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.2025-11-28

Clinical Milestones10View all 10

Results posted 4

Results expected 5

Recently completed 1

2025-12-29An 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)Completed

+2 more in the activity timeline below

Regulatory Updates1View

2025-11-28Approval — 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 timeline11

2026-07-23ClinicalA 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)Results expected Q3 2028
2026-07-08ClinicalA 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)Results expected Q4 2028
2026-06-23ClinicalA 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 PhaseResults expected Q2 2027
2026-06-01ClinicalA 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 PhaseResults expected Q1 2028
2026-05-27ClinicalA 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 GravisResults posted
2026-05-15ClinicalA 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 GravisResults posted
2026-03-27ClinicalA 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 GravisResults posted
2026-01-22ClinicalA Long-Term, Single-Arm, Open-label, Multicenter Phase 3 Study to Evaluate the Safety and Tolerability of Multiple Subcutaneous Injections of Efgartigimod PH20 SC in Patients With Generalized Myasthenia GravisResults posted
2026-01-21ClinicalImmediate Corticosteroid Therapy and Rituximab to Prevent Generalization in Ocular Myasthenia: a PROBE Multicenter Open-label Randomized Controlled Trial.Results expected Q2 2028
2025-12-29ClinicalAn 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)Completed

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

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

Rozanolixizumabapproved

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

Zilucoplanapproved

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

Efgartigimod alfaapproved

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

Azathioprineapproved

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

Ravulizumabapproved

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

Eculizumabapproved

Accelerated 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

All trials

Active

Late-stage

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

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 · Recruiting · Novartis Pharmaceuticals

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, 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

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

Recruiting

Phase 3 · Recruiting · Novartis Pharmaceuticals

Phase 3 · Recruiting · Dianthus Therapeutics

Phase 3 · Recruiting · Novartis Pharmaceuticals

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

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

Recently completed

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 Long-Term, Single-Arm, Open-label, Multicenter Phase 3 Study to Evaluate the Safety and Tolerability of Multiple Subcutaneous Injections of Efgartigimod PH20 SC in Patients With Generalized Myasthenia Gravis

Phase 3 · Completed · argenx

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.

Phase 3 · Completed · argenx

Regulatory timeline

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

First approvals

2025emaApproval — 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 ↗

2024emaApproval — 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 ↗

2023emaApproval — 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 ↗

2022emaApproval — 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 ↗

2021emaApproval — 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 ↗

2019emaApproval — 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 ↗

2007emaAccelerated 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 papers

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

Most influential

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

Journal of translational medicine · 2023 · 22 cites

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

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.