FDA Investigates Deaths in Pediatric Patients Following Elevidys Treatment for DMD

The Food and Drug Administration (FDA) is investigating 2 cases of fatal acute liver failure that have been reported in male nonambulatory pediatric patients with Duchenne muscular dystrophy (DMD) who were treated with Elevidys (delandistrogene moxeparvovec-rokl; Sarepta Therapeutics, Cambridge, MA). The deaths occurred within 2 months of administration, were reported through clinical trial and postmarketing surveillance data, and according to a communication published by the FDA, appear to be related to treatment with Elevidys. Elevidys is an adeno-associated virus (AAV) vector-based gene therapy approved for the treatment of pediatric patients with DMD in 2023.

In both cases of fatal acute liver failure, patients exhibited elevated transaminase levels and were hospitalized prior to death. Although the current prescribing information for Elevidys includes warnings and precautions about acute serious liver injury, it does not explicitly mention liver failure or fatal outcomes, and the label does not include a boxed warning. The FDA has not yet issued new prescribing restrictions, but in the published communication, the organization has stated that it is evaluating whether additional regulatory action is necessary.

Elevidys, developed as a targeted therapy to deliver a shortened form of the dystrophin gene, is currently approved for use in ambulatory and nonambulatory pediatric patients with DMD, although the recent deaths were reported exclusively in the nonambulatory group.

The FDA urges clinicians to monitor liver function closely in patients receiving Elevidys—particularly in the first 2 months posttreatment—and to report suspected adverse events, including hepatotoxicity, to the FDA via the MedWatch program at www.fda.gov/medwatch or by calling 1-800-FDA-1088.