About Us
Our mission is to empower people living with neuromuscular diseases to live longer, more independent lives.
Statement from the Muscular Dystrophy Association on the FDA Cellular, Tissue, and Gene Therapies Advisory Committee Vote on Deramiocel
Washington, D.C., July 29, 2026 – Today, the U.S. Food and Drug Administration's Cellular, Tissue, and Gene Therapies Advisory Committee convened to provide advice and expertise on the Biologics License Application for deramiocel, an investigational therapy for Duchenne muscular dystrophy. The advisory committee voted 9-3 that the available evidence did not support deramiocel’s effectiveness in treating cardiomyopathy. The committee's vote is non-binding and is purely advisory in nature, and the FDA should make a regulatory decision on marketing approval prior to the August 22 PDUFA date.
Earlier today, MDA Executive Vice President of Public Policy and Advocacy Paul Melmeyer addressed the advisory committee on behalf of the Muscular Dystrophy Association (MDA) and the neuromuscular disease community we serve. In his testimony, he emphasized that while the Muscular Dystrophy Association does not engage in product-specific advocacy, we urge the FDA and the advisory committee to flexibly evaluate therapies for rare neuromuscular diseases by considering the totality of the evidence, the seriousness of these diseases, the limited treatment options available, and the lived experiences of patients and caregivers.
"We know today’s vote is deeply disappointing for many in the Duchenne community – the room was heavy with the frustration the Duchenne community feels," said Melmeyer. “But we are immensely grateful to every community member who submitted comments, and particularly those who traveled to Maryland to testify. They did an excellent job emphasizing that muscle loss and heart function continue to decline over time, and families cannot get that time back. It was loud and clear that the urgent need for safe and effective therapies has not changed. Neither has the Muscular Dystrophy Association’s commitment to ensuring that people living with Duchenne have access to meaningful treatment options as quickly as the science allows."
As we evaluate today’s outcomes and conclusions, MDA will partner with our fellow Duchenne muscular dystrophy advocacy organizations to convene the community to discuss what comes next. We encourage everyone to join us for a Town Hall: Deramiocel FDA Advisory Committee Meeting Update at 1pm ET, August 4, on what happens after an Advisory Committee hearing.
The Muscular Dystrophy Association remains committed to accelerating the development of more and better therapies for people living with Duchenne and all neuromuscular diseases. We will continue working alongside the FDA, researchers, clinicians, industry, policymakers, and the patient community to ensure the regulatory process reflects both rigorous science and the urgency faced by families waiting for new treatment options.
Media contact: press@mdausa.org.
About MDA Advocacy
The Muscular Dystrophy Association is a nonpartisan 501(c)(3) organization. As strategic partners with the federal government, we work with public officials and administrations of all parties to advance policies that protect the rights, inclusion, dignity, and agency of people with neuromuscular disease. We do not endorse or support candidates or engage in political campaigns or activities. Join us at MDA.org/Advocacy to continue to defend access to care and services and to fight for robust neuromuscular disease research funding, independence, and community connection.
About Muscular Dystrophy Association
Muscular Dystrophy Association (MDA) has been at the center of progress for people living with muscular dystrophy, ALS, and over 300 other neuromuscular conditions for over 75 years. We unite researchers, clinicians, advocates, and families to speed the pace of discovery, improve access to expert care, and ensure inclusion in every aspect of life. Our mission is simple: give the people we serve the tools and opportunities to live longer, more independent lives. To learn more visit mda.org. Follow MDA on social media on Instagram, Facebook, X, TikTok, LinkedIn, and YouTube.
