, /PRNewswire/ -- , a nonprofit organization leading the fight to end , applauds the U.S. Food and Drug Administration (FDA) for its decision to expand the labeled indication for ELEVIDYS, a micro-dystrophin gene therapy, to include individuals at least 4 years of age and remove ambulation restrictions, marking a significant milestone in the Duchenne treatment landscape.
Additionally, the FDA has converted the ELEVIDYS accelerated approval to a traditional approval for ambulatory patients. The indication in non-ambulatory patients is under the accelerated approval pathway and will require confirmatory studies to convert to traditional approval. The ELEVIDYS label now states that the adeno-associated virus (AAV) vector-based gene therapy is indicated in individuals at least 4 years of age: "For the treatment of Duchenne muscular dystrophy (DMD) in patients who are ambulatory and have a confirmed mutation in the gene," as well as, "For the treatment of DMD in patients who are non-ambulatory and have a confirmed mutation in the gene," expanding the label previously approved and filed with the FDA through Accelerated Approval in .
This expanded indication offers hope and opportunity for a broader spectrum of individuals living with Duchenne and their families. "This decision by the FDA represents a monumental leap forward in our collective efforts to end Duchenne muscular dystrophy," said PPMD Founding President and CEO . "By expanding the label's age inclusion and removing ambul.