Fierce Pharma June 21, 2024
There’s no slowing the momentum of Sarepta’s groundbreaking Duchenne muscular dystrophy (DMD) gene therapy Elevidys—not even the failure of a confirmatory trial.
On Thursday, the FDA expanded the label for Elevidys (delandistrogene moxeparvovec-rokl) to all DMD patients ages 4 and older. It’s a major boost for the first gene therapy to treat the inherited disorder, which received an accelerated approval a year ago—nearly to the date—but only for ambulatory boys ages 4 to 5.
Confirming the drug’s functional benefits, the FDA has blessed Elevidys with traditional approval for ambulatory patients while granting accelerated approval for those who are non-ambulatory. Continued approval for non-ambulatory patients may be contingent upon verification of clinical benefit in a confirmatory trial.
“The (expansion) is a...
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