BioPharma Dive June 20, 2024
Despite mixed results in testing, agency officials determined Elevidys showed enough to warrant a label that includes all patients at least 4 years of age and specific genetic mutations.
The Food and Drug Administration has loosened limits on the first gene therapy for Duchenne muscular dystrophy in a decision that could greatly expand its use even as questions remain about its effectiveness.
The agency on Thursday made the therapy, called Elevidys and sold by biotechnology company Sarepta Therapeutics, available to people with Duchenne who are at least four years of age and have mutations in a specific gene, regardless of whether they can still walk.
For those who can still walk, the agency also converted Elevidys’ conditional approval to full,...
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