dc.contributor.author | Howard, James F. | |
dc.contributor.author | Vissing, John | |
dc.contributor.author | Gilhus, Nils Erik | |
dc.contributor.author | Leite, Maria Isabel | |
dc.contributor.author | Utsugisawa, Kimiaki | |
dc.contributor.author | Duda, Petra W. | |
dc.contributor.author | Farzaneh-Far, Ramin | |
dc.contributor.author | Murai, Hiroyuki | |
dc.contributor.author | Wiendl, Heinz | |
dc.date.accessioned | 2021-08-23T09:07:58Z | |
dc.date.available | 2021-08-23T09:07:58Z | |
dc.date.created | 2021-08-16T11:38:28Z | |
dc.date.issued | 2021 | |
dc.identifier.issn | 1354-3784 | |
dc.identifier.uri | https://hdl.handle.net/11250/2770699 | |
dc.description.abstract | Generalized myasthenia gravis (gMG) is an autoimmune disorder in which pathogenic autoantibodies damage the neuromuscular junction, causing disabling or life-threatening muscle weakness. Most treatments nonspecifically inhibit aspects of the immune system, do not directly address the causal mechanisms of tissue damage, and often have side-effect profiles that negatively impact patients. Understanding of the central pathogenic role of the complement cascade in gMG is advancing, and a new complement-targeting treatment is under investigation.
Areas covered
We provide an overview of gMG etiology, the complement cascade, current treatments, and the investigational gMG therapy zilucoplan. Zilucoplan is a small, subcutaneously administered, macrocyclic peptide that inhibits cleavage of complement component C5 and the subsequent formation of the membrane attack complex.
Expert opinion
In a randomized, double-blind, placebo-controlled, phase 2 clinical trial, zilucoplan demonstrated clinically meaningful complement inhibition in patients with acetylcholine receptor-positive gMG. Zilucoplan, a first-of-its-kind cyclic peptide targeting C5, appears to be a therapeutic option for the treatment of gMG based on available pharmacokinetic/pharmacodynamic data and phase 1 and 2 efficacy, safety, and tolerability data with limited long-term follow-up. Zilucoplan use earlier in the treatment paradigm would be suitable in this population should phase 3 efficacy and safety data be equally favorable. | en_US |
dc.language.iso | eng | en_US |
dc.publisher | Taylor and Francis | en_US |
dc.rights | Attribution-NonCommercial-NoDerivatives 4.0 Internasjonal | * |
dc.rights.uri | http://creativecommons.org/licenses/by-nc-nd/4.0/deed.no | * |
dc.title | Zilucoplan: An Investigational Complement C5 Inhibitor for the Treatment of Acetylcholine Receptor Autoantibody–Positive Generalized Myasthenia Gravis | en_US |
dc.type | Journal article | en_US |
dc.type | Peer reviewed | en_US |
dc.description.version | publishedVersion | en_US |
dc.rights.holder | Copyright 2021 The Author(s) | en_US |
cristin.ispublished | true | |
cristin.fulltext | original | |
cristin.qualitycode | 1 | |
dc.identifier.doi | 10.1080/13543784.2021.1897567 | |
dc.identifier.cristin | 1926254 | |
dc.source.journal | Expert Opinion on Investigational Drugs | en_US |
dc.source.pagenumber | 483-493 | en_US |
dc.identifier.citation | Expert Opinion on Investigational Drugs. 2021, 30 (5), 483-493. | en_US |
dc.source.volume | 30 | en_US |
dc.source.issue | 5 | en_US |