Sarepta has received US Food and Drug Administration (FDA) approval for updated prescribing information for Elevidys (delandistrogene moxeparvovec), an adeno-associated virus (AAV)-based gene transfer therapy, which addresses the specific genetic causes of Duchenne muscular dystrophy (DMD) in certain patient groups.
The updated Elevidys label now carries a boxed warning regarding the risk of acute liver failure and injury.
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It also no longer includes non-ambulatory patients in the indication and usage section.
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.
Expanded prescriber guidance recommends a modified oral corticosteroid regimen before and after infusion, along with weekly monitoring for three months following treatment.
A new warning and precaution has also been added about increased susceptibility to serious infections owing to immunosuppression.
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By GlobalDataSarepta research and development and technical operations president Louise Rodino-Klapac stated: “We want to thank the FDA for their thorough and collaborative review. Completion of the safety labelling 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.”
Elevidys has so far been administered to more than 1,100 patients worldwide, in both clinical trials and real-world environments.
It has been approved for use in ambulatory patients aged four years and older who have confirmed mutations in the DMD gene.
In May 2025, Sarepta Therapeutics announced new outcomes from its open-label Phase Ib Study 9001-103, also referred to as the ENDEAVOR trial, of Elevidys to treat DMD.
Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.
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