New York, Dec. 12, 2025 (GLOBE NEWSWIRE) — The Muscular Dystrophy Association (MDA) recognizes the U.S. Food and Drug Administration (FDA) approval of Amgen’s UPLIZNA® (inebilizumab-cdon) for the treatment of generalized myasthenia gravis (gMG) in adults who are anti-acetylcholine receptor (AChR) and anti-muscle specific tyrosine kinase (MuSK) antibody positive. This is the first and only CD19-Targeted B-Cell therapy approved for gMG. The approval offers patients a new treatment option that has the potential for long-term disease control with just two doses a year, after two initial loading doses. Read the Amgen press release here.
Uplizna is already FDA-approved for neuromyelitis optica spectrum disorder (NMOSD) and IgG4-related disease (IgG4-RD) — two immune-mediated disorders in which B cell depletion has shown meaningful clinical benefit. Myasthenia gravis (MG) shares similar B cell-driven autoimmune mechanisms, making Uplizna a strong therapeutic candidate for patients whose symptoms are caused by pathogenic autoantibodies. Myasthenia gravis (MG) shares similar B cell-driven autoimmune mechanisms, making Uplizna a strong therapeutic candidate for patients whose symptoms are caused by pathogenic autoantibodies. The FDA’s decision reflects this growing body of evidence and marks an important expansion of treatment options for people living with gMG.
MDA applauds the scientists, clinicians, patients, and partners across the neuromuscular and autoimmune communities whose combined efforts made this milestone possible. The MDA Care Center Network continues to serve as a critical hub for clinical trials, multidisciplinary care, and patient support across the United States.
Angela Lek, PhD, Chief Research Officer, MDA, said, “The approval of Uplizna represents meaningful progress for people living with generalized myasthenia gravis. This therapy builds on scientific advances made across the broader autoimmune and neuromuscular research community in MG. Muscular Dystrophy Association is proud to support comprehensive care through our nationwide Care Center Network and to help ensure that patients and families have access to the latest clinical information, resources, and treatment options.”
“Growing up attending the Muscular Dystrophy Association’s Summer Camp shaped my understanding of the power of community, care, and advocacy for people living with neuromuscular diseases. As someone living with myasthenia gravis, I’ve seen firsthand how MDA’s continued commitment to research, care, and support drives real progress for families like mine. The past several years have been a momentous time for the MG community, with new therapies transforming what is possible. The approval of Uplizna continues that momentum—bringing renewed hope, more choices, and greater stability for patients and families,” said Allison Foss, Executive Director, Myasthenia Gravis Association.
Clinical Impact of UPLIZNA® (inebilizumab-cdon)
UPLIZNA® (inebilizumab-cdon) is a CD19+ B cell-directed monoclonal antibody administered via intravenous infusion. Already authorized by the FDA for IgG4-related disease (IgG4-RD) and neuromyelitis optica spectrum disorder (NMOSD), the therapy brings forward a proven track record in treating antibody-mediated conditions. These prior approvals contribute to a well-established foundation of safety and efficacy that supports its use for a range of immune-driven diseases.
In clinical trials for generalized myasthenia gravis, Uplizna demonstrated strong clinical results across adult age groups. One of the features that truly sets Uplizna apart is its durability: following an initial loading dose, it requires only two doses per year. This stands in contrast to other approved therapies, which often require administration every 2–8 weeks, representing a meaningful difference for people seeking consistent, sustained symptom control with fewer treatment visits.
Key findings from the trials include:
• Significant improvements in MG-ADL and QMG functional scores
• Reduced frequency and severity of exacerbations
• Early onset and sustained benefit across measures of disease activity
• A safety profile consistent with prior inebilizumab-cdon indications
“The approval of Uplizna represents a meaningful advancement grounded in years of research into the immunobiology of myasthenia gravis, including work that has helped clarify the role of B cell subsets that drive disease activity. Uplizna’s targeted mechanism of action against CD19-expressing B cells reflects the scientific progress made across the field and offers clinicians a new way to intervene more precisely in the underlying pathology. It’s a powerful example of how sustained investigation — and the partnership of the patients who participate in these studies — can translate into durable, antibody-directed treatment options for people living with gMG,” said Kevin C. O’Connor, PhD, Professor of Neurology and Immunobiology at Yale School of Medicine.
About Generalized Myasthenia Gravis
Generalized myasthenia gravis is an autoimmune disorder in which antibodies disrupt communication between nerves and muscles, leading to variable and often serious muscle weakness. Symptoms may affect the eyes, face, throat, limbs, and respiratory muscles. While several treatment options exist, many individuals require additional therapeutic choices to manage the disease effectively.
“Uplizna’s approval represents an important step forward for patients with generalized myasthenia gravis and reflects the continued evolution of targeted, mechanism-based treatments. For years, the field has worked to better understand the complex immune pathways that drive MG. This therapy builds on that progress by specifically targeting key B cell populations involved in disease activity. For clinicians, having an option with durable dosing and a well-characterized safety profile provides another meaningful tool to tailor care to the needs of each person. For patients, it brings renewed possibility for stability and improved quality of life,” Barry J. Byrne, MD, PhD, Chief Medical Advisor and Board Member, Muscular Dystrophy Association, and Associate Chair of Pediatrics and Director of the Powell Gene Therapy Center at the University of Florida.
MDA’s Commitment to MG Research and Care
The Muscular Dystrophy Association is one of the largest nonprofit supporters of myasthenia gravis research in the world, demonstrating decades of commitment with more than $57 million invested since its founding. MDA’s recent investments include over $400,000 in MG research from 2020–2025, and more than $100,000 supporting two active MG research grants in 2025 alone. These investments are advancing critical areas such as immunobiology, diagnostics, therapeutic development, and clinical trial readiness. Beyond research funding, MDA provides comprehensive patient support through its nationwide network of more than 150 Care Centers, offering access to essential multidisciplinary care and enabling patient participation in clinical trials. Additionally, MDA empowers families in the MG community by providing essential education, advocacy, and resources.
Support and Guidance for Families
For more information on myasthenia gravis, and ongoing support for families and medical professionals, contact the MDA Resource Center by phone 1-833-ASK-MDA1 (1-833-275-6321) or email [email protected].
Media inquiries contact [email protected].
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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, Bluesky, TikTok, LinkedIn, and YouTube.
About Muscular Dystrophy Association’s 75th Anniversary
In 2025, the Muscular Dystrophy Association proudly marks 75 years of 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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