Efgartigimod for Generalized Myasthenia Gravis and Beyond: A Narrative Review of Its Pharmacological Profile, Clinical Utility, and Expanding Applications
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).
Authors
- Reem Alhammad (ORCID: https://orcid.org/0000-0001-7931-6830)
- Ghaith K. Mansour
- Ahmad W. Hajjar (ORCID: https://orcid.org/0009-0009-0024-3751)
- Leen Alangari (ORCID: https://orcid.org/0009-0004-9921-8305)
- Leen Khosyfan
Institutions
- Princess Nourah bint Abdulrahman University (SA)
- Alfaisal University (SA)
- King Saud University (SA)
Publication Details
- Journal
- Biomedicines
- Published
- 2025-12-04
- DOI
- https://doi.org/10.3390/biomedicines13122975
- Primary Topic
- Myasthenia Gravis and Thymoma
- Type
- article
- Field-Weighted Citation Impact
- 0.00