FDA Safety Communication – June 24, 2025
Summary of the Issue
Since approval, the Food and Drug Administration (FDA) has received two reports of fatal acute liver failure following treatment of non-ambulatory pediatric male patients with Duchenne Muscular Dystrophy (DMD) with ELEVIDYS (delandistrogene moxeparvovec-rokl), an adeno-associated virus vector-based gene therapy.
The two reported deaths in non-ambulatory DMD patients appear to be related to treatment with ELEVIDYS. Reports were received from clinical trial and postmarketing data. In these two cases, the patients presented with elevated transaminases and were hospitalized less than two months after treatment with ELEVIDYS.
FDA is investigating the risk of acute liver failure with serious outcomes, including those such as hospitalization and death, following ELEVIDYS, and is evaluating the need for further regulatory action.
The U.S. prescribing information (USPI) includes information on the risk of acute serious liver injury following treatment with ELEVIDYS under Warnings and Precautions, Adverse Reactions and Patient Counseling Information, but does not include warnings regarding liver failure or death.
To report suspected adverse events including liver injury, contact the FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.
