Efficacy and Safety of Vemircopan in Generalized Myasthenia Gravis

Is vemircopan efficacious in adults with acetylcholine receptor antibody-positive generalized myasthenia gravis?In this double-blind, multicenter, phase 2 randomized clinical trial, differences in the proportion of participants achieving the primary endpoint (≥2-point reduction from baseline in total Myasthenia Gravis–Activities of Daily Living score for 4 consecutive weeks during the 8-week primary evaluation period, without rescue therapy use) were not statistically significant between vemircopan and placebo groups. No significant differences were observed in secondary efficacy end points either.The trial was terminated, as the minimal efficacy threshold was not met; inhibition of the complement alternative pathway as a potentially viable and efficacious strategy to treat patients with acetylcholine receptor antibody-positive generalized myasthenia gravis requires further investigation in clinical settings.

Corresponding Author: Francesco Saccà, MD, Federico II University of Naples, Via Sergio Pansini 5, Naples, 80131 Italy (francesco.sacca@unina.it).

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