FDA Approves Amgen’s Inebilizumab-cdon (UPLIZNA) to Treat Generalized MG
Amgen Press Release
December 12, 2025
A new treatment for generalized myasthenia gravis has been approved by the Food and Drug Administration.
Inebilizumab-cdon – brand-name UPLIZNA – is a monoclonal antibody treatment that selectively targets CD19-positive B cells. This approach addresses a biological root cause of the disease. The treatment is FDA-approved in adults diagnosed with AChR and MuSK generalized MG. Patients need only two doses a year, following two initial loading doses.
“Managing a rare and chronic illness can mean facing unpredictable relapsing symptoms and demanding treatment schedules,” said Samantha Masterson, the MGFA’s president and chief executive officer. “This approval marks an important milestone, offering durable efficacy and a dosing schedule that provides people living with generalized myasthenia gravis six months of treatment-free time between maintenance doses.”
Study data come from the MINT trial, which commenced in 2020. The most recent phase 3 trial data were presented by Dr. Richard Nowak at the MGFA Scientific Session on October 29, 2025. Read more about this announcement on Amgen’s website
Read MoreIgA autoantibodies demonstrate a novel mechanism of 1 MuSK myasthenia gravis pathology
Brain
October 28, 2025
A study published in Brain offers new insights into MuSK myasthenia gravis (MG), a rare form of MG that causes significant muscle weakness. Until now, most research has focused on MuSK IgG4, the main autoantibody in the disease, which disrupts acetylcholine receptor clustering and weakens nerve-muscle communication.
This study, led by Dr. Gianvito Masi in Dr. Kevin O’Connor’s lab at Yale University, shows that another antibody class, IgA, may play a role in the disease. IgA antibodies are usually found at mucosal sites, such as in the gut and lungs, where they help protect against infections. Researchers discovered that a subgroup of patients with MuSK MG also carries IgA autoantibodies targeting MuSK.
Using patient-derived IgA autoantibodies, B-cell sequencing technology, muscle cell cultures, and in vivo experiments, the team demonstrated that:
1) MuSK-specific IgA-producing cells can survive for years and are not fully eliminated by treatments that target B cells (such as anti-CD20 therapy).
2) Individual IgA autoantibodies can activate MuSK, but when multiple IgA antibodies act together, they can instead block this process and cause weakness. Indeed, in experimental models, the combination of patient-derived IgA autoantibodies produced myasthenic symptoms, suggesting that MuSK IgA may directly contribute to disease pathology.
This is the first evidence showing that IgA autoantibodies can be involved in the autoimmune response against MuSK. These findings raise new questions about the role of mucosal immunity (such as the influence of infections or environmental exposures) in the disease. A better understanding of this process could open new avenues for research and future treatment.
Read MoreTherapeutic IgG-and IgM-specific proteases disarm the acetylcholine receptor autoantibodies that drive myasthenia gravis pathology
Proceedings of the National Academy of Sciences (PNAS)
October 23, 2025
The MG group at Yale University, led by Alexandra Bayer and in collaboration with Seismic Therapeutic, uncovered a new role for IgM autoantibodies in myasthenia gravis (MG).
In MG, the body’s immune system makes autoantibodies that interfere with the signals between nerves and muscles, leading to weakness. Most often, these harmful autoantibodies belong to the IgG class, but their new research has shown that in some people, another type, called IgM, can also play a major role.
They tested a new treatment approach called S-1117, an enzyme designed to “disarm” IgG autoantibodies so they can no longer cause damage. In most patient samples, this worked well. However, in some samples where the treatment was less effective, they learned that IgM autoantibodies were driving the disease in those cases. By pairing S-1117 with another enzyme that targets IgM, they were able to completely stop the harmful effects of both types of autoantibodies.
For people living with MG, these findings are important because they point toward a future of more personalized treatments. Instead of a one-size-fits-all approach, physicians may be able to identify which type of autoantibody is causing the problem in each patient and tailor therapy to block it. This discovery offers new hope for patients who do not respond to current treatments and shows the power of science and collaboration in moving us closer to better care and better lives for everyone with MG.
Read MorePositive Phase III clinical trial results for a potential new MG treatment
AstraZeneca Press Release
July 24, 2025
Study data for a medication called gefurulimab demonstrated a statistically significant and clinically meaningful improvement from baseline in MG-ADL total score at week 26 compared to placebo. The double-blind study included patients from 20 countries.
Kelly Gwathmey, MD, vice chair of the MGFA Medical & Scientific Advisory Council and principal investigator in the trial, said, “Rapidly fluctuating symptoms and the unpredictable disability associated with gMG can affect nearly every aspect of a patient’s life, making early intervention and sustained disease control a critical treatment goal. A once-weekly, self-administered C5 treatment option would offer patients greater convenience and independence in managing their condition, empowering them to have more control over their therapy.”
Read the press release below.
Read MoreJohnson & Johnson Receives FDA Approval for IMAAVYTM (nipocalimab-aahu)
New treatment approved for treatment of AChR+ or MuSK+ generalized myasthenia gravis
April 30, 2025
The FDA has approved IMAAVYTM (nipocalimab) for the treatment of people ages 12 and older who are living with antibody positive (AChR+ or MuSK+) generalized myasthenia gravis. Johnson & Johnson states that this new FcRn blocker offers long-lasting disease control in the broadest population of people living with MG.
“We consistently hear from individuals living with myasthenia gravis who are hopeful for new treatment options that may help bring greater stability, independence, and predictability to their lives,” said Samantha Masterson, president and CEO of the Myasthenia Gravis Foundation of America.
“This treatment provides another option which could help address the constant uncertainty and heavy physical and mental toll that MG symptom relapse presents to patients and their families.”
The approval, which follows FDA Priority Review designation, offers a new treatment option in a proven class that can be used by adults and pediatric patients 12 years of age and older who are anti-acetylcholine receptor (AChR) or anti-muscle-specific kinase (MuSK) antibody positive. IMAAVY demonstrated a rapid and sustained reduction in autoantibody levels by up to 75% from the first dose and throughout a 24-week period of monitoring.
Read more about this amazing announcement and latest MG treatment.
Read MoreSerum fibrinogen is not elevated in patients with myasthenia gravis
April 15, 2025
A study in the journal Scientific Reports set out to confirm findings of a previous study suggesting that residual serum fibrinogen could represent a universal diagnostic biomarker for MG. Using multiple methodologies to compare fibrinogen levels between MG patients and controls, the authors did not find elevated serum fibrinogen levels in MG patients. These results underscore the need for replication of novel findings to ensure our common goal of identifying effective biomarkers for MG.
Read MoreVyvgart Hytrulo for self-injection is now FDA approved
April 10, 2025
Vyvgart Hytrulo, an FDA-approved treatment for generalized AcHR myasthenia gravis, is now available in the United States as a self-injection. Those taking the drug can take the injection at home, in a single dose, providing more flexibility to patients.
Read MoreU.S. FDA Approves Expanded Use of Eculizumab for Pediatric Myasthenia Gravis
March 12, 2025
Good news for young people who are diagnosed with myasthenia gravis: the United States Food and Drug Administration (FDA) has approved expanded use of Alexion/AstraZeneca’s eculizumab (SOLIRIS®) for the treatment of pediatric patients aged six years and older with generalized myasthenia gravis (gMG) who are acetylcholine receptor antibody positive (AChR-Ab+). This action makes eculizumab the first and only FDA-approved treatment available for pediatric gMG patients.
Eculizumab is a monoclonal antibody that works by inhibiting the complement protein C5 to prevent complement-mediated tissue damage and was first approved for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) in 2007. Based on the results from a pivotal phase 3 clinical trial (REGAIN, NCT01997229), it was approved as a treatment for adults with gMG in 2017. A recent positive single-arm study (NCT03759366) in refractory AChR-Ab+ gMG patients aged 12-17 years provided evidence in support of its use in the pediatric population.
“Myasthenia gravis can impact anyone at any age and is extremely devastating for young patients and their families,” said Samantha Masterson, president and CEO of the Myasthenia Gravis Foundation of America. “Through groundbreaking research and a commitment to advancing therapeutics, pediatric gMG patients now have a new treatment option that could help them live an improved quality of life with MG. We recognize the amazing work of Alexion/AstraZeneca and the FDA to drive this forward. This would not be possible without the support of our global MG community in making sure that awareness and education about this debilitating disease spreads around the world.”
Eculizumab is given by an intravenous infusion. It has several safety considerations, including an increased risk of a serious and life-threating infection caused by Neisseria meningitidis. Patients may only access the therapy through a restricted program under a Risk Evaluation Management Strategy (REMS) program to mitigate risk.
References:
https://alexion.us/-/media/alexion_global/documents/regulatory/north-america/usa/2024/english/soliris_uspi.pdf
https://www.sciencedirect.com/science/article/pii/S1474442217303691?via%3Dihub
https://www.sciencedirect.com/science/article/pii/S0887899424001498?via%3Dihub
Does Surgical Removal of the Thymus Have Deleterious Consequences?
Neurology
May 24, 2024
Recently, a concern has been raised about deleterious consequences of the surgical removal of thymic tissue, including for patients who undergo thymectomy for myasthenia gravis (MG) or resection of a thymoma. This review adopts a multidisciplinary approach to scrutinize the evidence concerning the long-term risks of cancer and autoimmunity postthymectomy. We conclude that for patients with acetylcholine receptor antibody-positive MG
and those diagnosed with thymoma, the removal of the thymus offers prominent benefits that well outweigh the potential risks. However, incidental removal of thymic tissue during other thoracic surgeries should be minimized whenever feasible.
NMD Pharma Successfully Completes First Phase of NMD670 in MG Clinical Study – Phase 2 Active and Recruiting
Science Translational Medicine
March 20, 2024
After successfully completing phase 1 single ascending dose in healthy volunteers, NMD670 was tested in patients with MG in a randomized, placebo-controlled, single-dose, three-way crossover clinical trial. The clinical trial evaluated safety, pharmacokinetics, and pharmacodynamics of NMD670 in 12 patients with mild MG. NMD670 had a favorable safety profile and led to clinically relevant improvements in the quantitative myasthenia gravis (QMG) total score. This translational study spanning from single muscle fiber recordings to patients provides proof of mechanism for ClC-1 inhibition as a potential therapeutic approach in MG and supports further development of NMD670.
NMD has opened phase 2 and is currently active and recruiting MG patients. Visit the study page on ClinicalTrials.gov to determine your eligibility and how to apply to be part of this exciting trial.
Comparative effectiveness of azathioprine and mycophenolate mofetil for myasthenia gravis (PROMISE-MG): a prospective cohort study
The Lancet Neurology
March 2024
This study aimed to evaluate the comparative effectiveness of azathioprine and mycophenolate mofetil in MG treatment, and to assess the effect of the dose and duration of treatment. In the study, more than half of patients treated with azathioprine and mycophenolate mofetil felt their quality of life improved; no difference in clinical outcomes was noted between the two drugs. Adverse events associated with azathioprine were potentially more serious than those with mycophenolate mofetil, although mycophenolate mofetil is teratogenic. Lower-than-recommended doses of azathioprine might be effective, with reduced dose-dependent adverse events. More comparative effectiveness studies are required to inform treatment choices in myasthenia gravis.
This study was funded, in part, by the Myasthenia Gravis Foundation of America.
Fifty Plus Advocate
February 2024
Glenda Thomas, MGFA support group leader, has been sharing her story for several years, including in a recent article in a local paper – check it out on page 16. She also discusses the importance of support groups when you are navigating a chronic illness like myasthenia gravis.
ZILBRYSQ® (zilucoplan) Is Now Commercially Available in the U.S. for the Treatment of Generalized Myasthenia Gravis (gMG) in Adult Patients Who Are Anti-Acetylcholine Receptor (AChR) Antibody Positive
UCB Website
January 3, 2024
UCB, a global biopharmaceutical company, announced today that ZILBRYSQ® (zilucoplan) is now available in the U.S. for the treatment of generalized myasthenia gravis (gMG) in adult patients who are anti-acetylcholine receptor (AChR) antibody positive. ZILBRYSQ was approved by the U.S. Food and Drug Administration (FDA) on October 17, 2023.1
ZILBRYSQ is available by prescription as a ready-to-use pre-filled syringe that is a once-daily administration.
UCB Announces U.S. FDA Approval of ZILBRYSQ[®] (zilucoplan) for the Subcutaneous Treatment of Adults with Generalized Myasthenia Gravis
UCB Website
October 17, 2023
UCB announced that ZILBRYSQ® (zilucoplan) has been approved by the U.S. Food and Drug Administration (FDA) for the treatment of generalized myasthenia gravis (gMG) in adult patients who are anti-acetylcholine receptor (AChR) antibody-positive. Zilucoplan is the first once-daily subcutaneous, targeted C5 complement inhibitor for gMG and is self-administered.
Zilucoplan is the first once-daily subcutaneous (SC), targeted peptide inhibitor of complement component 5 (C5 inhibitor). It is the only once-daily gMG target therapy for self-administration by adult patients with anti-AChR antibody-positive gMG. Benefits of self-administered treatment can include reduced traveling time to and from hospitals, decreased interference with work obligations, and increased independence. Unlike monoclonal antibody C5 inhibitors, as a peptide, zilucoplan can be used with intravenous immunoglobulin and plasma exchange, without the need for supplemental dosing.
The FDA approval of zilucoplan1 is supported by safety and efficacy data from the RAISE study (NCT04115293), published in The Lancet Neurology in May 2023. The RAISE study was a multi-center, phase 3, randomized, double-blind, placebo-controlled study to assess the efficacy, safety profile, and tolerability of zilucoplan in adult patients with anti-acetylcholine receptor (AChR) antibody-positive gMG.
“This is an important development for the community because, with more FDA-approved treatments for generalized myasthenia gravis, physicians have additional tools to treat this disease in individualized ways that are the right fit for each individual patient,” said Samantha Masterson, President and Chief Executive Officer of the Myasthenia Gravis Foundation of America. “We are so grateful to UCB for being part of the myasthenia gravis community and their continued commitment to finding solutions for people living with this chronic, autoimmune, neuromuscular disease.”
With the approval of zilucoplan, alongside the company’s neonatal Fc receptor (FcRn) blocker RYSTIGGO® (rozanolixizumab-noli), which was approved earlier this year by the FDA, UCB’s portfolio provides healthcare professionals the option of addressing either complement activation or pathogenic auto-antibodies for appropriate patients.
Read the Official News Announcement from UCB.
Assessing High-dose Chemotherapy and Hematopoietic Cell Transplantation in Severe Myasthenia Gravis
Annals of Clinical and Translational Neurology
September 19, 2023
Summary by: Dakota Campbell –Communications Program Manager- Rare Diseases Clinical Research Network, Cincinnati Children’s, Cincinnati, OH 45229
Myasthenia gravis (MG) is a rare neuromuscular disorder caused by an autoimmune response which blocks or damages acetylcholine receptors on muscles. High-dose chemotherapy (HDIT) and autologous hematopoietic cell transplantation (HCT), also known as bone marrow transplant, are potential treatments for MG.
In this study, researchers investigated the safety and efficacy of HDIT and HCT in a patient with severe, treatment-resistant MG.
Results show that HDIT and HCT induced remission of MG. The team assessed the effect of treatment on the underlying immunopathology. Intriguingly, the acetylcholine receptor autoantibodies (AChR)—the known pathogenic mediators of the disease—did not appreciably lower after the treatment.
Authors state that these findings suggest a cell-based disease mechanism, which responds to high-dose therapy, may play a role in the pathology in addition to AChR autoantibodies. Further studies are needed to establish whether HDIT and HCT can be an effective therapy for severe MG.
Telehealth in Myasthenia Gravis: What We’re Learning from a Pilot Study
Myasthenia Gravis Rare Disease Network
August 22, 2023
To learn more about the use of telehealth in MG, the Myasthenia Gravis Rare Disease Network (MGNet) is conducting a pilot study, “Adapting Disease Specific Outcome Measures Pilot Trial for Telehealth in Myasthenia Gravis (ADAPT-teleMG)” (clinicaltrials.gov ID NCT05917184). The team is evaluating telehealth visits and remote disease-specific assessments for patients with MG.
Here, lead investigator Amanda Guidon, MD, MPH, and Meridith O’Connor, MG patient and assistant vice president of patient engagement, advocacy, and policy at the MGFA, share more about the study and its impact on the patient and research community.
Read more about this published research
Modifying Patient T-cells Creates Novel Approach to Treating Myasthenia Gravis and other Diseases
Cartesian Website
July 1, 2023
Cartesian Therapeutics announced the publication of positive results of the first successful clinical trial of RNA cell therapy for patients with autoimmune disease. By modifying patients’ T-cells with mRNA (a form of rCAR-T therapy), the study has created a novel approach for potentially treating myasthenia gravis (MG) and other autoimmune diseases.
The data demonstrates potent and durable clinical improvement in patients with MG, representing the first successful Phase 2 trial using RNA cell therapy.
“We are grateful to our community of MG patients and physicians for enabling clinical development of novel therapeutics such as rCAR-T,” said Samantha Masterson, President & CEO of Myasthenia Gravis Foundation of America. “A safe, personalized therapy with durable clinical benefit would be a welcome addition to the growing toolkit of MG treatments.”
The results described in The Lancet Neurology paper suggest that rCAR-T may be useful in treating a variety of other autoimmune diseases and may overcome many of the risks and toxicities associated with conventional DNA-based CAR-T cells. The news release “Safety and Efficacy of Autologous RNA Chimeric Antigen Receptor T-cell (rCAR-T) Therapy in Myasthenia Gravis: a prospective, multicenter, open-label, non-randomized phase 1b/2a study”, shares more details.
You can also download the full manuscript.
To learn more about Cartesian’s study with rCAR-T, or review other open and recruiting MG clinical trials that you can apply to, visit the MGFA Clinical Trials page.
UCB announces U.S. FDA approval of RYSTIGGO® for treatment of generalized myasthenia gravis
UCB Website
June 27, 2023
UCB announced U.S. Food and Drug Administration (FDA) approval of a new treatment called RYSTIGGO (rozanolixizumab-noli) for adults with generalized myasthenia gravis who are anti-acetylcholine receptor (AChR) or anti-muscle-specific tyrosine kinase (MuSK) antibody positive.
Rozanolixizumab-noli is a subcutaneous infusion injection and the only FDA-approved treatment in adults for both anti-AChR AND anti-MuSK antibody-positive gMG, the two most common subtypes of gMG.
U.S. FDA approval is based on the pivotal Phase 3 MycarinG study in gMG2, a large phase 3 study which demonstrated treatment with rozanolixizumab-noli that resulted in statistically significant improvements in gMG-specific outcomes, including everyday activities such as breathing, talking, swallowing, and being able to rise from a chair.
“We want to thank UCB for their continued commitment to the MG community to bring a new FDA-approved treatment option for generalized myasthenia gravis to patients and their treating physicians,” said Samantha Masterson, President and Chief Executive Officer of the Myasthenia Gravis Foundation of America (MGFA). “People living with generalized myasthenia gravis continue to experience significant unmet medical needs, this means expanding the number of FDA-approved treatment options is particularly important to treat this chronic, autoimmune, neuromuscular disease.”
Rozanolixizumab-noli will be commercially available in the U.S. during the 3rd quarter of 2023.
Read the official news release from UCB.
argenx Announces U.S. FDA Approval of VYVGART Hytrulo Injection for Subcue Use in Generalized MG
argenx website
June 21, 2023
Another effective treatment for generalized myasthenia gravis has been approved by the U.S. Food & Drug Administration. argenx has announced that VYVGART® Hytrulo (efgartigimod alfa and hyaluronidase-qvfc), a subcutaneous injection for adult MG patients who are anti-acetylcholine receptor (AChR) antibody positive.
VYVGART Hytrulo is a subcutaneous product combination of efgartigimod alfa, a human IgG1 antibody fragment marketed for intravenous use as VYVGART, and recombinant human hyaluronidase PH20 (rHuPH20), Halozyme’s ENHANZE® drug delivery technology to facilitate subcutaneous delivery of biologics. The product is to be administered subcutaneously by a healthcare professional as a single injection (1,008 mg fixed dose) over 30-90 seconds in cycles of once weekly injections for four weeks.
“The MG Community is energized and excited about another effective FDA-approved treatment available for those diagnosed with generalized MG. Thank you to our industry partner, argenx,” said Samantha Masterson, president and chief executive officer at the Myasthenia Gravis Foundation of America. “The ease and convenience of a subcutaneous injectable treatment will undoubtedly be very well-received by the patient community because the treatment process fits into the daily lives of patients around the country. Patients have an individualized approach and another important option for managing their personally unique symptoms of MG.”
For more information, you can read the news release. Check out the official website brought to you by argenx, vyvgarthytrulo.com.
Dr. James Howard on Complications with Myasthenia Gravis
AJMC.com
May 26, 2023
Current myasthenia gravis therapies can create other complications, said James F. Howard Jr, MD, professor of neurology at the University of North Carolina at Chapel Hill, former chief of the Neuromuscular Disorders Division, and former James F. Howard Distinguished Professor of Neuromuscular Disease.
Clinicoserological Insights into Patients with Immune Checkpoint Inhibitor-induced Myasthenia Gravis
Annals of Clinical and Translational Neurology
March 2023
Gianvito Masi, MD and Kevin C O’Connor, PhD
Cases of myasthenia gravis (MG) have been recently described as rare but life-threatening adverse events following the administration of immune checkpoint inhibitors (ICI), a novel type of cancer immunotherapy. Patients with ICI-MG often test positive for acetylcholine receptor (AChR) autoantibodies, but unlike idiopathic MG, the role of AChR autoantibodies in ICI-MG pathology is unknown. To address this, we studied a cohort of ICI-MG patients by functionally profiling their AChR autoantibodies. We found that a subset of patients may harbor AChR autoantibodies with molecular features similar to those of idiopathic MG. In other cases, however, such autoantibodies lack overt pathogenic potential, suggesting alternative factors as key mediators of disease. These findings have direct clinical implications, as they challenge the role of AChR autoantibody testing in establishing definite ICI-MG diagnoses and corroborate the need for a thorough assessment when evaluating ICI-related adverse events.
Read entire published paper HERE.