Sarepta's Gene Therapy for Duchenne Muscular Dystrophy Receives FDA Approval

The FDA has granted accelerated approval to Sarepta Therapeutics' gene therapy, Elevidys, for boys aged 4 and 5 with Duchenne muscular dystrophy, making it the first one-time treatment for the debilitating muscle disease. Elevidys is the first in vivo gene therapy to win accelerated approval, and it did so with limited data. The decision shows the FDA's flexibility towards granting gene therapies accelerated approval for fatal diseases with few treatment options based on biomarker data.