Sarepta Therapeutics' Gene Therapy Elevidys Under FDA Investigation Following Death of 8-Year-Old Boy

The FDA is investigating the death of an 8-year-old boy who received Sarepta's Elevidys, a gene therapy for Duchenne muscular dystrophy. The death occurred on June 7, 2025. The FDA has not disclosed further details about the case, but it is conducting a thorough investigation to determine the cause of the death.

The FDA is investigating the death of an 8-year-old boy who received Sarepta Therapeutics' Elevidys, a gene therapy for Duchenne muscular dystrophy. The death occurred on June 7, 2025. The FDA has not disclosed further details about the case, but it is conducting a thorough investigation to determine the cause of the death.

Sarepta Therapeutics, a biotechnology company, has faced significant scrutiny following the recent deaths of three patients treated with its gene therapies. The company initially refused to suspend shipments of Elevidys after the FDA requested it on July 18, 2025, following the death of a third patient. However, Sarepta later agreed to the FDA's recommendation to suspend shipments of Elevidys for non-walking patients [1].

The FDA's investigation into the death of the 8-year-old boy comes amid a broader review of Sarepta's gene therapies. The agency has placed Sarepta's investigational gene therapy clinical trials for limb-girdle muscular dystrophy on clinical hold and is reviewing the safety of Elevidys. Sarepta has denied the FDA's request to halt all shipments of Elevidys, stating that it will continue shipping the drug for younger Duchenne patients who can still walk [2].

The recent events have raised questions about the safety of Sarepta's gene therapies and the company's transparency in reporting adverse events. The FDA's investigation is expected to provide more clarity on the safety of Elevidys and the broader implications for the gene therapy field.

References:

[1] Luke Taylor. (2025). Sarepta Therapeutics suspends Elevidys treatment for Duchenne muscular dystrophy. BMJ. Retrieved from https://www.bmj.com/content/390/bmj.r1548

[2] Reuters. (2025). Sarepta Therapeutics' SRPT tanks 36% after third patient death. TradingView. Retrieved from https://www.tradingview.com/news/reuters.com,2025-07-25:newsml_NFC8nBvkT:0-sarepta-therapeutics-srpt-tanks-36-after-3d-patient-death-srpt-securities-class-action-pending-investors-with-substantial-losses-and-persons-with-knowledge-encouraged-to-contact-hagens-berman/