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Muscular Dystrophy Association Announces Expanded US FDA Approval of ELEVIDYS Gene Therapy for Duchenne Muscular Dystrophy Patients Ages 4 and Above

Milestone Expanded Approval Brings Hope for Families Living with Duchenne Muscular Dystrophy

NEW YORK, June 20, 2024 - The Muscular Dystrophy Association (MDA) announces 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. Read more in the Sarepta press release.

“The FDA’s expanded approval for ELEVIDYS marks a pivotal moment for the community, offering renewed hope and tangible progress in our fight against DMD,” said Sharon Hesterlee, PhD, Chief Research Officer, Muscular Dystrophy Association. "At the Muscular Dystrophy Association, we continue our legacy and impact with innovative scientific and clinical research to propel treatments and therapies forward for the families we serve.”

"Today's FDA approval of ELEVIDYS is a significant step forward in the fight against Duchenne muscular dystrophy. The full approval for all ambulant patients and accelerated approval for all non-ambulant patients is not only a scientific accomplishment but a major source of hope for countless families affected by this relentless disease. As the Chief Medical Advisor of Muscular Dystrophy Association, I am proud to stand with our community in celebrating this milestone. Together, we are transforming what is possible in medical research and patient care, reaffirming our commitment to improving lives and finding a cure," said Barry Byrne, MD, PhD, Chief Medical Advisor, Muscular Dystrophy Association, and Associate Chair of Pediatrics and Director of the Powell Gene Therapy Center at the University of Florida.

“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. "Furthermore, transitioning from accelerated approval to full approval for the ambulatory population is impactful as it solidifies the evidence of ELEVIDYS' clinical benefits and facilitates continued access to this critical therapy. Confirming the therapy’s effectiveness will provide greater assurance to families and healthcare providers, enhancing the long-term treatment landscape for Duchenne muscular dystrophy (DMD). While the expanded approval of ELEVIDYS marks a significant milestone, we must acknowledge the individuals who will not be included within the current label. Our commitment remains steadfast to continue advocating for inclusive research and development efforts that strive to expand access to all individuals living with DMD, ensuring no one is left behind."

“There weren’t many options when Conner was diagnosed with Duchenne muscular dystrophy nine years ago but with the MDA they’re accelerating research, and he did get gene therapy. I believe he would be in a wheelchair now full time if he hadn’t received it. Muscular Dystrophy Association was an intricate part of that process,” said Jessica Curran, MDA family member.

MDA remains steadfast in its mission to accelerate the development of treatments and cures for neuromuscular diseases. The organization recently announced expanded support for the families and clinical community through the MDA Gene Therapy Support Network, which provides guidance and support on novel gene therapy treatments, including ELEVIDYS.

MDA implemented the MDA Gene Therapy Support Network (GTxSN) to support the MDA Care Center Network and the neuromuscular (NMD) community address the challenges and achieve the promise of gene therapy. An element of the GTxSN is to establish a clinical operational readiness network to support existing and new gene therapy treatments for the NMD patient community within the MDA Care Center Network. To help evaluate and expand gene therapy access in a way that optimizes safety and long-term outcomes, MDA created a dedicated Care Center Network Information Hub (CCNH) to host educational resources and clinical best practices sharing amongst healthcare providers within the MDA Care Center Network. The CCNH fosters greater cross-clinic engagement and gene therapy best practice sharing; focus on real time expert learnings and experiences (i.e. best practices, protocols, infrastructure check lists, etc.).

With the help of the MDA GTxSN, the goal is for MDA Care Center clinicians and staff to feel that they are ready and confident to dose patients and perform post-administration monitoring of patients, minimize the number of adverse events experienced post gene therapy at MDA Care Center sites, effectively manage and schedule resources at MDA Care Centers sites to maximize the number of patients treated per month, and celebrate success stories from patients and families post-treatment.

MDA has been at the forefront of advancements in DMD research for over 70 years, investing nearly $227 million in DMD research and clinical trials. Recent collaborations with CureDuchenne and Parent Project Muscular Dystrophy have focused on strategizing approaches to reduce anti-AAV antibodies to enable safe and effective delivery of gene therapy in DMD patients. Historic contributions include discovering the DMD-causing gene in the 1980s. Recognized by The American Society for Gene and Cell Therapy, MDA has invested over $125 million in gene therapy development. The organization’s dedication earned them the Sonia Skarlatos Public Service Award in 2019 for their impactful contributions to genetic medicine.

About Muscular Dystrophy Association

Muscular Dystrophy Association (MDA) is the #1 voluntary health organization in the United States for people living with muscular dystrophy, ALS, and related neuromuscular diseases. For over 70 years, MDA has led the way in accelerating research, advancing care, and advocating for the support of our families. MDA's mission is to empower the people we serve to live longer, more independent lives. To learn more visit mda.org and follow MDA on Instagram, Facebook, X, Threads, TikTok, LinkedIn, and YouTube.