When Advocacy Meets Science in the Care of People Living with gMG

For many people living with generalized myasthenia gravis (gMG), everyday tasks can take unexpected effort. The muscles that make walking, speaking or drinking coffee feel effortless one day can suddenly weaken the next. The disease disrupts communication between nerves and muscles, turning routine activities into challenges that can shift from day to day—or even hour to hour.¹

With the FDA approval of UPLIZNA^® (inebilizumab-cdon) for the treatment of gMG in adult patients who are anti-acetylcholine receptor (AChR) or anti-muscle specific tyrosine kinase (MuSK) antibody positive (Ab+), patients and clinicians now have another option for managing this complex and unpredictable autoimmune condition.

A “Snowflake Disease” That Defies Uniformity

Myasthenia gravis affects an estimated 80,000 to 100,000 people in the U.S.,^2,3 with around 85% having the generalized form, or gMG.⁴ Yet no two patients experience it in the same way. Symptoms can vary in ways that are deeply personal.

“Every patient's experience looks different and that's why gMG is called a 'snowflake disease,'” explains Samantha Masterson, longtime patient advocate, president and chief executive officer of the Myasthenia Gravis Foundation of America (MGFA).^* “The way gMG symptoms ebb and flow, and the way they change lives are highly individualized. Treatment options for use in disease management must meet that reality.”

Masterson has spent more than 25 years working in patient advocacy, and she's seen firsthand how difficult and frustrating the path to diagnosis and effective treatment can be.

“It is not a one-size-fits-all situation,” Masterson says. “Individuals with gMG often feel marginalized and unheard by those around them, and there is a critical need for the availability of multiple treatment options due to the unique challenges each individual faces.”

Expanding Options for gMG Care

Against this backdrop of varied experiences and needs, UPLIZNA offers a new approach as the first and only FDA-approved treatment for anti-AChR or anti-MuSK Ab+ gMG that works upstream to target CD19+ B cells, a key source of autoantibody production and subsequent disease activity.^5,6**

In the Phase 3 Myasthenia Gravis Inebilizumab Trial (MINT), UPLIZNA helped people with anti-AChR or anti-MuSK Ab+ gMG achieve meaningful improvements in daily activities. At Week 26, UPLIZNA demonstrated a 1.9-point difference in the Myasthenia Gravis Activities of Daily Living (MG-ADL) score^† compared with placebo (−4.2 vs. −2.2; p<0.0001). UPLIZNA also has a dosing schedule that offers patients six months of infusion-free time between doses. Infusions are twice-yearly following two initial infusions two weeks apart. The most common adverse reactions (at least 10% of patients treated with UPLIZNA and greater than placebo) were headache and infusion-related reactions.^5,6

“At the MGFA, we often hear from community members that treatment schedules are an important consideration in how they manage their disease,” explains Masterson. “The idea of having half a year in between treatments could be meaningful to many as they think about what fits their lifestyle.”

Advocacy and Research: Stronger Together

For Masterson, this milestone underscores the impact of research and advocacy working together.

“It is a significant moment for people living with gMG,” she says. “It represents the outcome of years of dedicated research and collective advocacy efforts, providing individuals with another option to help manage their disease.”

Amgen works with organizations like the MGFA to ensure that patient perspectives directly inform research priorities and educational initiatives. “Collaboration among patients, advocacy, industry and researchers is how we move results forward,” Masterson notes.

Continuing Our Mission in Rare Disease Care

The FDA approval of UPLIZNA in adults with anti-AChR or anti-MuSK Ab+ gMG marks a meaningful milestone—one that furthers Amgen's mission to serve patients.

“This approval reflects not only our deep scientific understanding of gMG but also Amgen's commitment to advancing care options in rare and complex autoimmune diseases,” says Sumita Bhatta, Vice President and Global Therapeutic Area Head, Rare Disease. “The approval of UPLIZNA in gMG is the third FDA approval for the medicine and adds to a growing foundation of work aimed at delivering meaningful therapies for patient populations with significant unmet need.”

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Footnotes

*The MGFA is an independent nonprofit organization. Amgen has supported MGFA programs through charitable contributions.

**The mechanism by which UPLIZNA exerts its therapeutic effect in gMG is unknown.⁶

†The MG-ADL scale assesses the impact of gMG on daily functions of eight signs or symptoms that are typically affected in gMG. Impairment in each symptom is graded from 0 (normal) to 3 (most severe impairment). The scores are added together to provide a total from 0 to 24, with higher scores indicating more severe disease. A 2-point improvement in the MG-ADL score is considered a clinically meaningful improvement.^5,7

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INDICATIONS

UPLIZNA^® (inebilizumab-cdon) is indicated in adult patients for the treatment of: anti-aquaporin-4 (AQP4) antibody positive neuromyelitis optica spectrum disorder (NMOSD); Immunoglobulin G4-related disease (IgG4-RD); anti-acetylcholine receptor (AChR) or anti-muscle specific tyrosine kinase (MuSK) antibody positive (Ab+) generalized myasthenia gravis (gMG).

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IMPORTANT SAFETY INFORMATION

CONTRAINDICATIONS

UPLIZNA^® (inebilizumab-cdon) is contraindicated in patients with a history of a life-threatening infusion reaction to UPLIZNA, active hepatitis B infection, or active or untreated latent tuberculosis.

WARNINGS AND PRECAUTIONS

• Infusion Reactions: Infusion reactions, including anaphylaxis, can occur. Symptoms can include headache, nausea, somnolence, dyspnea, fever, myalgia, rash, or palpitations. Infusion reactions were observed in 9.3%, 7.4%, and 10.1% of patients treated with UPLIZNA during the randomized controlled periods (RCPs) of Study 1 in patients with NMOSD, Study 2 in patients with IgG4-RD, and Study 3 in patients with gMG, respectively. Infusion reactions were most common with the first infusion but were also observed during subsequent infusions.

  Administer pre-medication with a corticosteroid, an antihistamine, and an antipyretic. For life-threatening infusion reactions, immediately and permanently stop UPLIZNA and administer appropriate supportive treatment. For less severe infusion reactions, management may involve temporarily stopping the infusion, reducing the infusion rate, and/or administering symptomatic treatment.

• Infections: Serious, including life-threatening or fatal, bacterial, fungal, and new or reactivated viral infections have been observed during and following completion of treatment with B-cell depleting therapies, including UPLIZNA. The most common infections reported by UPLIZNA-treated patients in the NMOSD randomized and open-label clinical trial periods for NMOSD were urinary tract infection (20%), nasopharyngitis (13%), upper respiratory tract infection (8%), and influenza (7%). In the IgG4-RD RCP, the most common infections reported by UPLIZNA-treated patients were urinary tract infection, influenza, and pneumonia. In the gMG RCP, the most common infections reported by UPLIZNA-treated patients were urinary tract infection and nasopharyngitis. Delay UPLIZNA administration in patients with an active infection until the infection is resolved.

  Possible Increased Risk of Immunosuppressant Effects with Other Immunosuppressants: If combining UPLIZNA with another immunosuppressive therapy, consider the potential for increased immunosuppressive effects.

  Hepatitis B Virus (HBV) Reactivation: HBV reactivation has been observed with B-cell-depleting therapies, including UPLIZNA. Fulminant hepatitis, hepatic failure, and death caused by HBV reactivation have occurred in patients treated with B-cell depleting therapies. HBV reactivation was observed in a patient treated with UPLIZNA during the gMG clinical trial and in the postmarketing setting. Patients with active or chronic HBV infection were excluded from clinical trials. Perform HBV screening in all patients before initiation of treatment. Do not administer to patients with active HBV confirmed by positive results for HBsAg and anti-HB tests. For patients who are negative for HBsAg and positive for HBcAb, or who are carriers of HBV (i.e., HBsAg+), consult liver disease experts before starting and during treatment.

  Progressive Multifocal Leukoencephalopathy (PML): Although no confirmed cases of PML were identified in UPLIZNA clinical trials, JC virus infection resulting in PML has been observed in patients treated with other B-cell-depleting antibodies and other therapies that affect immune competence. In UPLIZNA clinical trials one subject died following the development of new brain lesions for which a definitive diagnosis could not be established, though the differential diagnosis included an atypical NMOSD relapse, PML, or acute disseminated encephalomyelitis. At the first sign or symptom suggestive of PML, withhold UPLIZNA and perform an appropriate diagnostic evaluation. MRI findings may be apparent before clinical signs or symptoms. Typical symptoms associated with PML are diverse, progress over days to weeks, and include progressive weakness on one side of the body or clumsiness of limbs, disturbance of vision, and changes in thinking, memory, and orientation leading to confusion and personality changes.

  Tuberculosis
  Patients should be evaluated for tuberculosis risk factors and tested for latent infection prior to initiating UPLIZNA. Consider anti-tuberculosis therapy prior to initiation of UPLIZNA in patients with a history of latent active tuberculosis in whom an adequate course of treatment cannot be confirmed, and for patients with a negative test for latent tuberculosis but having risk factors for tuberculosis infection. Consult infectious disease experts regarding whether initiating anti-tuberculosis therapy is appropriate before starting treatment.

  Vaccinations
  Administer all immunizations according to immunization guidelines at least 4 weeks prior to initiation of UPLIZNA. The safety of immunization with live or live-attenuated vaccines following UPLIZNA therapy has not been studied, and vaccination with live-attenuated or live vaccines is not recommended during treatment and until B-cell repletion.

  Vaccination of Infants Born to Mothers Treated with UPLIZNA During Pregnancy
  In infants of mothers exposed to UPLIZNA during pregnancy, do not administer live or live-attenuated vaccines before confirming recovery of B-cell counts in the infant. Depletion of B cells in these exposed infants may increase the risks from live or live-attenuated vaccines. Non-live vaccines, as indicated, may be administered prior to recovery from B-cell and immunoglobulin level depletion, but consultation with a qualified specialist should be considered to assess whether a protective immune response was mounted.

• Reductions in Immunoglobulins: There may be a progressive and prolonged hypogammaglobulinemia or decline in the levels of total and individual immunoglobulins such as immunoglobulins G and M (IgG and IgM) with continued UPLIZNA treatment. Monitor the levels of quantitative serum immunoglobulins during treatment with UPLIZNA, especially in patients with opportunistic or recurrent infections, and until B-cell repletion after discontinuation of therapy. Consider discontinuing UPLIZNA therapy if a patient with low immunoglobulin G or M develops a serious opportunistic infection or recurrent infections, or if prolonged hypogammaglobulinemia requires treatment with intravenous immunoglobulins.

• Fetal Risk: Based on animal data, UPLIZNA can cause fetal harm due to B-cell lymphopenia and reduce antibody response in offspring exposed to UPLIZNA even after B-cell repletion. Transient peripheral B-cell depletion and lymphocytopenia have been reported in infants born to mothers exposed to other B-cell-depleting antibodies during pregnancy. Advise females of reproductive potential to use effective contraception while receiving UPLIZNA and for at least 6 months after the last dose.

ADVERSE REACTIONS

• The most common adverse reactions (at least 10% of patients treated with UPLIZNA and greater than placebo): urinary tract infection and arthralgia in NMOSD; urinary tract infection and lymphopenia in IgG4-RD; headache and infusion-related reactions in gMG.

Please see UPLIZNA^® full Prescribing Information.

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References

1. Myasthenia Gravis Foundation of America. What is Myasthenia Gravis? MGFA. Updated 2025. Accessed December 10, 2025. https://myasthenia.org/understanding-mg/what-is-myasthenia-gravis/
2. Ye Y, Murdock DJ, Chen C, Liedtke W, Knox CA. Epidemiology of myasthenia gravis in the United States. Front Neurol. 2024;15:1339167.
3. Rodrigues E, Umeh E, Aishwarya, Navaratnarajah N, Cole A, Moy K. Incidence and prevalence of myasthenia gravis in the United States: a claims-based analysis. Muscle Nerve. 2024;69(2):166-171.
4. Engel-Nitz NM, Boscoe A, Wolbeck R, Johnson J, Silvestri NJ. Burden of illness in patients with treatment refractory myasthenia gravis. Muscle Nerve. 2018. doi:10.1002/mus.26114.
5. Nowak R, Benatar M, Ciafaloni E, et al. A phase 3 trial of inebilizumab in generalized myasthenia gravis. N Engl J Med. 2025;392(23):2309-2320.
6. UPLIZNA^® (inebilizumab-cdon) prescribing information, Amgen.
7. Muppidi S, Silvestri NJ, Tan R, Riggs K, Leighton T, Phillips GA. Utilization of MG-ADL in myasthenia gravis clinical research and care. Muscle Nerve. 2022;65(6):630-639. doi:10.1002/mus.27476.