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Myasthenia gravis

Disease ID:651
Name:Myasthenia gravis
Associated with:1 target
4 immuno-relevant ligands
Myasthenia gravis is an autoimmune disease caused by auto-antibody attack on nicotinic acetylcholine receptors at the neuro-muscular junction. This disrupts nerve-muscle communication and results in muscle weakness and fatigue. Treatment has historically been limited to nonselective, chronic immunosuppressive therapies which have long-term toxicities. More selective and targeted therapies are now being developed, including the monoclonal antibody eculizumab (approved) and an investigational CAR-T cell therapy that targets antibody-producing plasma cells (Descartes-08; Cartesian Therapeutics; Phase 1/2).
Database Links
Disease Ontology: DOID:437
OMIM: 254200
Orphanet: ORPHA589


nicotinic acetylcholine receptor α1 subunit
Role:  Target for autoantibodies
References:  4


Key to terms and symbols Click ligand name to view ligand summary Click column headers to sort
Ligand References Clinical and Disease comments
efgartigimod alfa 1
Immuno Disease Comments: Phase 2 clinical trial NCT02965573 in MG patients has been completed. FDA orphan drug for MG (granted Sept 2017)
Clinical Use: The reference version of efgartigimod (efgartigimod alfa-fcab; ARGX-113) was advanced to clinical evaluations in patients with myasthenia gravis (MG), pemphigus vulgaris and primary immune thrombocytopenia. Click here to view's registered ARGX-113 trials. ARGX-113 was granted FDA orphan drug designation for the treatment of MG in September 2017 [1]. In December 2021 the US FDA approved efgartigimod alfa-fcab (Vyvgart®) as a treatment for MG. EMA authorisation for Vyvgart® was issued in 2022. | View clinical data
Immuno Disease Comments: Phase 2 clinical candidate for myasthenia gravis- see NCT03052751
Clinical Use: Rozanolixizumab (as research code UCB7665) was progressed into clinical trial in patients with myasthenia gravis and primary immune thrombocytopenia. EMA orphan designation as a treatment for myasthenia gravis has been in place since 2019.
First FDA approval was granted in June 2023, with rozanolixizumab authorised to treat patients with generalized myasthenia gravis who are positive for anti-acetylcholine receptor or anti-muscle-specific tyrosine kinase antibodies [3]. | View clinical data
Immuno Disease Comments: Phase 2 clinical candidate for moderate to severe Myasthenia gravis- see NCT02565576
Clinical Use: CFZ533 is being evaluated in clinical trials as a therapy for some autoimmune conditions and for preventing/reducing solid organ transplant rejection. Click here to link to's full list of CFZ533 trials. | View clinical data
Bioactivity Comments: CFZ533 (mAb1) shows no agonist activity in an in vitro assay using isolated human PBMCs, blocks -mediated PBMC proliferation (IC50 58 ng/ml), inhibits CD40L-mediated TNF-α release from human monocyte derived dendritic cells (IC50 40 ng/ml), and has ADCC activity of < 1% (measuring specific lysis) [2]. | View biological activity
Immuno Disease Comments: Approved to treat generalized myasthenia gravis in patients with anti-acetylcholine receptor autoantibodies (granted in 2017 by FDA)
Clinical Use: Used to treat paroxysmal nocturnal hemoglobinuria (PNH). In the UK, NICE (National Institute for Health and Care Excellence) has approved eculizumab as a treatment for the rare, but fatal blood disorder, atypical haemolytic uraemic syndrome (aHUS). This decision was reached because eculizumab represents a significant innovation for a disease with a high unmet clinical need. Eculizumab treatment is expected to increase life expectancy of aHUS patients by decades. The FDA expanded approval in October 2017, to include treatment of patients with generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor (AchR) antibody-positive.

SARS-C0V-2 and COVID-19: Eculizumab has been entered into clinical trials that aim to determine its ability to combat the dysregulated immune response that drives organ damage (lung and other organs) in patients with severe COVID-19. | View clinical data
Bioactivity Comments: Eculizumab binds to and blocks the cleavage of the complement protein C5, thereby preventing the formation of . The terminal half-life of eculizumab is 11.3 ± 3.4 days [5]. | View biological activity


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1. FDA. human anti-neonatal Fc receptor IgG1 Fc fragment. Accessed on 25/01/2018. Modified on 25/01/2018.,

2. Heusser C, Rush J, Vincent K. (2012) Silent fc variants of anti-cd40 antibodies. Patent number: WO2012065950. Assignee: Novartis Ag. Priority date: 15/11/2010. Publication date: 24/05/2012.

3. Hoy SM. (2023) Rozanolixizumab: First Approval. Drugs, 83 (14): 1341-1347. [PMID:37656420]

4. Lindstrom JM. (2000) Acetylcholine receptors and myasthenia. Muscle Nerve, 23 (4): 453-77. [PMID:10716755]

5. Rother RP, Rollins SA, Mojcik CF, Brodsky RA, Bell L. (2007) Discovery and development of the complement inhibitor eculizumab for the treatment of paroxysmal nocturnal hemoglobinuria. Nat Biotechnol, 25 (11): 1256-64. [PMID:17989688]