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

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Am J Ther. 2026 Jan-Feb 01;33(1):e46-e49. doi: 10.1097/MJT.0000000000002052.

ABSTRACT

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.

PMID:41505161 | DOI:10.1097/MJT.0000000000002052


Source: https://pubmed.ncbi.nlm.nih.gov/4150516 ... 22+67771e2