Gene Therapy for Duchenne Muscular Dystrophy Approved by FDA

FDA announced on June 22, 2023 the approval of Elevidys (delandistrogene moxeparvovec-rokl), the first gene therapy for the treatment of children ages four through five years that have Duchenne muscular dystrophy (DMD) and do not have a pre-existing medical reason that would prevent this kind of treatment. DMD is a rare genetic condition that leads to weakness and “wasting away” of the body’s muscles due to a defective gene, which creates an absence of a protein that keeps muscles intact.

Elevidys was approved through FDA’s accelerated approval pathway based on an evaluation of sponsor-submitted data. According to the agency, a randomized clinical trial showed Elevidys increased the expression of the Elvidys micro-dystrophin protein in children ages four to five years with DMD “who do not have significant pre-existing antibody titers against the AAV [adeno-associated virus] rh74 vector or have other contraindications based on the inclusion criteria of the clinical trials.” The approval was granted to Sarepta Therapeutics.

“Today’s approval addresses an urgent unmet medical need and is an important advancement in the treatment of Duchenne muscular dystrophy, a devastating condition with limited treatment options that leads to a progressive deterioration of an individual’s health over time,” said Peter Marks, MD, PhD, director of FDA’s Center for Biologics Evaluation and Research. “The FDA remains committed to facilitating the development of innovative new therapies to reduce the impact of debilitating diseases and to improve outcomes and quality of life for those affected.”

Source: FDA