Sarepta Therapeutics gene therapy Elevidys, initially failed its confirmatory test but has now converted its accelerated FDA approval to a traditional one. The therapy can now be used for all Duchenne muscular dystrophy patients above the age of 4. The therapy works by producing micro-dystrophin to address the deficiency of dystrophin in Duchenne patients. While the therapy did not meet the main goal of its Phase 3 trial, it showed clinically meaningful results in secondary endpoints. The FDA approved the therapy despite concerns from agency staff, citing evidence supporting its effectiveness for Duchenne patients.
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FDA Approves Sarepta Gene Therapy, But Only After Top FDA Official Bucks Agency Staff
