Work

Efgartigimod for Generalized Myasthenia Gravis and Beyond: A Narrative Review of Its Pharmacological Profile, Clinical Utility, and Expanding Applications

Year: 2025

Type: review

Abstract: 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: Biomedicines

DOI: 10.3390/biomedicines13122975

Authors: Ghaith Mansour, Leen Alangari, Leen Khosyfan, Reem Alhammad, Ahmad Hajjar

Institutions: Alfaisal University, Princess Nourah bint Abdulrahman University, King Saud University

Language: English

Topic: Myasthenia Gravis and Thymoma

Subfield: Neurology

Field: Medicine

Domain: Health Sciences

SDG: Good health and well-being

Open Access status: gold

Estimated APC paid: $3,137