
FDA Investigates Pediatric Elevidys Death: 5 Things to Know
While little information is known about this particular case, its possible implications are far-reaching.
FDA announced on July 25, 2025 that the agency is investigating the death of an eight-year-old boy with Duchenne muscular dystrophy (DMD) who the agency said was treated with delandistrogene moxeparvovec, the Sarepta Therapeutics gene therapy marketed as Elevidys (1). In view of Sarepta’s recent actions related to other fatalities and resultant safety concerns involving Elevidys, the following are five key points about the status of both the therapy and the company, followed by a look ahead at the news’ potential impact on industry.
1. Two pediatric deaths previously known
From the information provided in a July 25, 2025 press release, it is unclear whether the death of this eight-year-old, which occurred on June 7, is one of the two pediatric deaths previously reported by FDA in a safety communication issued June 24 (1,2).
2. FDA action prompted by three fatalities
On July 18, reports of three deaths—the two male pediatric patients and a third which occurred during a clinical trial for an investigational new drug application for treatment of limb girdle muscular dystrophy (LGMD)—prompted FDA to pause all Sarepta trials for LGMD and ask the company to stop shipping Elevidys within the United States (3). After an initial refusal, Sarepta
3. FDA revises drug’s DMD indication
FDA
4. Sarepta layoffs preluded Elevidys fallout
Two days before FDA’s request was made, and five days before the company eventually agreed to it, Sarepta announced the reduction of 36% of its workforce, approximately 500 employees (7). Sarepta also said it intended to refocus its pipeline on a potentially best-in-class small interfering RNA platform while de-emphasizing most of its gene therapies in development for LGMD.
5. Plenty of companies downsizing
Although the layoffs at Sarepta dovetailed with the recent headlines about Elevidys, the company is
Short- and long-term implications
Industry professionals keeping abreast of the Elevidys developments are likely to be keeping the following safeguards front of mind:
- Drug development teams may need to prioritize long-term safety monitoring and early risk detection in trial design.
- Regulatory scrutiny of gene and cell therapies is likely to increase, potentially extending approval timelines and requiring more extensive data.
- Investment may shift toward therapeutic platforms with more predictable safety profiles, such as RNA-based technologies.
- Manufacturers can expect heightened oversight of production consistency and post-market pharmacovigilance systems.
- Companies may reevaluate commercialization strategies and strengthen contingency planning to mitigate regulatory disruptions.
- Cross-functional coordination between R&D, regulatory, and supply chain teams will become increasingly critical to ensure compliance and continuity.
Pharmaceutical Technology® Group has reached out to the US Health and Human Services Office of the Assistant Secretary for Public Affairs, which is handling media requests on FDA’s behalf for this matter, and this story will be updated with any further information or comment.
References
1. FDA.
2. FDA.
3. FDA.
4. Sarepta Therapeutics.
5. FDA.
6. FDA.
7. Sarepta Therapeutics.
8. Cole, C.
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