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

Area of Science:

• Neurology
• Immunology
• Pharmacology

Background:

• Myasthenia gravis (MG) is a rare autoimmune neuromuscular disorder with unmet needs.
• FcRn and complement inhibitors represent a paradigm shift in generalized MG treatment.

Purpose of the Study:

• To review the clinical efficacy, safety, and practical implications of novel FcRn and complement inhibitors for generalized MG.
• To synthesize data from clinical trials and literature published between 2013 and 2025.

Main Methods:

• Review of FDA-approved FcRn inhibitors (efgartigimod, rozanolixizumab, nipocalimab).
• Review of FDA-approved complement inhibitors (eculizumab, ravulizumab, zilucoplan).
• Analysis of clinical trial data and scientific literature.

Main Results:

• FcRn inhibitors significantly improve MG-ADL scores in anti-AChR and MuSK antibody-positive patients.
• Rozanolixizumab and nipocalimab are approved for MuSK-MG; complement inhibitors show rapid efficacy in refractory anti-AChR MG.
• Adverse events include infections and hypersensitivity reactions, necessitating vaccination and monitoring.

Conclusions:

• Novel FcRn and complement inhibitors provide personalized, effective options for MG management.
• Challenges in cost and accessibility remain significant barriers.
• Future research should focus on long-term outcomes and identifying predictive biomarkers.