FDA Adds Boxed Warning for Sarepta’s DMD Gene Therapy

The Food and Drug Administration recently updated the labeling for delandistrogene moxeparvovec-rokl (Elevidys), Sarepta’s Duchenne muscular dystrophy (DMD) gene therapy, to include a boxed warning for the risk of serious liver injury and acute liver failure. The agency also limited the therapy’s indication to ambulatory patients aged 4 years and older with a confirmed DMD gene mutation. The new labeling removes the previous indication for non-ambulatory patients.

The action follows FDA’s investigation of two reports of fatal acute liver failure in non-ambulatory pediatric males with DMD after receiving delandistrogene moxeparvovec-rokl. In both cases, patients experienced sharply elevated liver enzymes and required hospitalization within two months of infusion.

Following its safety review, FDA also approved additional revisions to the label, including updated monitoring recommendations and a new limitations-of-use statement and a medication guide. The agency also required a postmarketing observational study to further assess the risk of serious liver injury.