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
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.
