FDA Approves First Gene Therapy for Duchenne Muscular Dystrophy

The FDA has approved Elevidys, the first gene therapy for the treatment of pediatric patients aged 4 to 5 years with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene who do not have a pre-existing medical reason preventing treatment with this therapy. DMD is a rare and serious genetic condition that leads to weakness and wasting away of the body’s muscles. Elevidys is a recombinant gene therapy designed to deliver into the body a gene that leads to production of Elevidys micro-dystrophin, a shortened protein that contains selected domains of the dystrophin protein present in normal muscle cells. The product is administered as a single intravenous dose.