American journal of therapeutics · 2026
Novel Therapies for Generalized Myasthenia Gravis: Insights Into FcRn and Complement Inhibition.
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Samara Verena C, Thottempudi Neeharika, Franke Olivia, Tabaac Burton J
Аннотация
BACKGROUND: Myasthenia gravis (MG) is a rare autoimmune neuromuscular disorder with significant unmet needs despite conventional therapies. Novel FcRn and complement inhibitors have transformed the treatment paradigm for generalized MG.
METHODS: This commentary reviews the clinical efficacy, safety, and practical implications of FDA-approved FcRn inhibitors (efgartigimod, rozanolixizumab, nipocalimab) and complements inhibitors (eculizumab, ravulizumab, zilucoplan), drawing on clinical trial data and literature from 2013 to 2025.
RESULTS: FcRn inhibitors significantly improve MG-ADL scores in anti-AChR and MuSK antibody-positive patients, with rozanolixizumab and nipocalimab uniquely approved for MuSK-MG. Complement inhibitors show rapid efficacy in refractory anti-AChR MG, with zilucoplan enabling self-administration. Adverse events include infections and hypersensitivity reactions. These medications require vaccination and close monitoring.
CONCLUSIONS: These therapies offer personalized, effective options for MG management, though challenges in cost and access remain. Future research should address long-term outcomes and biomarkers.
METHODS: This commentary reviews the clinical efficacy, safety, and practical implications of FDA-approved FcRn inhibitors (efgartigimod, rozanolixizumab, nipocalimab) and complements inhibitors (eculizumab, ravulizumab, zilucoplan), drawing on clinical trial data and literature from 2013 to 2025.
RESULTS: FcRn inhibitors significantly improve MG-ADL scores in anti-AChR and MuSK antibody-positive patients, with rozanolixizumab and nipocalimab uniquely approved for MuSK-MG. Complement inhibitors show rapid efficacy in refractory anti-AChR MG, with zilucoplan enabling self-administration. Adverse events include infections and hypersensitivity reactions. These medications require vaccination and close monitoring.
CONCLUSIONS: These therapies offer personalized, effective options for MG management, though challenges in cost and access remain. Future research should address long-term outcomes and biomarkers.