FDA Expands Approval of Gene Therapy for Patients with DMD

June 24, 2024

The Food and Drug Administration recently expanded approval for delandistrogene moxeparvovec-rokl, (Elevidys, Sarepta Therapeutics Inc.), a gene therapy approved to treat Duchenne muscular dystrophy (DMD). The expansion applies to ambulatory and non-ambulatory individuals 4 years of age and older with a confirmed mutation in the DMD gene. The therapy was previously approved under accelerated approval for ambulatory individuals 4 through 5 years of age with DMD with a confirmed mutation in the DMD gene.