Elevidys Label Updated to Reflect Risk of Serious Liver Injury and Acute Liver Disease

Following an investigation of reports of fatal liver failure, the Food and Drug Administration (FDA) has approved updated prescribing information for treatment with Elevidys (delandistrogene moxeparvovec-rokl; Sarepta Therapeutics, Cambridge, MA) for people with Duchenne Muscular Dystrophy (DMD). The revised prescribing information includes a Boxed Warning for risk of serious liver injury and acute liver failure including fatal outcomes, as well as an updated indication, limiting treatment to ambulatory individuals with DMD aged 4 years and older.

In the summer of 2025, the FDA announced that it was investigating 2 cases of fatal liver failure that were reported in male, nonambulatory, pediatric patients with DMD who were treated with Elevidys. In both cases, patients exhibited elevated transaminase levels and were hospitalized prior to death. Sarepta Therapeutics paused the distribution of Elevidys for treatment of nonambulatory patients in response, according to a statement released by the FDA.

The newly announced changes to the labeling of Elevidys include the following:

• The addition of a Boxed Warning for acute serious liver injury and acute liver failure.
• An updated indication: “ELEVIDYS is indicated for the treatment of patients 4 years of age and older with Duchenne muscular dystrophy (DMD), who are ambulatory and have a confirmed mutation in the DMD gene.”
• The removal of the prior indication for treatment of nonambulatory individuals.
• The addition of a Limitations of Use statement.
• The inclusion of a new Medication Guide for both patients and caregivers.
• Additional updates to sections including Warnings and Precautions, Dosage and Administration, Adverse Reactions, Use in Specific Populations, Clinical Studies, and Patient Counseling Information.

The FDA noted that health care professionals and patients should report adverse events to the FDA MedWatch program at www.fda.gov/medwatch or by calling 1-800-FDA-1088.

Source: Elevidys (delandistrogene moxeparvovec-rokl). Prescribing information. Sarepta Therapeutics, Inc., 2025. Accessed November 14. https://www.elevidys.com/pi

US Food and Drug Administration. FDA approves new safety warning and revised indication that limits use for Elevidys following reports of fatal liver injury. FDA Newsroom. Published November 14, 2025. Accessed November 14, 2025. https://www.fda.gov/news-events/press-announcements/fda-approves-new-safety-warning-and-revised-indication-limits-use-elevidys-following-reports-fatal?utm_medium=email&utm_source=govdelivery