The effectiveness of C5 inhibition in human trials, which will be discussed later in this review, offers further compelling evidence that complement is a critical mediator of MG. The relative contribution of non-complement-mediated mechanisms to human MG is poorly defined.

2024年11月1日 · 目前MG领域获批的C5补体抑制剂有多肽Zilucoplan、依库珠单抗和瑞利珠单抗。 其中Zilucoplan是全球首个皮下注射补体C5抑制剂，也是截止目前唯一可自行给药的gMG靶向疗法。

Preclinical studies have confirmed the efficacy of complement inhibition in ameliorating MG symptoms. Eculizumab, an antibody directed towards C5, has recently been approved for the treatment of AChR antibody-positive gMG. Other complement inhibitors, targeting C5 as well, are currently under phase III study.

2023年10月17日 · FDA approval of ZILBRYSQ® (zilucoplan) has been granted for the treatment of generalized myasthenia gravis (gMG) in adult patients who are anti-acetylcholine receptor (AChR) antibody-positive1 Zilucoplan is the first once-daily subcutaneous, targeted C5 complement inhibitor for gMG.

2019年6月14日 · Dose-dependent serum C5 suppression was observed in non-human primates, with a lowering of serum C5 of up to 97.5% and the concomitant inhibition of serum complement activity. C5 silencing was efficacious in ameliorating disease symptoms in two standard rat models of MG, demonstrating the key role of circulating C5 in pathology at the NMJ.

2024年10月1日 · Myasthenia gravis (MG) is the most prevalent autoimmune disorder of the neuromuscular junction characterized by antibody-mediated destruction of the postsynaptic membrane. Clinical hallmarks of MG include fatigable weakness of ocular, bulbar, limb and respiratory muscles.

2023年10月18日 · With the approval, zilucoplan becomes the first once-daily subcutaneous C5 complement inhibitor available for adults with generalized myasthenia gravis. According to an announcement, the FDA has approved UCB Pharma’s investigational agent zilucoplan, a complement C5 inhibitor, to treat patients with myasthenia gravis (MG).

Emerging treatments such as complement factor C5 inhibition (C5IT) and neonatal Fc receptor (FcRn) antagonism hold promise for these patients. However, the current landscape is hindered by a paucity of comparative data that is crucial for treatment decisions.

2023年8月31日 · Gefurulimab 是 Alexion Pharmaceuticals （阿斯利康子公司）开发的第三代 C5 抑制剂，是一种迷你双抗 (25kD) ，只包含了靶向 C5 的抗体重链可变区 (VH) 和与白蛋白 (Albumin) 特异性结合的抗体片段。较小的分子量可以带来更好的渗透性，并且与白蛋白的结合能够延长其半衰期。

2020年6月3日 · Complement activation as a driver of pathology in myasthenia gravis (MG) has been appreciated for decades. The terminal complement component [membrane attack complex (MAC)] is found at the neuromuscular junctions of patients with MG. Animals with experimental autoimmune MG are dependent predominantl …