Elevidys Now Approved For Pediatric Patients

FDA Expands Approval of Gene Therapy for Duchenne Muscular Dystrophy

Elevidys Now Approved for Pediatric Patients

Third Approval for Innovative Treatment

The U.S. Food and Drug Administration (FDA) has expanded its approval of Elevidys delandistrogene moxeparvovec-rokl, a gene therapy for Duchenne muscular dystrophy (DMD). The approval now includes pediatric patients 4 years of age and older with confirmed mutations in the DMD gene. Elevidys is the first gene therapy approved for DMD, a genetic disorder that causes progressive muscle weakness and loss. It works by delivering a functional copy of the dystrophin gene to the patient's muscle cells, helping to restore the production of dystrophin protein, which is essential for muscle function. The expanded approval was based on data from a clinical trial involving 39 boys with DMD who received Elevidys. The results showed that the therapy led to significant improvements in motor function and reduced the need for corticosteroids, which are commonly used to treat DMD but can have side effects. The FDA's approval is a major step forward for the treatment of DMD. It provides a new option for young patients with this debilitating disorder and offers hope for improved quality of life and longer survival.