Sarepta, bowing to FDA pressure, pauses shipments of Duchenne gene therapy Elevidys

Sarepta Therapeutics has announced it will pause all shipments of its Duchenne muscular dystrophy gene therapy, Elevidys, in the United States, effective at the end of business on July 22, 2025134.

This decision follows a request from the FDA, made after multiple patient deaths, including two involving Elevidys and a third related to a separate Sarepta investigational therapy for limb-girdle muscular dystrophy (LGMD)15.

Sarepta initially declined to comply with the FDA's request, stating there were no new safety signals and citing its own scientific interpretation of safety data23.

The company reversed its position, emphasizing the importance of maintaining a positive regulatory relationship and addressing FDA questions while completing the Elevidys label supplement process123.

Sarepta has committed to working collaboratively with the FDA during this pause and will respond to the agency’s information requests23.

Sources:

1. https://www.fiercepharma.com/pharma/sarepta-getting-back-fdas-good-side-pauses-shipments-duchenne-gene-therapy-elevidys

2. https://www.clinicaltrialsarena.com/news/sarepta-halts-elevidys-shipments-following-fda-pressure/

3. https://www.cgtlive.com/view/sarepta-voluntarily-pauses-shipments-dmd-gene-therapy-elevidys-several-days-informal-request-fda

4. https://www.statnews.com/2025/07/21/sarepta-will-stop-shipping-elevidys-fda-duchenne-muscular-dystrophy/

5. https://www.fda.gov/news-events/press-announcements/fda-requests-sarepta-therapeutics-suspend-distribution-elevidys-and-places-clinical-trials-hold