Current and Emerging Therapies for Generalized Myasthenia Gravis: Toward Precision Medicine

The therapeutic landscape of myasthenia gravis (MG) has changed substantially with advances in immunopathogenesis and the introduction of targeted, mechanism-based immunotherapies. For decades, treatment relied primarily on corticosteroids and conventional immunosuppressive agents, which are effective but often limited by delayed onset, long-term toxicity, and incomplete disease control. This review summarizes current and emerging therapies for generalized MG, focusing on mechanisms of action, clinical efficacy, safety, and practical considerations, while also addressing real-world evidence, treatment switching and sequencing, and factors relevant to individualized decision-making. Complement-targeted therapies, including eculizumab, ravulizumab, zilucoplan, gefurulimab, and the emerging RNA interference-based agent cemdisiran, and neonatal Fc receptor (FcRn) inhibitors, including efgartigimod, rozanolixizumab, and nipocalimab, have substantially expanded therapeutic options. B cell-targeted therapies, particularly rituximab and inebilizumab, have also broadened the therapeutic landscape, especially for refractory disease and muscle-specific kinase antibody-positive MG. Emerging approaches, including chimeric antigen receptor T cell and chimeric autoantibody receptor T cell therapies, plasma cell-directed therapies, regulatory T cell-based therapies, and antigen-specific immune tolerance strategies, may further improve long-term disease control by targeting the underlying autoimmune process. Important unmet needs remain, including limited treatment accessibility, insufficient evidence for specific patient populations, and the lack of validated predictive biomarkers. In the absence of direct head-to-head trials or validated predictive biomarkers, individualized treatment selection should integrate autoantibody status, disease activity and urgency, clinical characteristics, prior treatment response and toxicity, comorbidities and safety considerations, treatment burden, reproductive considerations, patient preferences, and access. Future progress will depend on the development and validation of predictive biomarkers to enable biomarker-guided precision medicine, together with advances in targeted immunotherapy and immune tolerance-inducing strategies, with the ultimate goal of achieving durable remission and improving long-term outcomes and quality of life.