FDA expands approval of gene therapy for patients with Duchenne muscular dystrophy

June 21, 2024
Duchenne muscular dystrophy is a rare and serious genetic condition which worsens over time, leading to weakness and wasting away of the body’s muscles.

The U.S. Food and Drug Administration expanded the approval of Elevidys (delandistrogene moxeparvovec-rokl), a gene therapy for the treatment of Duchenne muscular dystrophy (DMD) for ambulatory and non-ambulatory individuals 4 years of age and older with DMD with a confirmed mutation in the DMD gene. 

Elevidys 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. With the new action, Elevidys received traditional approval in ambulatory individuals 4 years of age and older with DMD with a confirmed mutation in the DMD gene, and accelerated approval in non-ambulatory individuals 4 years of age and older with DMD with a confirmed mutation in the DMD gene. In making this decision, the FDA considered the totality of the evidence, including the potential risks associated with the product, the life-threatening and debilitating nature of the disease and the urgent unmet medical need.

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 (138 kDa, compared to the 427 kDa dystrophin protein of normal muscle cells) that contains selected domains of the dystrophin protein present in normal muscle cells. The product is administered as a single intravenous dose. 

FDA release