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

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

  • Biomedicines. 2025 Dec 4;13(12):2975. doi: 10.3390/biomedicines13122975.
Ghaith K Mansour 1 Leen Alangari 2 Leen Khosyfan 2 Reem Alhammad 3 Ahmad W Hajjar 4
Affiliations

Affiliations

  • 1 College of Pharmacy, Alfaisal University, Riyadh 11533, Saudi Arabia.
  • 2 College of Medicine, Princess Nourah Bint Abdulrahman University, Riyadh 11671, Saudi Arabia.
  • 3 College of Medicine, King Saud University, Riyadh 11472, Saudi Arabia.
  • 4 College of Medicine, Alfaisal University, Riyadh 11533, Saudi Arabia.
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).

Keywords

FcRn antagonist; IgG modulation; autoimmune therapy; efgartigimod; gMG.

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