What is the FDA's decision on Sarepta's Elevidys gene therapy?

The FDA has approved updated prescribing information for Sarepta's Elevidys gene therapy for Duchenne muscular dystrophy (DMD).

What are the key changes to the Elevidys label?

The updated Elevidys label now carries a boxed warning regarding the risk of acute liver failure and injury, and no longer includes non-ambulatory patients in the indication and usage section.

What is Sarepta's plan to address the safety concerns with Elevidys?

Sarepta plans to begin a study of an enhanced sirolimus immunosuppressive regimen to address the risks, aiming to resume dosing for non-ambulatory DMD patients with FDA approval.

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

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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

FDA Approves Sarepta's Elevidys with New Safety Warnings for DMD Treatment

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.