PPMD is excited to share that the FDA has made the decision to expand the labeled indication for ELEVIDYS, a micro-dystrophin gene therapy used to treat people with Duchenne muscular dystrophy, to include individuals at least 4 years of age and remove ambulation restrictions. ELEVIDYS is made available and marketed in the United States by Sarepta Therapeutics.

• ELEVIDYS is currently approved to treat people with Duchenne who are at least 4 years of age and do not have antibodies to AAVrh74. If you are unsure if your child is eligible to receive ELEVIDYS, call your neuromuscular specialist, who will be able to review eligibility criteria with you and discuss whether it is appropriate for your child.
• People with certain DMD gene variants, including any deletion in exon 8 and/or exon 9, are not able to receive ELEVIDYS due to safety concerns. Your neuromuscular specialist will be able to discuss this in more detail with you.
• 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.

“This decision by the FDA represents a monumental leap forward in our collective efforts to end Duchenne muscular dystrophy,” said PPMD’s 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. 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.“

If you have more questions about ELEVIDYS, talk to your neuromuscular provider. If you have nonmedical questions about ELEVIDYS, you can visit Elevidys.com. If you have questions about accessing one of Sarepta’s approved therapies and you live inside the United States, you can contact SareptAssist by calling 1-888-SAREPTA (1-888-727-3782), or emailing sareptassist@Sarepta.com. More information about SareptAssist may be found here: https://www.sarepta.com/sareptassist

If you have general questions about approved or investigational therapies in Duchenne and whether your child is eligible, contact the PPMD Genetic Counselors at coordinator@parentprojectmd.org. If you have questions about access, navigating care, finding a neuromuscular specialist, or are looking for other types of individualized support, connect with the PPMD Team directly through PPMD for You.

PPMD will begin to update our Access & Coverage Resources page to reflect this expanded indication. We remain committed to working towards community access to all approved products and will continue to work with all relevant stakeholders to ensure a favorable access environment for our community.

What Does This Mean?

PPMD’s updated resource of FAQs related to ELEVIDYS, including questions on eligibility and access, is now available.

ELEVIDYS FAQ

Sarepta will also 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.

Continued Progress in the Fight for Every Future

PPMD is pleased to learn this news and reinforce our commitment to advocate for a broader labeling of approved therapies to strive for access for all eligible individuals with Duchenne. 

PPMD has long been optimistic that gene therapy could be a potential treatment for Duchenne. PPMD launched its Gene Therapy Initiative in 2017 to accelerate the potential of gene therapy as a therapeutic for Duchenne. Since the launch of the Initiative, PPMD has funded over $7 million in 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. 

We extend our sincere appreciation to the FDA for its 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.

Read PPMD’s press release here.