June 22, 2023 / Advocacy,Care

FDA grants approval via accelerated approval pathway to ELEVIDYS: First-ever gene therapy for individuals with Duchenne ages 4-5

Today is an incredible milestone for the entire Duchenne community. It is a first step. But a huge step forward. Today, the FDA has granted accelerated approval to ELEVIDYS (delandistrogene moxeparvovec-rokl), a micro-dystrophin gene therapy used to treat people ages 4-5 with Duchenne muscular dystrophy. 

  • ELEVIDYS is currently approved to treat people with Duchenne who are 4-5 years of age, able to walk, 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, will not be able to receive ELEVIDYS due to safety concerns. Your neuromuscular specialist will be able to discuss this in more detail with you.

If you have questions about accessing one of Sarepta’s approved therapies and you live inside the United States please contact SareptAssist by calling Call 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

PPMD will also begin including ELEVIDYS on Access & Coverage Resources page in the coming weeks as more information becomes available. 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 Approval Mean?

PPMD will be hosting a webinar on Wednesday, July 12, 2023 at 1:00pm EDT to discuss ELEVIDYS eligibility criteria, the treatment process, and what clinics are doing to treat individuals as quickly as they can. Registration is open for the webinar and attendees can submit questions in advance here.

Register for the webinar

The PPMD team has compiled a resource of FAQs surrounding the accelerated approval of ELEVIDYS and what this means, including questions on eligibility and access.

View FAQ

The PPMD team has also created a short video explaining the ELEVIDYS label and genetic variants.

Watch video

Celebrating a Victory Decades in the Making

PPMD has long been optimistic that gene therapy could be a potential treatment for Duchenne. In 2017, PPMD launched our Gene Therapy Initiative seeking to accelerate the potential of gene therapy as a therapeutic for Duchenne. Our early strategy was to bring attention to and fund key questions that must be answered in order for the technology to progress toward regulatory consideration. 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 Dr. Jerry Mendell’s work at Nationwide Children’s Hospital’s Research Institute, which led to the development of ELEVIDYS. 

Today, we thank YOU. 

We thank the boys and their families who have participated, not just in these first gene therapy trials, but in all of the trials that seek to advance our fight to treat Duchenne. We thank the families who have joined natural history studies and patient registries such as The Duchenne Registry, which have helped strengthen our understanding of this disease. We thank everyone who has participated in PPMD’s patient preference studies so that our community’s views can be quantified and measured by regulators when they are evaluating the benefit-risk assessment of new potential therapies. All of these efforts have been crucial to get us to this incredible milestone today, one that seemed impossible when PPMD was founded nearly 30 years ago.

And we thank our advocates for raising your voices to deliver a clear, concise message about the importance of gene therapy and its relevance to treatments for Duchenne. Whether you sent in written testimony or provided an oral testimony to the Advisory Committee, we thank you for sharing your story so that the FDA could make this decision fully understanding the perspectives of our community.

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.

There is still so much work to do. We know that it will take a combination of therapies to fully halt the progression of Duchenne. And we know that there are many in our community who are not included in the prescribing label for ELEVIDYS. There are ongoing studies in some of these groups to determine if ELEVIDYS is safe and effective. Know that PPMD is here for you, and we will continue our fight until every person with Duchenne has access to the full array of treatment options that cutting-edge scientific and clinical research can provide. 

We also know that 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. PPMD remains focused on working with all stakeholders and the community to do all we possibly can for patients who are candidates for ELEVIDYS obtain access to the drug. 

But today, we also recognize – You spoke. They listened. We hope that you take a moment to celebrate a victory that has been decades in the making, one that every single member of this community has played a part in. 

A first gene therapy approval in Duchenne.

Read PPMD’s press release.

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