Myasthenia gravis

Disease ID:651
Name:Myasthenia gravis
Associated with:1 target
3 immuno-relevant ligands
Database Links
Disease Ontology: DOID:437
OMIM: 254200
Orphanet: ORPHA589

Targets

nicotinic acetylcholine receptor α1 subunit
Role:  Target for autoantibodies
References:  3

Ligands

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: ARGX-113 has completed Phase 2 trial in patients with myasthenia gravis (MG), and will be evaluated in separate Phase 2 clinical trials for pemphigus vulgaris and primary immune thrombocytopenia. Click here to view ClinicalTrials.gov's registered ARGX-113 trials. ARGX-113 was granted FDA orphan drug designation for the treatment of MG in September 2017 [1]. | View clinical data
rozanolixizumab
Immuno Disease Comments: Phase 2 clinical candidate for myasthenia gravis- see NCT03052751
Clinical Use: Rozanolixizumab (as research code UCB7665) has progressed to Phase 2 clinical trial in patients with myasthenia gravis (NCT03052751) and primary immune thrombocytopenia (NCT02718716). | View clinical data
CFZ533
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 ClinicalTrials.gov'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

References

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1. FDA. human anti-neonatal Fc receptor IgG1 Fc fragment. Accessed on 25/01/2018. Modified on 25/01/2018. www.accessdata.fda.gov, https://www.accessdata.fda.gov/scripts/opdlisting/oopd/detailedIndex.cfm?cfgridkey=527316

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