Muscular Dystrophy Association applauds expanded FDA approval of ELEVIDYS Gene Therapy for Duchenne Muscular Dystrophy

Monday, July 1, 2024

The Muscular Dystrophy Association (MDA) celebrates the U.S. Food and Drug Administration’s expanded approval of ELEVIDYS (delandistrogene moxeparvovec-rokl). 

The efficacy supplement approval will expand the labeled indication of ELEVIDYS to include boys and men with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene who are at least 4 years of age.

The FDA granted traditional approval for ambulatory patients and accelerated approval for non-ambulatory patients, confirming the functional benefits of the treatment.

Developed by Sarepta Therapeutics, ELEVIDYS targets the genetic root cause of Duchenne muscular dystrophy (DMD) and works by introducing an engineered gene that codes for a shortened version of dystrophin, referred to as ELEVIDYS micro-dystrophin into muscle cells.

The FDA granted accelerated approval for boys ages 4 and 5 in June of 2023.

“By expanding the label beyond current age groups, the FDA is exercising flexibility in its approach to a rare disease with high unmet need. 
"Many older boys and young men urgently need access to new therapies that could alter the disease's progression. 
"By broadening the indication, hope can be offered to more families and improve the quality of life for a larger segment of the DMD community”, said Paul Melmeyer, EVP, Public Policy and Advocacy, MDA.


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