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Sarepta Obtains Broader DMD Label For Elevidys

 
• By Joseph Haas

The gene therapy is now approved for all ambulatory boys with Duchenne muscular dystrophy ages 4 and older. The revised label also includes accelerated approval for non-ambulatory patients.

Expanded
Expanded label for Elevidys covers all ambulatory DMD patients 4 and older • Source: Shutterstock

Sarepta Therapeutics, Inc. obtained expanded US Food and Drug Administration approval of its Duchenne muscular dystrophy gene therapy Elevidys in ambulatory boys aged 4 and older, overcoming concerns after a confirmatory trial missed its primary endpoint last October. The 20 June FDA decision, which built on the June 2023 accelerated approval that cleared the drug for ambulatory boys aged 4-5, also provides a new accelerated approval for Elevidys to treat non-ambulatory boys ages 4 and older.

In announcing the label expansion by both age and ambulatory status, the FDA’s Center for Biologics Evaluation and Research said that while the Phase III EMBARK study failed to meet...

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