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dc.contributor.authorHoward, James F.
dc.contributor.authorVissing, John
dc.contributor.authorGilhus, Nils Erik
dc.contributor.authorLeite, Maria Isabel
dc.contributor.authorUtsugisawa, Kimiaki
dc.contributor.authorDuda, Petra W.
dc.contributor.authorFarzaneh-Far, Ramin
dc.contributor.authorMurai, Hiroyuki
dc.contributor.authorWiendl, Heinz
dc.description.abstractGeneralized 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.publisherTaylor and Francisen_US
dc.rightsAttribution-NonCommercial-NoDerivatives 4.0 Internasjonal*
dc.titleZilucoplan: An Investigational Complement C5 Inhibitor for the Treatment of Acetylcholine Receptor Autoantibody–Positive Generalized Myasthenia Gravisen_US
dc.typeJournal articleen_US
dc.typePeer revieweden_US
dc.rights.holderCopyright 2021 The Author(s)en_US
dc.source.journalExpert Opinion on Investigational Drugsen_US
dc.identifier.citationExpert Opinion on Investigational Drugs. 2021, 30 (5), 483-493.en_US

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Attribution-NonCommercial-NoDerivatives 4.0 Internasjonal
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