Sarepta Stands Ground Against HHS Request To Stop Elevidys Shipments

Sarepta Therapeutics has refused the FDA's (and by extension HHS's) informal request to halt U.S. shipments of its gene therapy Elevidys, despite recent patient deaths linked to the product.125

The FDA placed Sarepta's clinical trials for limb girdle muscular dystrophy on hold, citing three deaths (from acute liver failure) potentially associated with Sarepta's AAVrh74-based gene therapies, including Elevidys, and deemed participants at unreasonable and significant risk.5

Last month, the FDA granted, but has now revoked, a new 'platform designation' for Sarepta’s AAVrh74 vector technology in response to the safety concerns.15

Shipments of Elevidys for non-ambulant patients have already been paused voluntarily by Sarepta since June 2025, but the company says it will continue to supply Elevidys to ambulant Duchenne muscular dystrophy patients, citing its own interpretation of safety data.13

Sarepta asserts it only learned of the FDA's request to halt all shipments via media reports on July 18, with formal communication from the FDA following later that afternoon.134

The FDA, in a public statement, emphasized it will not allow marketed products 'whose harms are greater than benefits' and will halt clinical trials for products presenting unreasonable safety risks.15

Sources:

1. https://www.biospace.com/business/sarepta-stands-ground-against-hhs-request-to-stop-elevidys-shipments

2. https://www.fiercepharma.com/pharma/sarepta-stands-ground-elevidys-sales-after-fda-requests-gene-therapy-be-pulled-market

3. https://www.sarepta.com/community-letter-update-regarding-elevidys

4. https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-provides-statement-elevidys

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