Parent Project Muscular Dystrophy (PPMD), a nonprofit organization leading the fight to end Duchenne muscular dystrophy (Duchenne), applauds the U.S. Food and Drug Administration (FDA) for its decision to expand the labeled indication for ELEVIDYS, a micro-dystrophin gene therapy, to include individuals at least 4 years of age and remove ambulation restrictions, marking a significant milestone in the Duchenne treatment landscape. Additionally, the FDA has converted the ELEVIDYS accelerated approval to a traditional approval for ambulatory patients. The indication in non-ambulatory patients is under the accelerated approval pathway and will require confirmatory studies to convert to traditional approval.

The ELEVIDYS label now states that the adeno-associated virus (AAV) vector-based gene therapy is indicated in individuals at least 4 years of age: “For the treatment of Duchenne muscular dystrophy (DMD) in patients who are ambulatory and have a confirmed mutation in the DMD gene,” as well as, “For the treatment of DMD in patients who are non-ambulatory and have a confirmed mutation in the DMD gene,” expanding the label previously approved and filed with the FDA through Accelerated Approval in June 2023. This expanded indication offers hope and opportunity for a broader spectrum of individuals living with Duchenne and their families.

“This decision by the FDA represents a monumental leap forward in our collective efforts to end Duchenne muscular dystrophy,” said PPMD Founding President and CEO Pat Furlong. “By expanding the label’s age inclusion and removing ambulation restrictions, ELEVIDYS can now reach and benefit a more diverse population of Duchenne patients, providing more families with the opportunity to consider this treatment as an option for their child or themselves.”

ELEVIDYS is made available and marketed in the United States by Sarepta Therapeutics. The FDA had previously granted an Accelerated Approval to ELEVIDYS for patients ages 4-5 years old in June 2023. In February 2024, FDA accepted and filed Sarepta Therapeutics’ efficacy supplement to the Biologics License Application (BLA) for ELEVIDYS, with the efficacy supplement seeking to expand the therapy’s labeled indication.

During its review, FDA also evaluated the efficacy supplement to convert the ELEVIDYS accelerated approval to a traditional approval. FDA approval of a drug means that data on the drug’s effects have been reviewed by the Center for Biologics Evaluation and Research (CBER), and the drug is determined to provide benefits that outweigh its known and potential risks for the intended population. After review of the efficacy supplement, FDA has converted the ELEVIDYS accelerated approval to a traditional approval for ambulatory patients. ELEVIDYS is approved for non-ambulatory patients through the Accelerated Approval pathway based on the surrogate outcome of micro-dystrophin expression. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.

“We extend our sincere appreciation to the FDA for its flexibility and dedication to patient-centric decision-making and its commitment to advancing therapies for rare diseases. Furthermore, we are eternally grateful to the Duchenne community, including patients, families, clinicians, researchers, and advocates, whose unwavering support and collaboration have been instrumental in driving progress in the fight to end Duchenne,” Furlong added. “Patients and families need treatment options, and we applaud the expansion of these options. We also recognize this is just the beginning for AAV-based gene therapies, and we eagerly look forward to continued innovation and progress.”

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 younger than age 4. Sarepta has ongoing studies in these patients to determine if ELEVIDYS is safe and effective. Additionally, ELEVIDYS is currently not available for people with DMD gene deletions in exons 8 and/or 9 due to safety concerns.

Sarepta will participate in PPMD’s 30th Annual Conference in Orlando, Florida, taking place June 27-29, 2024, where we look forward to learning more about the expanded label.

For three decades, PPMD has been dedicated to working with researchers, clinicians, industry, and families to advance treatments for all people living with Duchenne and Becker. The organization employs a comprehensive approach to identify opportunities to accelerate development of all therapies for Duchenne, including regulatory, research, and patient-recruitment counsel to help expedite the progress of investigational products.

PPMD’s patient-report registry, The Duchenne Registry, as well as PPMD’s Duchenne Outcomes Research Interchange, serve as resources for researchers and industry to access aggregated, de-identified data provided by patients and families. Additionally, PPMD remains committed to its innovative 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 also takes a lead role in advocating for national newborn screening efforts for Duchenne, Becker, and carrier identification, as well as policy efforts around access and reimbursement.

 

ABOUT PARENT PROJECT MUSCULAR DYSTROPHY

Duchenne is a 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 eight 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.

Source: https://www.prnewswire.com/news-releases/ppmd-applauds-fda-decision-to-expand-elevidys-label-for-ages-four-years-and-older-302178583.html
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