FDA Revokes Sarepta’s Platform Technology Designation for Muscular Dystrophy Gene Therapy Vector Following Patient Deaths

Sarepta Therapeutics’ platform technology designation from the FDA for the AAVrh74 viral vector it uses in gene therapy products for multiple muscular dystrophy indications has been revoked by the agency.¹

The FDA’s decision follows the recent death of a 51-year-old patient who was treated with Sarepta’s investigational gene therapySRP-9004 for nonambulant limb-girdle muscular dystrophy (LGMD) type 2D/R3, which uses the AAVrh74 serotype, in a phase 1 clinical trial (DISCOVERY; NCT01976091).^2,3 In its announcement of the revocation, the FDA also made reference to 2 previous deaths that occurred inpatients who received delandistrogene moxeparvovec-rokl (Elevidys), the company’s marketed adeno-associated virus (AAV) vector-based gene therapy for Duchenne muscular dystrophy (DMD) that also uses the AAVrh74 vector.¹ The agency noted that all 3 deaths seem to have been caused by acute liver failure (ALF).

In addition to revoking the platform technology designation, the FDA also 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 (LGMD2E/R4, also known as beta sarcoglycanopathy) that also uses the AAVrh74 vector.^3,4 On July 18, the FDA also informally requested that Sarepta pause all shipment of Elevidys in the United States, a request that Sarepta initially stated it would not fulfill until reversing its position on the matter several days later.^1,5

“Protecting patient safety is our highest priority, and the FDA will not allow products whose harms are greater than benefits,” Vinay Prasad, MD, MPH, the director of the FDA’s Center for Biologics Evaluation and Research, said in a statement.¹ “The FDA will halt any clinical trial of an investigational product if clinical trial participants would be exposed to an unreasonable and significant risk of illness or injury.”

The FDA originally granted the platform technology designation to Sarepta for the rAAVrh74 viral vector in June 2025.⁴ Notably, Sarepta was one of the first companies to receive the designation, which was recently established by the FDA with the intent of allowing companies developing a product that uses a well-understood technology that has been previously used in other programs to utilize data regarding that technology from the other programs in support of investigational new drug applications and biologic license applications.

“Today, we’ve shown that this FDA takes swift action when patient safety is at risk,” FDA Commissioner Marty Makary, MD, MPH, added to the statement.¹ “We believe in access to drugs for unmet medical needs but are not afraid to take immediate action when a serious safety signal emerges.”

The FDA is currently investigating the 2 deaths of the patients who were treated with Elevidys.⁶ 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 ALF and the FDA statedthat it is evaluating the risk of ALF that results in hospitalization or death after treatment with the gene therapy and whether additionalregulatory measures will be necessary.

“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.⁷ “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. 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 22, 2025. https://www.fda.gov/news-events/press-announcements/fda-requests-sarepta-therapeutics-suspend-distribution-elevidys-and-places-clinical-trials-hold
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. StockWatch: Sarepta shares nosedive after LGMD gene therapy patient dies. News article. Alex Philippidis. Genetic Engineering & Biotechnology News. July 20, 2025. Accessed July 22, 2025.https://www.genengnews.com/topics/genome-editing/stockwatch-sarepta-shares-nosedive-after-lgmd-gene-therapy-patient-dies/
4. 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 22, 2025. https://investorrelations.sarepta.com/news-releases/news-release-details/us-fda-grants-platform-technology-designation-viral-vector-used
5. 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
6. FDA investigating deaths due to acute liver failure in non-ambulatory Duchenne muscular dystrophy patients following ELEVIDYS. News release. FDA. June 24, 2025. Accessed July 22, 2025. https://www.fda.gov/vaccines-blood-biologics/safety-availability-biologics/fda-investigating-deaths-due-acute-liver-failure-non-ambulatory-duchenne-muscular-dystrophy-patients
7. 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 22, 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