Elevidys Receives Expanded Indication for Duchenne Muscular Dystrophy

The Food and Drug Administration (FDA) has broadened the approval for Elevidys (delandistrogene moxeparvovec-rokl; Sarepta Therapeutics, Cambridge, MA) as a treatment for patients diagnosed with Duchenne muscular dystrophy (DMD) aged ≥4 years with a confirmed mutation in the DMD gene, granting traditional approval for ambulatory patients and accelerated approval for non-ambulatory patients. Elevidys is a recombinant gene therapy injected as a single intravenous dose causing cells to produce a shortened protein called Elevidys micro-dystrophin which contains domains of the dystrophin protein found in healthy muscle cells.

This expanded approval builds upon the previous FDA accelerated approval for ambulatory DMD patients, which was based on data from a phase 1 clinical trial called ENDEAVOR (NCT04626674), a phase 1/2 clinical trial (NCT03375164), and a phase 2 clinical trial (NCT03769116). Continued approval for non-ambulatory patients is contingent on confirmatory trial results from the ongoing global, randomized, double-blind, placebo-controlled phase 3 ENVISION clinical trial (NCT05881408).

“Representing many years of dedicated research, development, investment and creative energy, the expansion of the Elevidys label to treat Duchenne patients aged 4 and above, regardless of ambulatory status, is a defining moment for the Duchenne community. Today also stands as a watershed occasion for the promise of gene therapy and a win for science,” said Doug Ingram, President and CEO of Sarepta.