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18 Nov 2025
Admin
News Article

Sarepta Updates Prescribing Information for Elevidys Gene Therapy for Duchenne Muscular Dystrophy

Sarepta Therapeutics has announced updated prescribing information for Elevidys (delandistrogene moxeparvovec-rokl), the only approved gene therapy for Duchenne muscular dystrophy (DMD). The revisions follow a regulatory review by the U.S. Food and Drug Administration.

Key Changes to the Elevidys Label

The updated label includes several significant modifications:

Boxed Warning Added

A boxed warning has been added highlighting the risk of acute serious liver injury (ALI) and acute liver failure (ALF).

Non-Ambulatory Indication Removed

The indication for non-ambulatory patients has been removed. Sarepta plans to begin a study evaluating an enhanced sirolimus-based immunosuppressive regimen to mitigate liver-related risks. The company aims to resume dosing in non-ambulatory patients pending FDA concurrence.

Expanded Prescriber Guidance

The updated label provides:

A modified pre- and post-infusion corticosteroid regimen
Strengthened monitoring recommendations, including weekly assessments for the first three months after infusion

New Warning on Infection Risk

A new precaution notes an increased susceptibility to serious infections due to immunosuppression.

Company Statement

Louise Rodino-Klapac, Ph.D., president of research & development and technical operations, said the updated safety information will help families and clinicians make informed treatment decisions. She thanked the FDA for its collaborative review.

Real-World Use and Ongoing Collaboration

More than 1,100 patients worldwide have received Elevidys in clinical trials and real-world settings. Sarepta said it remains in close communication with the FDA to ensure that regulatory actions reflect scientific evidence and patient needs.

About Elevidys

Elevidys is a single-dose AAV-based gene transfer therapy delivered via intravenous infusion. It is designed to address mutations in the DMD gene, which prevent the production of dystrophin. The therapy delivers a transgene enabling production of Elevidys micro-dystrophin in skeletal muscle.

Current Approved Indication

Elevidys is approved for ambulatory patients aged 4 years and older with a confirmed DMD gene mutation.

Patients for Whom Elevidys Is Not Recommended

Elevidys is not advised for patients with:

Preexisting liver impairment or active hepatic viral infection
Recent vaccination within four weeks prior to therapy
Active or recent infections, also within four weeks

Sarepta’s Broader Mission

Sarepta continues to focus on developing precision genetic medicines for rare diseases, particularly those affecting muscle, the central nervous system, and the heart.

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