FDA

FDA adds boxed warning to Elevidys, limits use to ambulatory DMD patients

November 17, 2025

FDA added a boxed warning to Elevidys and restricted its indication to ambulatory Duchenne muscular dystrophy (DMD) patients ≥4 years, removing approval for non-ambulatory use after two pediatric deaths and another serious liver injury occurred within two months of infusion. New label guidance mandates weekly liver tests for 3 months post-infusion, cardiac monitoring for 1 month, proximity to medical facilities, and revised corticosteroid instructions. Contraindications include DMD exon 8/9 deletions, liver impairment, recent vaccination or infection. FDA is requiring the manufacturer to conduct a postmarketing observational study to further assess the risk of serious liver injury. The study will enroll approximately 200 patients with DMD and follow them for at least 12 months after administration of Elevidys, with periodic liver function assessments.

Source:

(2025, November 14). FDA. FDA Approves New Safety Warning and Revised Indication that Limits Use for Elevidys Following Reports of Fatal Liver Injury. https://www.fda.gov/news-events/press-announcements/fda-approves-new-safety-warning-and-revised-indication-limits-use-elevidys-following-reports-fatal