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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...

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