New Death of Patient Treated With Sarepta’s Elevidys Being Investigated by FDA, but Company Claims it is Unrelated to the Therapy

On July 25, 2025, the FDA announced that it is investigating another death of a patient who was treated with delandistrogene moxeparvovec-rokl (Elevidys), Sarepta Therapetuics’ marketed adeno-associated virus (AAV) vector-based gene therapy for Duchenne muscular dystrophy (DMD).¹

The patient, an 8-year-old boy, was treated with Elevidys in Brazil and died on June 7, 2025.^1,2 Sarepta noted that its partner Roche provided additional context for the death, pointing out that the physician who reported the death deemed it to be unrelated to Elevidys. Furthermore, Roche stated that the patient did not receive Elevidys in the context of a clinical trial. Although, the company chose not to reveal additional details about the case. Sarepta added that it had reported the death to the FDA through the FDA Adverse Event Reporting System (FAERS) on June 18, 2025.

“I'm told this is the case from Brazil,” Angelica Peebles, a pharma reporter at CNBC, said in a social media post on X.com on July, 25, 2025, which linked to the FDA’s statement that it would be investigating the case.³ “Regulators there determined the boy likely died from flu intensified by immunosuppression.”

The patient’s death follows the recent deaths of several other patients treated with Sarepta gene therapy products.⁴ In June 2025, the FDA announced that it is investigating 2 deaths that Sarepta had reported in patients who received treatment with Elevidys. Both deaths occurred in patients who were nonambulatory at the time of treatment and were attributed to acute liver failure (ALF); 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. After the reports of these deaths it was also later revealed that a 51-year-old patient who was 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, in a phase 1 clinical trial (DISCOVERY; NCT01976091), had died.^5,6 According to the FDA, this death also appeared to be related to ALF.⁷ Notably, after these 3 deaths had been announced, the FDA revoked the platform technology designation it had previously granted to Sarepta for the AAVrh74 viral vector. 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 (also known as β-sarcoglycanopathy) that also uses the AAVrh74 vector.^6,8 On July 18, the FDA also informally requested that Sarepta pause all shipments 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.^7,9

“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 July 18 statement regarding the aforementioned clinical holds.⁷ “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.”

Also of note, Sarepta announced in a July 16, 2025, statement that it would be letting go of 36% of its employees.¹⁰ The plans, which will reduce its workforce by 500 employees, are part of a broader strategic restructuring.

“Faced with environmental changes, we have decided to act decisively, implementing a focused strategy to ensure Sarepta remains a vibrant, financially enduring, patient-centric organization dedicated to improving the lives of those with rare genetic diseases,” Doug Ingram, JD, the president and chief executive officer of Sarepta, said in the statement.¹⁰

REFERENCES
1. FDA investigating death of 8-year-old boy who received Elevidys. News release. FDA. July 25, 2025. Accessed July 28, 2025. https://www.fda.gov/news-events/press-announcements/fda-investigating-death-8-year-old-boy-who-received-elevidys
2. Sarepta Therapeutics provides clarifying statement on Elevidys. News release. Sarepta Therapeutics, Inc. July 25, 2025. Accessed July 28, 2025.https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-provides-clarifying-statement-elevidys
3. Angelica Peebles. Social media post on X.com. July 25, 2025. Accessed July 28, 2025. https://x.com/angelicapeebles/status/1948865473342570527
4. 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 28, 2025. https://www.fda.gov/vaccines-blood-biologics/safety-availability-biologics/fda-investigating-deaths-due-acute-liver-failure-non-ambulatory-duchenne-muscular-dystrophy-patients
5. Sarepta Therapeutics provides statement on Elevidys. News release. Sarepta Therapeutics Inc. July 18, 2025. Accessed July 28, 2025. https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-provides-statement-elevidys
6. Philippidis A. StockWatch: Sarepta shares nosedive after LGMD gene therapy patient dies. Genetic Engineering & Biotechnology News. July 20, 2025. Accessed July 28, 2025. https://www.genengnews.com/topics/genome-editing/stockwatch-sarepta-shares-nosedive-after-lgmd-gene-therapy-patient-dies/
7. 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 28, 2025. https://www.fda.gov/news-events/press-announcements/fda-requests-sarepta-therapeutics-suspend-distribution-elevidys-and-places-clinical-trials-hold
8. 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 28, 2025. https://investorrelations.sarepta.com/news-releases/news-release-details/us-fda-grants-platform-technology-designation-viral-vector-used
9. Sarepta Therapeutics announces voluntary pause of Elevidys shipments in the U.S. News release. Sarepta Therapeutics Inc. July 21, 2025. Accessed July 28, 2025. https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-announces-voluntary-pause-elevidys
10. Sarepta therapeutics announces strategic restructuring and pipeline prioritization plan to maintain long-term, sustainable growth and provides update on Elevidys label. News release. Sarepta Therapeutics, Inc. July 16, 2025. Accessed July 28, 2025. https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-announces-strategic-restructuring-and