PPMD Applauds FDA for Landmark Approval of First-Ever Gene Therapy Treatment for Duchenne for Patients Ages 4-5 Years Old

Home PPMD Applauds FDA for Landmark Approval of First-Ever Gene Therapy Treatment for Duchenne for Patients Ages 4-5 Years Old
Written by Doug Hampton

Organization Will Continue to Support Patient Access and Drive Policies and Projects That Support Development and Approval of More Therapies

WASHINGTON, June 22, 2023 /PRNewswire/ — Parent Project Muscular Dystrophy (PPMD), a nonprofit organization leading the fight to end Duchenne muscular dystrophy (Duchenne), heralded today’s decision by the Food and Drug Administration (FDA) to approve the first-ever U.S. application for a gene therapy treatment for Duchenne patients ages 4-5 years old. ELEVIDYS (delandistrogene moxeparvovec-rokl) is the sixth approved therapy in Duchenne and the first therapy to replace the missing dystrophin protein by using a modified smaller version of the dystrophin gene, called micro-dystrophin, to produce a modified micro-dystrophin protein. ELEVIDYS will be made available and marketed in the United States by Sarepta Therapeutics.

“PPMD is thrilled that FDA has granted an Accelerated Approval to ELEVIDYS, a gene therapy that provides systemic delivery of a microdystrophin construct that targets both skeletal and cardiac muscle achieved through one IV injection. Despite the incredible advances made with five prior drug approvals for Duchenne, our community continues to have significant unmet needs. Today’s approval of a dystrophin replacement strategy further builds upon, and extends, the positive impact of Duchenne therapy development to date,” said PPMD Founding President and CEO Pat Furlong.

“We applaud Sarepta Therapeutics, Dr. Jerry Mendell, and the many other clinicians and scientists who have worked tirelessly on this therapy. And we are eternally grateful to all of the patients, and their families, who participated in these first gene therapy trials, as well as the organizations and advocates who worked collaboratively to make this historic day possible. Their efforts have forever changed the Duchenne landscape.”

“We want to commend FDA leadership for recognizing that ELEVIDYS has a favorable benefit-risk profile and that it met its Accelerated Approval requirements. This approval demonstrates an agency commitment to the needs of patients, particularly those with serious unmet medical needs,” Furlong added.

PPMD has long been optimistic that gene therapy could be a potential treatment for Duchenne. PPMD launched its Gene Therapy Initiative in 2017 as a long-term concept seeking to accelerate the potential of gene therapy as a therapeutic for Duchenne. Its early strategy was to bring attention to and fund key questions that must be answered in order for the technology to progress toward approvals. Since the launch of the Initiative, PPMD has funded over $7 million in a variety of gene therapy and related approaches to several institutions, including the work of Dr. Jerry Mendell at Nationwide Children’s Hospital’s Abigail Wexner Research Institute, which led to the development of ELEVIDYS.

The label for ELEVIDYS currently excludes patients who are under 4 years of age, older than 5 years of age, or non-ambulatory. Sarepta has ongoing studies in these patients to determine if ELEVIDYS is safe and effective. Additionally, ELEVIDYS is currently not available for people with genetic deletions in exons 8 and/or 9 due to safety concerns.

PPMD will be hosting a webinar on Wednesday, July 12, 2023 at 1:00pm ET to discuss ELEVIDYS eligibility criteria, the treatment process, and what clinics are doing to treat individuals as quickly as they can. Sarepta Therapeutics will also be participating in PPMD’s 2023 Annual Conference in Dallas, Texas on Saturday, July 1, 2023 during the Gene Therapy Town Hall session.

On June 14, 2023, PPMD along with other leaders in the Duchenne community sent a letter to FDA/CBER leadership calling on them to expedite the review of the EMBARK data when it becomes available, so that there will be no delays in considering a broadened label for ELEVIDYS.

“Going forward, PPMD remains focused on working with all stakeholders and the community to do all we possibly can to ensure all patients who are candidates for ELEVIDYS obtain access to the drug. It is not enough to clear the FDA. We must make sure that all those who can use these innovative therapies are able to do so as they reach the market. We also look forward to additional data coming from ongoing clinical trials of ELEVIDYS which could potentially broaden the approval to also cover all Duchenne patients,” said Furlong. “Additionally, PPMD knows that it will take a combination of therapies to fully halt the progression of Duchenne, and we will continue to support the development of additional tools and resources to drive forward approvals of additional therapies and treatments.”

One of the tools that is vital in driving forward new advances in therapies and treatments is PPMD’s patient-report registry, The Duchenne Registry. The Duchenne Registry was established in 2007 to serve as a resource for researchers and industry, allowing access to aggregated, de-identified information provided by patients and families. In 2022, the organization announced the launch of its Electronic Health Record (EHR) study, marking an important next step in the development of PPMD’s Duchenne Outcomes Research Interchange (“the Interchange”) – a patient- and clinician-reported data warehouse which can combine data from The Duchenne Registry with EHR data, as well as data provided by industry partners with approved therapies. By combining this data, the Interchange will provide a more complete picture of Duchenne and Becker muscular dystrophy, with the goal of helping researchers and clinicians address common research challenges and do their work better and faster, thereby accelerating scientific discovery and patient access to new therapies.

To support additional product development and approval, PPMD will also continue to build upon its sector-leading patient engagement efforts, which include multiple patient preference studies and a revised community-generated draft guidance document, recently updated to reflect advancements in knowledge, understanding, care, clinical trials, and approvals. PPMD has also served as the leading advocacy organization driving national Duchenne newborn screening efforts and policy efforts around access and reimbursement in anticipation of this remarkable day – the first gene therapy approval for Duchenne.


Duchenne is a fatal genetic disorder that slowly robs people of their muscle strength. Parent Project Muscular Dystrophy (PPMD) fights every single battle necessary to end Duchenne.

We demand optimal care standards and ensure every family has access to expert healthcare providers, cutting edge treatments, and a community of support. We invest deeply in treatments for this generation of Duchenne patients and in research that will benefit future generations. Our advocacy efforts have secured hundreds of millions of dollars in funding and won six FDA approvals.

Everything we do—and everything we have done since our founding in 1994—helps those with Duchenne live longer, stronger lives. We will not rest until we end Duchenne for every single person affected by the disease. Join our fight against Duchenne at EndDuchenne.org. Follow PPMD on Facebook, Twitter, Instagram, and YouTube.

SOURCE Parent Project Muscular Dystrophy (PPMD)

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