Matic Alexandria, Bril Vera
The Ellen & Martin Prosserman Centre for Neuromuscular Diseases, University Health Network, University of Toronto, Toronto, ON, Canada.
Immunotherapy. 2025 Apr;17(5):309-316. doi: 10.1080/1750743X.2025.2491295. Epub 2025 Apr 25.
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.
重症肌无力是一种罕见的慢性自身免疫性疾病，会影响突触后神经肌肉接头，主要由针对特定蛋白质（如乙酰胆碱受体（AChR）、肌肉特异性酪氨酸激酶（MuSK）和低密度脂蛋白受体相关蛋白4（LRP4））的致病性免疫球蛋白G（IgG）介导。调节致病性IgG是一种很有前景的疾病管理方法。罗扎诺利昔单抗是一种人IgG4新生儿Fc受体（FcRn）抑制剂，可将致病性IgG的降解提高78%，这标志着在治疗全身型重症肌无力方面取得了重大进展。它为患有AChR或MuSK抗体的患者提供了有效的治疗管理，通过皮下给药，不良事件为轻度至中度。然而，罗扎诺利昔单抗的安全性和有效性需要通过上市后真实世界研究进一步验证。如果目前的试验结果得到证实，罗扎诺利昔单抗可能在不久的将来成为重症肌无力的首选治疗方案。这篇综述研究了评估罗扎诺利昔单抗在全身型重症肌无力患者中的药代动力学、疗效和安全性的临床试验，并讨论了正在进行的试验和未来的研究方向。
