Rozanolixizumab for Myasthenia Gravis: a breakthrough treatment and future prospects.

Abstract

Myasthenia gravis is a rare chronic autoimmune disorder affecting the post-synaptic neuromuscular junction, primarily mediated by pathogenic immunoglobulin G (IgG) targeting specific proteins like acetylcholine receptor (AChR), muscle-specific tyrosine kinase (MuSK), and low-density lipoprotein receptor-related protein 4 (LRP4). Modulating pathogenic IgG is a promising approach for disease management. Rozanolixizumab, a human IgG4 neonatal Fc receptor (FcRn) inhibitor, enhances the degradation of pathogenic IgG by 78%, marking a significant advancement in treating generalized myasthenia gravis. It offers effective management for patients with AChR or MuSK antibodies and is administered subcutaneously with mild to moderate adverse events. However, the safety and efficacy of rozanolixizumab require further validation through real-world post-marketing studies. If current trial results are confirmed, rozanolixizumab may become a preferred treatment option for myasthenia gravis in the near future. This review examines clinical trials evaluating the pharmacokinetics, efficacy, and safety of rozanolixizumab in patients with generalized myasthenia gravis and discusses ongoing trials and future research directions.