Novel Therapies for Generalized Myasthenia Gravis: Insights Into FcRn and Complement Inhibition.

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.