FDA Imposes Boxed Warning and Restricts Use of Sarepta’s Elevidys Gene Therapy for Duchenne Muscular Dystrophy

The FDA has added a Boxed Warning (the strongest safety warning) to the labeling of Elevidys, Sarepta's gene therapy for Duchenne muscular dystrophy, due to the risk of serious liver injury and acute liver failure, including fatal cases523.

The therapy's approved use is now limited only to ambulatory patients aged 4 years and older with Duchenne muscular dystrophy and a confirmed mutation in the DMD gene; non-ambulatory patients are excluded5239.

These safety label changes follow reports of fatal acute liver failure in non-ambulatory pediatric patients who received Elevidys235.

The updated label also includes stronger clinical guidance such as modified corticosteroid use, enhanced monitoring of liver function for 3 months post-infusion, and warnings about increased infection risk3.

Elevidys should not be used in patients with deletions involving DMD exons 8 and/or 9, those with pre-existing liver impairment, recent vaccinations, or active infections2.

The FDA is requiring Sarepta to conduct a post-marketing observational study enrolling approximately 200 Duchenne patients to further assess risks of serious liver injury, with at least 12 months of monitoring after treatment123.

Sources:

1. https://www.parentprojectmd.org/fda-approves-new-safety-warning-and-revised-indication-for-elevidys/

2. https://globalgenes.org/raredaily/fda-approves-new-safety-warning-and-revised-indication-for-sareptas-dmd-gene-therapy/

3. https://cureduchenne.org/research/fda-approves-new-safety-warning-and-updated-prescribing-information-for-sareptas-elevidys/

5. https://www.fda.gov/news-events/press-announcements/fda-approves-new-safety-warning-and-revised-indication-limits-use-elevidys-following-reports-fatal

9. https://www.fda.gov/vaccines-blood-biologics/tissue-tissue-products/elevidys