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.
Sarepta Obtains Broader DMD Label For Elevidys
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.
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Pipeline Watch is a weekly snapshot of selected late-stage clinical trial events and approvals announced by pharmaceutical and biotech companies at medical and industry conferences, in financial and company presentations, and in company releases and statements.
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