[Pubmed] Efgartigimod for Generalized Myasthenia Gravis and Beyond: A Narrative Review of Its Pharmacological Profile, C

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Biomedicines. 2025 Dec 4;13(12):2975. doi: 10.3390/biomedicines13122975.

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).

PMID:41462987 | DOI:10.3390/biomedicines13122975


Source: https://pubmed.ncbi.nlm.nih.gov/4146298 ... 22+67771e2