FDA Gives Sarepta Green Light to Restart Shipment of DMD Gene Therapy Elevidys to Patients Who Are Ambulatory
The FDA also stated that it has concluded its investigation of the death an 8-year-old boy who was treated with Elevidys in Brazil.
The FDA has recommended that Sarepta Therapeutics remove its voluntary hold on United States shipments of Duchenne muscular dystrophy (DMD) gene therapy delandistrogene moxeparvovec-rokl (marketed as Elevidys) for patients who are ambulatory.1
In a press release announcing the recommendation, the FDA also stated that it has concluded its investigation of the death an 8-year-old boy who was treated with Elevidys in Brazil and died on June 7, 2025.1-3 The agency stated that it has deemed the boy’s death was not related to the Elevidys product.
Although the hold on shipments for Elevidys was voluntary, the FDA had informally requested that Sarepta suspend all shipments of the gene therapy product earlier in July.4 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 nonambulatory, but would continue to ship the product to patients who are ambulatory. 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
“Last week, at the suggestion of FDA, Sarepta made the difficult decision to pause shipments of Elevidys to provide the FDA with an opportunity to complete a review of available safety information,” Doug Ingram, JD, the president and chief executive officer of Sarepta, said in the statement.5 “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 Elevidys for ambulatory patients. 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 Elevidys and to discussing the approach to risk-mitigation for nonambulatory patients, who remain on pause pending the outcome of those discussions.”
The previous voluntary pause for shipments of Elevidys to patients who are nonambulant, which remains in effect, was made after 2 nonambulant patients who were treated with Elevidys died in separate incidents earlier this year.5 Both of these patients’ deaths were attributed to acute liver failure (ALF).6,7
“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 its press release recommending the hold for ambulatory patients be lifted.1 “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.”
In addition to the deaths of patients treated with Elevidys, a 51-year-old patient treated with Sarepta’s investigational gene therapy SRP-9004 for nonambulant limb-girdle muscular dystrophy (LGMD) type 2D/R3, which uses the same AAVrh74 serotype as Elevidys, also recently died after receiving the product in a phase 1 clinical trial (DISCOVERY; NCT01976091).8,9 According to the FDA, this death also appeared to be related to ALF.10 Notably, after this death was announced, the FDA revoked the platform technology designation it had previously granted to Sarepta for the AAVrh74 viral vector and placed clinical holds on all trials currently evaluating SRP-9004 and bidridistrogene xeboparvovec (also known as SRP-9003), which is an investigational gene therapy product in development by Sarepta for LGMD type 2E (also known as β-sarcoglycanopathy) that also uses the AAVrh74 vector.9-11
REFERENCES
1. 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
2. FDA investigating death of 8-year-old boy who received Elevidys. News release. FDA. July 25, 2025. Accessed July 30, 2025. https://www.fda.gov/news-events/press-announcements/fda-investigating-death-8-year-old-boy-who-received-elevidys
3. Sarepta Therapeutics provides clarifying statement on Elevidys. News release. Sarepta Therapeutics, Inc. July 25, 2025. Accessed July 30, 2025. https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-provides-clarifying-statement-elevidys
4. Sarepta Therapeutics provides statement on Elevidys. News release. Sarepta Therapeutics, Inc. July 18, 2025. Accessed July 30, 2025. https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-provides-statement-elevidys
5. 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, Inc. 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
6. 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 July 30, 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
7. Sarepta Therapeutics shares safetyupdate on Elevidys. News release. Sarepta Therapeutics, Inc. March 18, 2025. Accessed July 30, 2025. https://investorrelations.sarepta.com/static-files/0d505d91-6722-4528-aae0-1e99fcbc37e5
8. Sarepta Therapeutics provides statement on Elevidys. News release. Sarepta Therapeutics Inc. July 18, 2025. Accessed July 30, 2025. https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-provides-statement-elevidys
9. Philippidis A. StockWatch: Sarepta shares nosedive after LGMD gene therapy patient dies. Genetic Engineering & Biotechnology News. July 20, 2025. Accessed July 30, 2025. https://www.genengnews.com/topics/genome-editing/stockwatch-sarepta-shares-nosedive-after-lgmd-gene-therapy-patient-dies/
10. FDA requests Sarepta Therapeutics suspend distribution of Elevidys and places clinical trials on hold for multiple gene therapy products following 3 deaths. News release. FDA. July 18, 2025. Accessed July 30, 2025. https://www.fda.gov/news-events/press-announcements/fda-requests-sarepta-therapeutics-suspend-distribution-elevidys-and-places-clinical-trials-hold
11. U.S. FDA grants platform technology designation to the viral vector used in SRP-9003, Sarepta’s investigational gene therapy for the treatment of limb girdle muscular dystrophy type 2E/R4. News release. Sarepta Therapeutics Inc. June 4, 2025. Accessed July 30, 2025. https://investorrelations.sarepta.com/news-releases/news-release-details/us-fda-grants-platform-technology-designation-viral-vector-used
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