New York, March 10, 2025 (GLOBE NEWSWIRE) -- The Muscular Dystrophy Association (MDA) welcomes the U.S. Food and Drug Administration (FDA) approval of the expanded indication of Alexion/AstraZeneca’s eculizumab (Soliris) for pediatric patients aged six years and older with generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody positive. This landmark approval makes Soliris the first and only treatment available for pediatric patients living with this debilitating neuromuscular disease.
Previously approved for adults, the expanded use of Soliris is supported by clinical trial data from adults with gMG, as well as pharmacokinetic and safety data in pediatric populations. A 26-week, single-arm study of 11 pediatric patients aged 12 to 17 years demonstrated that adverse reactions to Soliris were consistent with those observed in adults.
“This approval represents a major advancement in the treatment of pediatric myasthenia gravis and provides hope to families navigating this complex disease,” said Sharon Hesterlee, PhD, Chief Research Officer, MDA. “The availability of Soliris for children underscores the importance of continued research and innovation in neuromuscular disease treatments.”
Soliris is administered via intravenous infusion over 35 minutes for adults and 1 to 4 hours for pediatric patients. It remains contraindicated for patients with unresolved serious Neisseria meningitidis infection. Because of the risk of meningococcal infections, Soliris is only available through a restricted Risk Evaluation Management Strategy (REMS) program, requiring patients to be vaccinated against meningococcal disease at least two weeks prior to initiating treatment.
Soliris, a monoclonal antibody first approved in 2007 for paroxysmal nocturnal hemoglobinuria, inhibits the complement system to prevent complement-mediated tissue damage. The treatment has since received multiple indications, including atypical hemolytic uremic syndrome (2011), adult gMG with AChR antibodies (2017), and neuromyelitis optica spectrum disorder (2019).
Donald S. Wood, PhD, President and CEO, MDA, emphasized the essential role of nonprofit organizations and their supporters in advancing treatments for rare diseases. "The approval of Soliris for pediatric myasthenia gravis is a major milestone—proof of how far science has come thanks to donors and supporters who generously give to organizations like Muscular Dystrophy Association because they believe in the power of scientific possibility. By funding research, advocating for the needs of the community, and working alongside our industry partners, MDA and our colleagues at organizations worldwide are pushing the boundaries to bring life-changing therapies to all people living with neuromuscular diseases."
MDA’s Foundational Support of Myasthenia Gravis Research
MDA remains committed to advocating for continued therapeutic advancements and supporting the neuromuscular disease community with resources, education, and research funding. Since its inception, MDA has invested more than $57 million in MG research. Families and individuals diagnosed with myasthenia gravis are encouraged to visit MDA.org here for more information on treatment options and support services and contact the MDA Resource Center for services and support from expert specialists.
Media contact press@mdausa.org.
About Muscular Dystrophy Association
Muscular Dystrophy Association (MDA) is the #1 voluntary health organization in the United States for people living with muscular dystrophy, ALS, and over 300 other neuromuscular conditions. For 75 years, MDA has led the way in accelerating research, advancing care, and advocating support and inclusion of families living with neuromuscular disease. MDA's mission is to empower the people we serve to live longer, more independent lives. To learn more visit mda.org and follow MDA on Instagram, Facebook, X, Threads, TikTok, LinkedIn, and YouTube.
About Muscular Dystrophy Association’s 75th Anniversary
In 2025, the Muscular Dystrophy Association proudly marks 75 years legacy, impact and momentum in the fight against neuromuscular diseases. Since our founding, MDA has been at the forefront of research breakthroughs, providing access to comprehensive care, and championing the rights of people living with muscular dystrophy, ALS, and over 300 other neuromuscular diseases. This milestone has been made possible by generations of dedicated support from people living with neuromuscular disease, their families, researchers, clinicians, volunteers, and donors—who boldly drive our mission forward. As we look ahead, we remain committed to honoring this legacy, building on the impact we’ve made together, and continuing our momentum toward transformative progress for people living with neuromuscular disorders. Learn more at MDA75.org.
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Mary Fiance, National Vice President, Strategic Communications Muscular Dystrophy Association press@mdausa.org
