Muscular Dystrophy Association Celebrates FDA Approval of Sarepta Therapeutics’ ELEVIDYS for Treatment of Duchenne Muscular Dystrophy | Muscular Dystrophy Association

Muscular Dystrophy Association Celebrates FDA Approval of Sarepta Therapeutics’ ELEVIDYS for Treatment of Duchenne Muscular Dystrophy

  • The FDA approval of the first gene therapy for Duchenne muscular dystrophy (DMD) by Sarepta Therapeutics is groundbreaking and designed to target the underlying cause of the disease.
  • The Muscular Dystrophy Association (MDA) has been around since the inception of gene therapy and recently created an MDA Gene Therapy Support Network (GTx) to provide resources and guidance to clinicians and families.
  • In the past six years, four targeted drugs have been approved by the FDA for the treatment of DMD: Exondys 51, Vyondys 53 and Amondys 45 from Sarepta Therapeutics and Viltepso from NS Pharma.

NEW YORK, June 22, 2023 — The Muscular Dystrophy Association (MDA) celebrates the accelerated approval by the U.S. Food and Drug Administration (FDA) of ELEVIDYS (delandistrogen moxeparvovec-rokl) for the treatment of pediatric outpatients aged between 4 and 5 year olds with Duchenne muscular dystrophy (DMD) with a confirmed mutation in dmd extension gene. ELEVIDYS is the first gene therapy for DMD and is designed to target the underlying cause of the disease. ELEVIDYS will be made available and marketed in the United States by Sarepta Therapeutics.

Image of a DNA strand with the words, Breaking News, New FDA Drug Approval for Duchenne Muscular Dystrophy (DMD).  The first gene therapy for DMD.
The Muscular Dystrophy Association celebrates the FDA approval of Sarepta Therapeutics ELEVIDYS for the treatment of Duchenne muscular dystrophy.

DMD is caused by mutations in the dystrophin (DMD) gene on the X chromosome that result in little or no production of dystrophin, a protein essential for keeping muscle cells intact. People with DMD experience progressive muscle wasting and weakness. Over time, DMD becomes fatal as muscle wasting disrupts lung and heart function.

In the past six years, four targeted drugs have been approved by the FDA for the treatment of DMD: Exondys 51, Vyondys 53 and Amondys 45 from Sarepta Therapeutics and Viltepso from NS Pharma. These drugs use a technology called exon-skipping that allows the body to make more of the protein dystrophin. Exon skipping therapies require regular intravenous infusions. ELEVIDYS is a different type of technology known as gene replacement therapy that requires only one administration to be effective. ELEVIDYS uses an adeno-associated virus (AAVrh74) to introduce a shortened version of the dystrophin gene (mini-dystrophin) into the muscle tissue of boys with DMD, partially compensating for their lack of a functional dystrophin gene and addressing the underlying genetic defect that cause DMD.

It’s really happening. We went from hope to actually seeing an amazing gene therapy. Being able to buy 5 to 10 years for Conner is, especially for Duchenne, a miracle, he said Christopher Curran, whose 12-year-old son, Conner, was diagnosed with DMD at age 4.

This is an exciting time as more gene therapies for neuromuscular diseases enter clinical translation. The approval of ELEVIDYS is groundbreaking as it represents the first gene therapy for DMD and is designed to target the underlying cause of the disease, he said Sharon Hesterlee, Ph.D., Chief Research Officer, MDA. MDA has been around since the inception of gene therapy, and we recently established a Gene Therapy Support Network to provide resources and guidance to clinicians and families as more gene therapies enter the market.

MDA recently announced expanded support to all the families the organization serves through the new MDA Gene Therapy Support Network (GTx) which offers support and guidance on newly approved gene therapies such as ELEVIDYS.

The approval of a gene transfer therapy for DMD is a very exciting and promising development for the DMD community. I look forward to the completion of the Phase III randomized controlled trial and the results of other gene transfer therapies in the pipeline, so this could be the first of many steps toward improving the lives of boys with DMD, he said. Craig M. Zaidman, MD, Washington University in St. Louis.

MDA has made substantial investments to enable the current approach to gene therapy for neuromuscular diseases over the past two decades, he said Barry Byrne, MD, Ph.D., Chief Medical Advisor, MDA and Associate Chair of Pediatrics and Director of the Powell Gene Therapy Center at the University of Florida. We now have many options for directly treating the cause of DMD by partially restoring dystrophin function in muscle cells. I am thrilled to see the benefits of these developments for boys with Duchenne and enable them to enjoy the most fulfilling childhood activities.

We are proud to celebrate the approval of ELEVIDYS as the first gene therapy for people with DMD Paul Melmeyer, vice chair, public policy and advocacy, MDA. Developing new treatments, especially first-rate treatments such as ELEVIDYS, in DMD is incredibly difficult as DMD progresses slowly and heterogeneously, thus making clinical trials of new treatment options very challenging. We are actively working with the FDA to seek ways to expedite the approval of life-changing therapies and hope that ELEVIDYS will soon make a difference in the lives of patients and their families.

Learn about Duchenne muscular dystrophy

Duchenne muscular dystrophy (DMD) is a rare, progressive genetic muscle disease caused by a lack of a critical membrane-stabilizing protein called dystrophin, which causes muscle weakness and wasting. The onset of DMD symptoms is in early childhood, usually between the ages of 2 and 3. The disease mainly affects boys, but in rare cases it can affect girls. In Europe and North America, the prevalence of DMD is approximately 6 individuals in 100,000. Doctors often diagnose the disease between the ages of 3 and 6, when children show the first signs of significant muscle weakness, such as a delayed ability to sit, stand, or walk, and difficulty learning to speak. Over time, DMD becomes fatal as muscle wasting disrupts lung and heart function.

There is no cure for DMD, however, life spans have been extended and quality of life improved for many through physical therapy and medications to address some symptoms. With the latest approval, ELEVIDYS becomes a new treatment option for people living with this rare form of muscular dystrophy.

Clinical studies support the approval of ELEVIDYS

ELEVIDYS has been evaluated in three ongoing clinical studies: SRP-9001-101, SRP-9001-102 and SRP-9001-103. The FDA granted accelerated approval primarily based on data from SRP-9001-102 and SRP-9001-103. More than 80 patients treated in the three studies contributed to the safety profile of ELEVIDYS. ELEVIDYS is also studied in study SRP-9001-301 (also known as EMBARK), a global, randomized, double-blind, placebo-controlled Phase 3 clinical study in 126 participants with DMD aged 4 to 7 years .

Consistent with the accelerated approval process, Sarepta is committed to completing a confirmation process. EMBARK will serve as a post-marketing confirmatory study and is fully enrolled with peak results expected in late 2023.

Muscular dystrophy associations invest in DMD research and gene therapy development

See the history of gene therapy for MDA – Duchenne Muscular Dystrophy – here.

MDA has been at the forefront of advances in DMD for over 7 decades. Since its inception, MDA has invested nearly $227 million in DMD and Becker muscular dystrophy (BMD) research, including more than $10 million in DMD grants over the past five years and nearly $2 million in grants for DMD in 2022 only. MDA recently partnered with CureDuchenne and Parent Project Muscular Dystropy to collaborate on a research grant to focus on gene therapy re-dosing in DMD. Read about the grant here. In the 1980s, MDA’s Genetic Task Force pioneered the discovery of the gene that causes DMD, setting the stage for the breakthroughs in genetic medicine.

In the early years, few people believed that gene therapy would ever work. However, MDA believed in my ideas, and without their support, the[microdystrophin genes]currently in clinical trials for Duchenne muscular dystrophy would never have been developed, said Jeff Chamberlin, MD, University of Washington .

MDA has invested more than $125 million in the development of gene therapy for neuromuscular diseases and has been recognized by the American Society for Gene and Cell Therapy (ASGCT) for its significant contributions to the field of genetic medicine. In 2019, MDA received the ASGCT’s Sonia Skarlatos Public Service Award, which recognizes an individual or group who has consistently promoted and improved the field of cell and gene therapy through government agencies, public policy groups, public education, or non-profit organizations. governmental charitable organizations.

About the Muscular Dystrophy Association

The Muscular Dystrophy Association (MDA) is the number 1 voluntary healthcare organization in the United States for people with muscular dystrophy, ALS, and related neuromuscular diseases. For more than 70 years, MDA has led the way in accelerating research, advancing care, and championing the support of our families. MDA’s mission is to enable the people we serve to live longer, more independent lives. To learn more visit mda.org and follow MDA on Instagram, Facebook, ChirpingTikTok, LinkedIn and YouTube.


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