Home / Health / FDA Approves Sarepta's Elevidys with New Safety Warnings for DMD Treatment
FDA Approves Sarepta's Elevidys with New Safety Warnings for DMD Treatment
17 Nov, 2025
Summary
- FDA approves updated prescribing information for Elevidys gene therapy for Duchenne muscular dystrophy
- Elevidys label now carries a boxed warning for acute liver failure and injury risk
- Sarepta plans to study enhanced immunosuppression to resume dosing for non-ambulatory DMD patients
The US Food and Drug Administration (FDA) has approved updated prescribing information for Sarepta's Elevidys, an adeno-associated virus (AAV)-based gene transfer therapy for Duchenne muscular dystrophy (DMD). The updated Elevidys label now carries a boxed warning regarding the risk of acute liver failure and injury.
Additionally, the indication and usage section no longer includes non-ambulatory patients. Sarepta plans to begin a study of an enhanced sirolimus immunosuppressive regimen to address these risks, aiming to resume dosing for non-ambulatory patients with FDA approval. The updated label also includes expanded prescriber guidance on a modified oral corticosteroid regimen and weekly monitoring for three months following treatment. A new warning has been added about increased susceptibility to serious infections due to immunosuppression.
Sarepta's research and development and technical operations president, Louise Rodino-Klapac, stated that the company wants to thank the FDA for their thorough and collaborative review, as the safety labeling changes will ensure families and healthcare professionals have clear information to guide treatment decisions. Elevidys has so far been administered to more than 1,100 patients worldwide, in both clinical trials and real-world environments.
