Biomedicines, Vol. 13, Pages 2975: Efgartigimod for Generalized Myasthenia Gravis and Beyond: A Narrative Review of Its Pharmacological Profile, Clinical Utility, and Expanding Applications

Biomedicines doi: 10.3390/biomedicines13122975

Authors:
Ghaith K. Mansour
Leen Alangari
Leen Khosyfan
Reem Alhammad
Ahmad W. Hajjar

Efgartigimod is a novel neonatal Fc receptor (FcRn) antagonist that reduces pathogenic immunoglobulin G (IgG) autoantibodies, offering a targeted therapeutic approach for generalized myasthenia gravis (gMG) and other antibody-mediated autoimmune diseases. This narrative review synthesizes clinical trial data, pharmacological insights, and real-world evidence to evaluate efgartigimod’s efficacy, safety, and emerging applications. Phase 3 randomized controlled trials and extension studies demonstrate rapid and sustained improvements in muscle strength and patient-reported outcomes with a favorable safety profile, including reduced reliance on corticosteroids and intravenous immunoglobulin (IVIg). Additionally, observational studies highlight its expanding utility in diverse IgG-mediated disorders such as immune thrombocytopenia (ITP) and autoimmune encephalitis. Efgartigimod thus represents a paradigm shift in autoimmune disease management, enabling precision immunomodulation with the potential for broad clinical impact and improved patient quality of life (QOL).

Source link

Ghaith K. Mansour www.mdpi.com