Efgartigimod: A First-in-class Investigational Antibody Fragment for the Treatment of Generalized Myasthenia Gravis

Author:

Vanoli Fiammetta,Mantegazza Renato, , ,

Abstract

Myasthenia gravis (MG) is a rare autoimmune disorder characterized by fatigue and muscle weakness. About 85–90% of patients with generalized MG display pathogenic immunoglobulin (Ig)G antibodies against the skeletal muscle nicotinic acetylcholine receptor (AChR), the muscle-specific tyrosine kinase or the lipoprotein receptor-related protein, which all exert their effect by disrupting neuromuscular transmission. Therapy for MG includes immunomodulation and non-specific immunosuppression; the latter comprises corticosteroids and non-steroidal immunosuppressive therapies, which non-selectively suppress the immune system and are frequently accompanied by burdensome side effects. This, together with the fact that up to 20% of patients are refractory to immunosuppressive therapy, highlights a compelling unmet need for more effective and better-tolerated therapies. Efgartigimod, a humanized IgG1-derived fragment crystallizable region that competitively blocks the neonatal fragment crystallizable receptor, holds great promise in meeting this need, having good tolerability and a more targeted effect. Efgartigimod has been recently approved by the US Food and Drug Administration (FDA) for the treatment of AChR-positive patients with generalized MG, making it the first FDA-approved neonatal fragment crystallizable receptor antagonist. This review focuses on the clinical development of efgartigimod, which offers an encouraging new therapeutic option for generalized MG.

Funder

This article is published under the Creative Commons Attribution Non-commercial License.

Publisher

Touch Medical Media, Ltd.

Subject

Neurology (clinical),General Neuroscience

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