FDA Official Says Sarepta Faces Major Hurdles to Reintroduce DMD Therapy Elevidys
Sarepta Therapeutics has voluntarily paused all shipments of its Duchenne muscular dystrophy therapy Elevidys in the US as of July 22, 2025, while it works with the FDA on safety-related labeling updates and responds to agency information requests.14
A senior FDA official stated the agency's Center for Biologics Evaluation and Research (CBER) is unanimously against allowing Elevidys back onto the market without further evidence of safety and efficacy, following concerns over serious liver injury and deaths linked to the therapy.23
The FDA is reportedly considering requiring Sarepta to conduct additional studies or clinical trials to validate the safety profile of Elevidys before reinstatement, with potential changes in dosing, manufacturing, or additional testing in more patients.23
The FDA's move follows controversy over Elevidys' approval in June 2024, which was granted by the CBER director despite failed Phase III trial results and internal reviewer opposition.2
According to media reports, the FDA is reviewing all possible regulatory actions in handling Elevidys’ status, indicating Sarepta faces major regulatory and legal challenges to reintroduce the therapy.34
Sources:
1. https://www.towardshealthcare.com/blog/sarepta-elevidys-shipment-pause-2025
2. https://www.biospace.com/fda/sareptas-future-increasingly-uncertain-as-fda-eyes-new-study-for-elevidys
3. https://www.fiercepharma.com/pharma/fda-weighs-new-study-requirements-sarepta-confirm-safety-gene-therapy-elevidys-report