FDA Threatens Action After Sarepta Refuses to Halt Elevidys Shipments

The FDA requested Sarepta Therapeutics immediately suspend all shipments of Elevidys, a gene therapy for Duchenne muscular dystrophy, after three reported patient deaths from liver failure134.

Sarepta refused the FDA's request and stated it would pause shipments only for patients confined to wheelchairs, not for ambulatory patients, citing no new or changed safety signals in that group1.

The FDA has placed Sarepta’s clinical trials on hold for limb-girdle muscular dystrophy and revoked the company’s platform technology designation due to safety concerns34.

FDA Commissioner Marty Makary stated the agency would take swift action to protect patient safety and is considering exercising its full regulatory authority, including possible market withdrawal of Elevidys123.

Sarepta's stock fell sharply (more than 35%) following the news, with broader impacts on the biotech index2.

This public standoff marks a significant escalation in regulatory scrutiny over gene therapies, testing the FDA's willingness to pull drugs from the market amid emerging safety signals13.

Sources:

1. https://www.axios.com/2025/07/21/fda-dispute-gene-therapy-pharma

2. https://www.fiercepharma.com/pharma/fda-mulls-elevidys-market-withdrawal-following-3rd-death-after-sarepta-gene-therapy

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

4. https://www.neurologylive.com/view/third-patient-death-leads-significant-concerns-sarepta-gene-therapy-program