FDA recommends hold removal for Sarepta’s Elevidys for ambulatory patients

The U.S. Food and Drug Administration is recommending the removal of the voluntary hold for ambulatory patients who may now receive Elevidys, a Sarepta (SRPT) Therapeutics gene therapy for Duchenne Muscular Dystrophy. The FDA’s investigation has concluded that the death of the 8-year-old boy is 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 patient community is an important voice, and the FDA will continue to listen to and respond to thoughts from the community impacted by DMD. Elevidys is an adeno-associated virus vector-based gene therapy using Sarepta Therapeutics, Inc.’s AAVrh74 Platform Technology for the treatment of Duchenne muscular dystrophy. The product is administered as a single intravenous dose. Duchenne muscular dystrophy is a rare genetic condition characterized by progressive muscular weakness. The disease occurs due to a defective gene.