FDA

FDA investigates liver failure deaths linked to Elevidys in DMD patients

June 25, 2025

FDA is investigating two reported deaths due to acute liver failure in non-ambulatory pediatric patients with Duchenne muscular dystrophy (DMD) who received Elevidys (delandistrogene moxeparvovec-rokl), a gene therapy developed using an adeno-associated virus vector. The cases, identified through clinical trials and postmarketing surveillance, involved patients who developed elevated liver enzymes and were hospitalized within two months of treatment. The agency is evaluating whether Elevidys may be linked to serious liver complications, including hospitalization and death.

Although Elevidys labeling includes warnings about serious liver injury, it doesn't specifically mention liver failure or fatal outcomes. FDA is assessing whether updates to the prescribing information or other regulatory actions are needed. Healthcare providers are urged to monitor liver function closely in patients receiving Elevidys and to report any adverse events through the MedWatch program to support ongoing safety evaluations.

Source:

(2025, June 24). FDA. FDA Investigating Deaths Due to Acute Liver Failure in Non-ambulatory Duchenne Muscular Dystrophy Patients Following ELEVIDYS. https://www.fda.gov/vaccines-blood-biologics/safety-availability-biologics/fda-investigating-deaths-due-acute-liver-failure-non-ambulatory-duchenne-muscular-dystrophy-patients