Mechanism of action and clinical research progress of novel targeted biologics for myasthenia gravis
- VernacularTitle:新型靶向生物制剂治疗重症肌无力的作用机制及临床研究进展
- Author:
Zijing ZHOU
1
;
Jinpin LI
1
Author Information
1. Dept. of Neurology,the First Affiliated Hospital of Guangxi Medical University,Nanning 530021,China
- Publication Type:Journal Article
- Keywords:
myasthenia gravis;
novel targeted biologics;
B-cell targeted therapy;
complement inhibitors;
FcRn antagonists
- From:
China Pharmacy
2025;36(23):3001-3006
- CountryChina
- Language:Chinese
-
Abstract:
The pharmacological treatment strategy for myasthenia gravis (MG) is transitioning from nonspecific immunosuppression with broad-spectrum immunosuppressive agents to precise therapy with novel targeted biologics. This review summarizes the mechanisms of action and clinical research progress of novel targeted biologics, revealing that these agents can improve MG symptom through a three-tiered mechanism:“ upstream inhibition of B-cell activation and pathogenic autoantibody production, midstream suppression of excessive complement activation, and downstream acceleration of pathogenic immunoglobulin G degradation”. Clinically, CD20 monoclonal antibodies (e.g. rituximab, ofatumumab), belimumab, telitacicept, complement inhibitors (e.g. eculizumab, ravulizumab), and neonatal Fc receptor antagonists (e.g. efgartigimod α) demonstrate efficacy via these mechanisms, while interleukin-6 inhibitors (e. g. tocilizumab) show promising results by suppressing inflammatory responses. However, current limitations include high costs leading to limited accessibility, drug efficacy restricted to specific antibody subtypes, and insufficient clinical data for special populations. Future research should deepen mechanistic studies, promote large-sample, long-term follow-up clinical trials, and explore personalized treatment strategies based on MG subtypes to provide more precise and accessible therapeutic options for MG patients.