Zilucoplan: a novel therapeutic approach to treat generalized myasthenia gravis

Abstract

Myasthenia gravis (MG) is an autoimmune neuromuscular disease characterized by muscle weakness due to autoantibodies targeting the neuromuscular junction (NMJ). Zilucoplan, a novel complement inhibitor, has shown promise in managing generalized MG (gMG) by blocking the terminal complement cascade. This article provides an overview of Zilucoplan's pharmacological properties, including its mechanism of action, pharmacokinetics, and pharmacodynamics. Clinical trials, including a phase 3 trial (RAISE), have demonstrated Zilucoplan's efficacy in improving muscle strength and function, as measured by the MG-ADL (MG-activities of daily life) score, compared to placebo. The safety profile of Zilucoplan is favorable, with injection-site reactions being the most common adverse event. Notably, Zilucoplan effectively inhibits both wild-type and clinical C5 variants, expanding its potential utility for patients who do not respond well to existing treatments. While further research is needed to assess its long-term safety and efficacy, Zilucoplan represents a valuable addition to the therapeutic armamentarium for managing gMG.