Sarepta Therapeutics has announced significant updates to the U.S. prescribing information for ELEVIDYS, the only approved gene therapy for Duchenne muscular dystrophy (DMD). The revisions, completed in coordination with the U.S. Food and Drug Administration (FDA), introduce new safety warnings and modify recommended treatment protocols.
A key change is the addition of a boxed warning—the FDA’s strongest safety alert—highlighting the risk of acute serious liver injury (ALI) and acute liver failure (ALF). Sarepta also confirmed that the non-ambulatory indication has been removed from the approved use of ELEVIDYS. The company plans to initiate a study of an enhanced sirolimus-based immunosuppressive regimen aimed at mitigating these liver-related risks, with hopes of resuming treatment for non-ambulatory patients pending FDA agreement.
The updated label includes expanded guidance for physicians, such as adjustments to the pre- and post-infusion oral corticosteroid regimen and enhanced patient monitoring. Weekly monitoring for three months following infusion is now recommended. Additionally, a new warning has been added regarding increased vulnerability to serious infections due to immunosuppression.
Louise Rodino-Klapac, Ph.D., Sarepta’s president of research and development and technical operations, expressed appreciation for the FDA’s collaboration. “Completion of the safety labeling change for ELEVIDYS will ensure that families and healthcare professionals have clear information, supported by a Medication Guide, to help understand these updates and guide treatment decisions,” she said.
More than 1,100 patients worldwide have been treated with ELEVIDYS across clinical trials and real-world use. Sarepta emphasized its ongoing commitment to working closely with regulators to ensure patient safety while advancing therapeutic options for those living with DMD, a rare and progressive genetic disorder with life-limiting consequences.