDA with an opportunity to complete a review of available safety information,” Doug Ingram, CEO at Sarepta, said in a statement.

Following an investigation, the FDA confirmed that shipments of delandistrogene moxeparvovec can resume. | Image Credit: immimagery - stock.adobe.com

The voluntary pause on shipments of delandistrogene moxeparvovec—a gene therapy indicated for the treatment of patients with DMD aged 4 years or older, including ambulatory and nonambulatory patients²—came following an informal request from the FDA to halt shipment of the therapy.³ Initially, only shipments to nonambulatory patients were paused, but all US shipments were later paused to allow Sarepta and the FDA to collaboratively review the safety label following recent deaths of nonambulatory patients due to acute liver failure.^4,5

“Our paramount priority is the safety and well-being of the patients we serve,” Louise Rodino-Klapac, PhD, chief scientific officer and head of research and development at Sarepta, said in a press release following the second reported acute liver failure–related death.⁵ “We are taking immediate, decisive steps to better understand and mitigate the risk of acute liver failure, including enhancing the immunosuppressive regimen, for those with Duchenne who are non-ambulatory.”

The FDA review of safety data in ambulatory patients included the case of an 8-year-old patient in Brazil, which Brazilian health authorities deemed unlikely to be related to the gene therapy.¹ Following the investigation, the FDA confirmed that shipments of delandistrogene moxeparvovec for ambulatory patients can resume.

"The FDA will continue to work with the sponsor regarding nonambulatory patients, which remains subject to a voluntary hold, following 2 deaths," the FDA wrote in a press release.⁶ "The patient community is an important voice, and the FDA will continue to listen to and respond to thoughts from the community impacted by DMD."

The initial approval of delandistrogene moxeparvovec came in June 2023 for patients with DMD aged 4 to 5 years.² An expanded approval in June 2024 included the treatment of all patients aged 4 years and older, while the therapy’s approval for the treatment of nonambulatory patients remains under the FDA’s accelerated approval pathway.² The ENVISION study (NCT05881408), which is the confirmatory trial required under the accelerated approval for nonambulatory patients, was also voluntarily paused to allow for evaluation of a protocol amendment to incorporate an enhanced immunosuppressive regimen for nonambulatory patients.⁵

Delandistrogene moxeparvovec, a single-dose, adeno-associated virus–based gene transfer therapy, addresses mutations in the DMD gene that cause a lack of dystrophin protein, which is the underlying cause of DMD.¹ It is the only approved therapy for DMD, which is a progressive inherited disease that leads to muscle atrophy and eventual death in affected patients. 

“We are very pleased that FDA chose to rapidly and comprehensively complete that review and to recommend that we remove our voluntary pause and resume shipment of [delandistrogene moxeparvovec] for ambulatory patients,” Klapac said.¹ “The FDA’s swift review evinces a commitment to the Duchenne population, a commitment shared by Sarepta. We look forward to working collaboratively with the FDA to complete the safety label update for [delandistrogene moxeparvovec] and to discussing the approach to risk mitigation for nonambulatory patients, who remain on pause pending the outcome of those discussions.”

References

1. FDA informs Sarepta that it recommends that Sarepta remove its pause and resume shipments of Elevidys for ambulatory individuals with Duchenne muscular dystrophy. News release. Sarepta Therapeutics. July 28, 2025. Accessed July 30, 2025. https://investorrelations.sarepta.com/news-releases/news-release-details/fda-informs-sarepta-it-recommends-sarepta-remove-its-pause-and

2. Shaw M. FDA grants 2 approvals to delandistrogene moxeparvovec for DMD. AJMC^®. June 20, 2024. Accessed May 23, 2025. https://www.ajmc.com/view/fda-grants-2-approvals-to-delandistrogene-moxeparvovec-for-dmd

3. Sarepta Therapeutics provides statement on Elevidys. News release. Sarepta Therapeutics. July 18, 2025. Accessed July 30, 2025. https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-provides-statement-elevidys

4. Sarepta Therapeutics announces voluntary pause of Elevidys shipments in the U.S. News release. Sarepta Therapeutics. July 21, 2025. Accessed July 30, 2025. https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-announces-voluntary-pause-elevidys

5. Sarepta provides safety update for Elevidys and initiates steps to strengthen safety in non-ambulatory individuals with Duchenne. News release. Sarepta Therapeutics. June 15, 2025. Accessed July 30, 2025. https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-provides-safety-update-elevidys-and-initiates-steps

6. FDA recommends removal of voluntary hold for Elevidys for ambulatory patients. News release. FDA. July 28, 2025. Accessed July 30, 2025. https://www.fda.gov/news-events/press-announcements/fda-recommends-removal-voluntary-hold-elevidys-ambulatory-patients