FDA official announcements and press releases

FDA Expands Approval of Sarepta's Elevidys Gene Therapy for Duchenne Muscular Dystrophy for Ages 4 and Above

Jun 20, 2024, 09:44 PM

The U.S. Food and Drug Administration (FDA) has expanded the approval of Sarepta Therapeutics' gene therapy, Elevidys, for Duchenne muscular dystrophy (DMD) patients aged four and above. The expanded approval now includes both ambulatory and non-ambulatory patients, with the non-ambulatory indication under accelerated approval and the ambulatory indication converted to full approval. This decision allows nearly all children suffering from DMD, a rare condition that can leave infants unable to walk, to access the treatment. Elevidys targets patients with a confirmed mutation in the DMD gene.