FDA Investigates Pediatric Elevidys Death: 5 Things to Know

Silver Spring, MD, USA - June 25, 2022: The FDA White Oak Campus, headquarters of the United States Food and Drug Administration, a federal agency of the Department of Health and Human Services (HHS). | Image Credit: © Tada Images - stock.adobe.com

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 agreed to do so on July 21 (4).

3. FDA revises drug’s DMD indication

FDA first granted Sarepta accelerated approval for Elevidys in June 2023, for ambulatory individuals four through five years of age with DMD who had a confirmed mutation in the DMD gene (5). This was expanded to traditional approval in June 2024 for both ambulatory and non-ambulatory patients aged four and up (6). However, following the deaths, FDA in July 2025 again restricted the use of the therapy to ambulatory individuals (3).

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 not alone in the industry when it comes to laying off workers in 2025. Allogene Therapeutics, Gilead Sciences, IGM Biosciences, Jasper Therapeutics, Merck & Co., Molecular Partners, Novartis, Prime Medicine, and Roche (Genentech) have also reduced their workforces in the year’s first seven months (8).

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. FDA Investigating Death of 8-Year-Old Boy Who Received Elevidys. Press Release. July 25, 2025.
2. FDA. FDA Investigating Deaths Due to Acute Liver Failure in Non-Ambulatory Duchenne Muscular Dystrophy Patients Following Elevidys. FDA.gov, June 24, 2025.
3. FDA. FDA Requests Sarepta Therapeutics Suspend Distribution of Elevidys and Places Clinical Trials on Hold for Multiple Gene Therapy Products Following 3 Deaths. Press Release. July 18, 2025.
4. Sarepta Therapeutics. Sarepta Therapeutics Announces Voluntary Pause of Elevidys Shipments in the US. Press Release. July 21, 2025.
5. FDA. FDA Approves First Gene Therapy for Treatment of Certain Patients with Duchenne Muscular Dystrophy. Press Release. June 22, 2023.
6. FDA. FDA Expands Approval of Gene Therapy for Patients with Duchenne Muscular Dystrophy. Press Release. June 20, 2024.
7. Sarepta Therapeutics. Sarepta Therapeutics Announces Strategic Restructuring and Pipeline Prioritization Plan to Maintain Long-Term, Sustainable Growth and Provides Update on Elevidys Label. Press Release. July 16, 2025.
8. Cole, C. What Sarepta’s Layoffs, and Others Across Biopharma, Mean for Industry Professionals. BioPharmInternational.com, July 17, 2025.