FDA Removes Voluntary Hold on Ambulatory Patients Taking Elevidys

The FDA is recommending the “removal of the voluntary hold for ambulatory patients who may now receive Elevidys, a Sarepta Therapeutics gene therapy for Duchenne Muscular Dystrophy (DMD).”

Previously, the FDA had placed a hold on use of the gene therapy because of the death of an eight-year-old boy who was using it. However, the agency has concluded that his death was “unrelated to the gene therapy product itself.”

The FDA will “continue to work with the sponsor regarding non ambulatory patients, which remains subject to a voluntary hold, following two deaths.” The agency is also committed to responding to thoughts from “the community impacted by DMD.”

Elevidys is an “adeno-associated virus vector-based gene therapy” administered by single intravenous dose. Duchenne muscular dystrophy is “a rare genetic condition characterized by progressive muscular weakness. The disease occurs due to a defective gene.”