Elevidys FDA Alerts
The FDA Alerts below may be specifically about Elevidys or relate to a group or class of drugs which include Elevidys.
MedWatch Safety Alerts are distributed by the FDA and published by Drugs.com. Following is a list of possible medication recalls, market withdrawals, alerts and warnings.
Recent FDA Alerts for Elevidys
FDA Investigating Deaths Due to Acute Liver Failure Following Treatment with Sarepta’s AAVrh74 Gene Therapies
As of July 18, 2025, the Food and Drug Administration (FDA) has received three reports of fatal acute liver failure following treatment of patients with Sarepta AAVrh74 gene therapies that appear to have been caused by the gene therapy products as a result of acute liver failure.
Two of these deaths were reported in non-ambulatory pediatric male patients with Duchenne Muscular Dystrophy (DMD) following treatment with ELEVIDYS (delandistrogene moxeparvovec-rokl), and previously described in our June 24, 2025 communication: FDA Investigating Deaths Due to Acute Liver Failure in Non-ambulatory Duchenne Muscular Dystrophy Patients Following ELEVIDYS. Elevidys is an AAVrh74 adeno-associated virus vector-based gene therapy.
A third death was reported in an adult male patient with Limb Girdle Muscular Dystrophy following treatment with a Sarepta AAVrh74 gene therapy product in a clinical trial, who presented with abnormal liver function tests. This patient presented with elevated transaminases and was hospitalized less than two months after treatment with the investigational gene therapy product. The patient progressed to liver failure and died 80 days after he received the investigational gene therapy.
FDA is investigating the risk of acute liver failure with serious outcomes, including those such as hospitalization and death, following gene therapies using Sarepta’s AAVrh74 gene therapy products, and the need for further regulatory action(s). More information describing FDA’s regulatory actions is available here: FDA Requests Sarepta Therapeutics Suspend Distribution of Elevidys and Places Clinical Trials on Hold for Multiple Gene Therapy Products Following 3 Deaths
To report suspected adverse events including liver injury, contact the FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.
Drug Safety Communication: FDA Investigating Deaths Due to Acute Liver Failure in Non-Ambulatory Duchenne Muscular Dystrophy Patients Following Elevidys
FDA Safety Communication – June 24, 2025 -- 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.
Source: FDA