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Disease

Myasthenia Gravis

Late-stage therapeutic developmentEmerging research
MeSHMyasthenia Gravis
1
Publications
19
Clinical trials
12
Associated genes
12
Related proteins
2023
Latest publication
Current focus
Acetylcholinesterase biologyComplement c5 biologyTherapeutic developmentInflammation & immunity
Latest activity
beta

Recent clinical, regulatory, research and industry developments relating to this disease.

A Randomized, Double-Blinded, Placebo-Controlled, Phase 3, Parallel-Group Design Study Evaluating the Efficacy and Safety of Efgartigimod IV in Adult Participants With Acetylcholine Receptor Binding Antibody Seronegative Generalized Myasthenia Gravis

Clinical trial2026-08-10Results posted · ClinicalTrials.gov

A Phase 1, Multicenter, Single-arm, Dose-escalation Study of CC-97540 (BMS-986353), CD19-Targeted NEX-T Chimeric Antigen Receptor (CAR) T Cells, Evaluating Safety and Tolerability in Participants With Autoimmune Neurological Diseases: Relapsing Forms of Multiple Sclerosis (RMS), Progressive Forms of Multiple Sclerosis (PMS), or Refractory Myasthenia Gravis (MG).

Clinical trial2026-08-03Status changed to Active, not recruiting · ClinicalTrials.gov

Marketing authorisation: Imaavy (EMA)

Regulatory2025-11-28EMA

Marketing authorisation: Rystiggo (EMA)

Regulatory2024-01-05EMA

Marketing authorisation: Zilbrysq (EMA)

Regulatory2023-12-01EMA

What's happening now

An analyst briefing on current research, clinical, regulatory and industry activity surrounding this disease.

Executive briefingUpdating summary…Momentum: Moderate
Key developments
  • 5 clinical trials expected to report results, the earliest in Q2 2027.
  • 11 industry developments reported.
Upcoming Milestones
Clinical Milestones10View all 10
Results posted 3
Results expected 5
Recently completed 2
+2 more in the activity timeline below
Industry & Market11View all 11
+6 more in the activity timeline below
Regulatory Updates1View
  • 2025-11-28Original application approval — NipocalimabImaavy is indicated as an add-on to standard therapy for the treatment of generalised myasthenia gravis (gMG) in adult and adolescent patients aged 12 years of age and older who are anti‑acetylcholine receptor (AChR) or anti-muscle-specific tyrosine kinase (MuSK) antibody positive.
Activity timeline22

Therapeutic landscape

Therapies with a regulatory footing for this condition, alongside the wider set of agents co-studied with it in the literature.

Approved & established therapies
Nipocalimabapproved

Original application approval — Imaavy is indicated as an add-on to standard therapy for the treatment of generalised mya… (2025)

Rozanolixizumabapproved

Original application approval — Rystiggo is indicated as an add-on to standard therapy for the treatment of generalised m… (2024)

Zilucoplanapproved

Original application approval — Zilbrysq is indicated as an add-on to standard therapy for the treatment of generalised m… (2023)

Efgartigimod alfaapproved

Original application approval — Vyvgart is indicated as- an add-on to standard therapy for the treatment of adult patient… (2022)

Azathioprineapproved

Original application approval — Jayempi is indicated in combination with other immunosuppressive agents for the prophylax… (2021)

Ravulizumabapproved

Original application approval — Paroxysmal nocturnal haemoglobinuria (PNH)Ultomiris is indicated in the treatment of adul… (2019)

Eculizumabapproved

Original application approval — Soliris is indicated in adults and children for the treatment of: Paroxysmal nocturnal h… (2007)

Clinical trials

15 sponsors · 1 new · 2 completed in the last 12 months (net +1)

The current development programme across all trial phases.

Clinical programme
19
All trials
6
Active
17
Late-stage
6
Completed
Late-stage studies

A Randomized, Double-blind, Placebo-controlled Phase III Study to Evaluate the Efficacy, Safety, and Tolerability of Remibrutinib in Patients With Generalized Myasthenia Gravis, Followed by an Open-label Extension Phase

Phase 3 · Recruiting · Novartis Pharmaceuticals

A Phase 3 Global, Multicenter, Randomized, Double-blind, Placebo-controlled Study to Demonstrate the Efficacy, Safety, and Tolerability of Claseprubart (DNTH103) in Patients With Generalized Myasthenia Gravis (EMERGE)

Phase 3 · Recruiting · Dianthus Therapeutics

Immediate Corticosteroid Therapy and Rituximab to Prevent Generalization in Ocular Myasthenia: a PROBE Multicenter Open-label Randomized Controlled Trial.

Phase 3 · Recruiting · Fondation Ophtalmologique Adolphe de Rothschild

A Randomized, Double-blind, Placebo-controlled Phase III Study to Evaluate the Efficacy, Safety, and Tolerability of Iptacopan in Patients With Generalized Myasthenia Gravis, Followed by an Open-label Extension Phase

Phase 3 · Active, not recruiting · Novartis Pharmaceuticals

A Randomized, Double-blind, Multicenter, Placebo-controlled Phase 3 Study With Open-label Period to Evaluate the Efficacy and Safety of Inebilizumab in Adults With Myasthenia Gravis

Phase 3 · Active, not recruiting · Amgen

A Phase 3, Randomized, Double-Blind, Placebo-Controlled, 3-Arm, 3-Period Study to Assess the Efficacy and Safety of a New Formulation of Oral Cladribine Compared With Placebo in Participants With Generalized Myasthenia Gravis (MyClad)

Phase 3 · Recruiting · Merck Healthcare KGaA, Darmstadt, Germany, an affiliate of Merck KGaA, Darmstadt, Germany
Recruiting

A Randomized, Double-blind, Placebo-controlled Phase III Study to Evaluate the Efficacy, Safety, and Tolerability of Remibrutinib in Patients With Generalized Myasthenia Gravis, Followed by an Open-label Extension Phase

Phase 3 · Recruiting · Novartis Pharmaceuticals

A Phase 3 Global, Multicenter, Randomized, Double-blind, Placebo-controlled Study to Demonstrate the Efficacy, Safety, and Tolerability of Claseprubart (DNTH103) in Patients With Generalized Myasthenia Gravis (EMERGE)

Phase 3 · Recruiting · Dianthus Therapeutics

Immediate Corticosteroid Therapy and Rituximab to Prevent Generalization in Ocular Myasthenia: a PROBE Multicenter Open-label Randomized Controlled Trial.

Phase 3 · Recruiting · Fondation Ophtalmologique Adolphe de Rothschild

A Phase 3, Randomized, Double-Blind, Placebo-Controlled, 3-Arm, 3-Period Study to Assess the Efficacy and Safety of a New Formulation of Oral Cladribine Compared With Placebo in Participants With Generalized Myasthenia Gravis (MyClad)

Phase 3 · Recruiting · Merck Healthcare KGaA, Darmstadt, Germany, an affiliate of Merck KGaA, Darmstadt, Germany
Recently completed

A Phase 3, Multicenter, Open-Label Extension Study of Zilucoplan in Subjects With Generalized Myasthenia Gravis

Phase 3 · Completed · UCB Biopharma SRL

An Open-label, Single-arm, Multi-center Study to Evaluate the Efficacy, Safety, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Eculizumab in Chinese Participants With Refractory Generalized Myasthenia Gravis (gMG)

Phase 3 · Completed · Alexion Pharmaceuticals, Inc.

A Multicenter, Open-Label, Outpatient Study to Evaluate the Safe And Effective Use of a Zilucoplan Auto-Injector Combination Product for Subcutaneous Self-Administration by Study Participants With Generalized Myasthenia Gravis

Phase 3 · Completed · UCB Biopharma SRL

A Multi-center, Randomized, Double-blind, Placebo-controlled, Phase III Study of Telitacicept in Patients With Generalized Myasthenia Gravis

Phase 3 · Completed · RemeGen Co., Ltd.

A Long-term Open-label Extension Study to Evaluate the Safety of HBM9161 (HL161) Subcutaneous Injection in Patients With Generalized Myasthenia Gravis

Phase 3 · Completed · Harbour BioMed (Guangzhou) Co. Ltd.

A Phase 3b, Randomized, Open-label, Parallel-Group Study to Evaluate Different Dosing Regimens of Intravenous Efgartigimod to Maximize and Maintain Clinical Benefit in Patients With Generalized Myasthenia Gravis

Phase 3 · Completed · argenx

Regulatory timeline

Drug regulatory events matched to this condition by indication — EMA.

Product approvals and authorisations
2025emaOriginal application approval — Nipocalimab· Imaavy is indicated as an add-on to standard therapy for the treatment of generalised myasthenia gravis (gMG) in adult and adolescent patients aged 12 years of age and older who are anti‑acetylcholine receptor (AChR) or anti-muscle-specific tyrosine kinase (MuSK) antibody positive. source ↗
2024emaOriginal application approval — Rozanolixizumab· Rystiggo is indicated as an add-on to standard therapy for the treatment of generalised myasthenia gravis (gMG) in adult patients who are anti-acetylcholine receptor (AChR) or anti-muscle-specific tyrosine kinase (MuSK) antibody positive. source ↗
2023emaOriginal application approval — Zilucoplan· Zilbrysq is indicated as an add-on to standard therapy for the treatment of generalised myasthenia gravis (gMG) in adult patients who are anti-acetylcholine receptor (AChR) antibody positive. source ↗
2022emaOriginal application approval — Efgartigimod alfa· Vyvgart is indicated as- an add-on to standard therapy for the treatment of adult patients with generalised Myasthenia Gravis (gMG) who are antiacetylcholine receptor (AChR) antibody positive.- monotherapy for the treatment of adult patients with progressive or relapsing active chronic inflammatory demyelinating polyneuropathy (CIDP) after prior treatment with corticosteroids or immunoglobulins. source ↗
2021emaOriginal application approval — Azathioprine· Jayempi is indicated in combination with other immunosuppressive agents for the prophylaxis of transplant rejection in patients receiving allogenic kidney, liver, heart, lung or pancreas transplants. Azathioprine is indicated in immunosuppressive regimens as an adjunct to immunosuppressive agents that form the mainstay of treatment (basis immunosuppression). Jayempi is used as an immunosuppressant antimetabolite either alone or, more commonly, in combination with other agents (usually corticosteroids) and/ or procedures which influence the immune response. Jayempi is indicated in patients who are intolerant to glucocorticosteroids or if the therapeutic response is inadequate despite treatment with high doses of glucocorticosteroids, in the following diseases: severe active rheumatoid arthritis (chronic polyarthritis) that cannot be kept under control by less toxic agents (disease-modifying anti-rheumatic -medicinal products – DMARDs) auto-immune hepatitis  systemic lupus erythematosus dermatomyositis polyarteritis nodosa pemphigus vulgaris and bullous pemphigoid Behçet’s disease refractory auto-immune haemolytic anaemia, caused by warm IgG antibodies chronic refractory idiopathic thrombocytopenic purpura Jayempi is used for the treatment of moderately severe to severe forms of chronic inflammatory bowel disease (IBD) (Crohn’s disease or ulcerative colitis) in patients in whom glucocorticosteroid therapy is necessary, but where glucocorticosteroids are not tolerated, or in whom the disease is untreatable with other common means of first choice. It is also indicated in adult patients in relapsing multiple sclerosis, if an immunomodulatory therapy is indicated but beta interferon therapy is not possible, or a stable course has been achieved with previous treatment with azathioprine. 3 Jayempi is indicated for the treatment of generalised myasthenia gravis. Depending on the severity of the disease, Jayempi should be given in combination with glucocorticosteroids because of slow onset of action at the beginning of treatment and the glucocorticosteroid dose should be gradually reduced after several months of treatment. source ↗
2019emaOriginal application approval — Ravulizumab· Paroxysmal nocturnal haemoglobinuria (PNH)Ultomiris is indicated in the treatment of adult and paediatric patients with a body weight of 10 kg or above with PNH:- in patients with haemolysis with clinical symptom(s) indicative of high disease activity.- in patients who are clinically stable after having been treated with eculizumab for at least the past 6 months (see section 5.1). Atypical haemolytic uremic syndrome (aHUS)Ultomiris is indicated in the treatment of patients with a body weight of 10 kg or above with aHUS who are complement inhibitor treatment-naïve or have received eculizumab for at least 3 months and have evidence of response to eculizumab (see section 5.1). Generalized myasthenia gravis (gMG)Ultomiris is indicated as an add-on to standard therapy for the treatment of adult patients with gMG who are anti-acetylcholine receptor (AChR) antibody-positive. Neuromyelitis Optica Spectrum Disorder (NMOSD)Ultomiris is indicated in the treatment of adult patients with NMOSD who are anti-aquaporin 4 (AQP4) antibody-positive (see section 5.1). Ultomiris is indicated in the treatment of adult patients with paroxysmal nocturnal haemoglobinuria (PNH):- in patients with haemolysis with clinical symptom(s) indicative of high disease activity.- in patients who are clinically stable after having been treated with eculizumab for at least the past 6 months. Ultomiris is indicated in the treatment of adult patients with atypical haemolytic uremic syndrome (aHUS) who are complement inhibitor treatment-naïve or have received eculizumab for at least 3 months and have evidence of response to eculizumab. source ↗
2007emaOriginal application approval — Eculizumab· Soliris is indicated in adults and children for the treatment of: Paroxysmal nocturnal haemoglobinuria (PNH). Evidence of clinical benefit is demonstrated in patients with haemolysis with clinical symptom(s) indicative of high disease activity, regardless of transfusion history (see section 5.1).  Atypical haemolytic uremic syndrome (aHUS). Soliris is indicated in adults for the treatment of: Refractory generalized myasthenia gravis (gMG) in patients who are anti-acetylcholine receptor (AChR) antibody-positive (see section 5.1). Neuromyelitis optica spectrum disorder (NMOSD) in patients who are anti-aquaporin-4 (AQP4) antibody-positive with a relapsing course of the disease. source ↗

European Medicines Agency (CC BY 4.0). Events are matched to this condition by drug indication text — approvals/updates for drugs indicated for it, not disease-specific acts.

Research activity

1 paper

Key research shaping understanding of this disease, combining the latest publications with the most influential evidence.

Recent publications

Peripheral immune landscape for hypercytokinemia in myasthenic crisis utilizing single-cell transcriptomics.

Journal of translational medicine · 2023 · 22 cites
Major themes2
  • Cytokine Release Syndrome1
  • Myasthenia Gravis1
Leading journals1
  • Journal of translational medicine1
Leading researchers8
  • Huan X1
  • Luo F1
  • Luo S1
  • Song J1
  • Su M1
  • Xi J1
  • Yan C1
  • Zhao C1
Affiliations (unnormalised)2
  • Huashan Hospital1
  • Huashan Rare Disease Center and Department of Neurology1

Associated genes

12 matches

Genes associated with this disease in the canonical knowledge graph (Open Targets evidence). Number shows the association score.

KCND30.63KCNS30.61FCGRT0.61KCNH50.61ACHE0.61KCNV20.59C50.59KCNA40.57KCNA10.57KCNA30.56AGBL10.41EPHB10.39

Disease biology

12 matches

Proteins whose encoding gene is associated with this disease, reached through the canonical gene→disease graph. Number shows the gene's association score.

A-type voltage-gated potassium channel KCND3via KCND30.63Delayed-rectifier potassium channel regulatory subunit KCNS3via KCNS30.61IgG receptor FcRn large subunit p51via FCGRT0.61Voltage-gated delayed rectifier potassium channel KCNH5via KCNH50.61Acetylcholinesterasevia ACHE0.61Potassium voltage-gated channel subfamily V member 2via KCNV20.59Complement C5via C50.59Potassium voltage-gated channel subfamily A member 4via KCNA40.57Potassium voltage-gated channel subfamily A member 1via KCNA10.57Potassium voltage-gated channel subfamily A member 3via KCNA30.56C-X-C motif chemokine 13via CXCL130.30Annexin A2via ANXA20.23

Reference

Authoritative identity, definition & identifiers.

Defined in MeSH

A disorder of neuromuscular transmission characterized by fatigable weakness of cranial and skeletal muscles with elevated titers of ACETYLCHOLINE RECEPTORS or muscle-specific receptor tyrosine kinase (MuSK) autoantibodies. Clinical manifestations may include ocular muscle weakness (fluctuating, asymmetric, external ophthalmoplegia; diplopia; ptosis; and weakness of eye closure) and extraocular fatigable weakness of facial, bulbar, respiratory, and proximal limb muscles. The disease may remain limited to the ocular muscles (ocular myasthenia). THYMOMA is commonly associated with this condition.

Identifiers
MeSHMyasthenia Gravis
References & data sources
  • Disease identity & definition — NLM Medical Subject Headings (MeSH), public domain
  • Clinical trials — ClinicalTrials.gov (U.S. National Library of Medicine)
  • Research activity — Europe PMC (EMBL-EBI) + OpenAlex-derived paper links
  • Related entities are derived from literature co-mention (studied together) — associative, not causal.