Soliris approved in 日本 for paediatric patients with generalised myasthenia gravis (gMG)

一流的C5抑制剂是日本唯一批准用于该患者群体的靶向治疗

Soliris在III期临床试验中降低了疾病严重程度和症状，持续改善超过26周

Soliris (eculizumab) has been approved in 日本 for expanded use to include the treatment 抗乙酰胆碱受体(AChR)抗体阳性且高剂量静脉免疫球蛋白(IVIG)治疗或血浆置换(PLEX)治疗难以控制症状的小儿全身性重症肌无力(gMG). Soliris 在日本，第一个也是唯一一个被批准用于治疗儿童和青少年gMG的靶向疗法是什么.

The approval by the 日本ese Ministry of Health, Labour and Welfare (MHLW) was based on results from the Phase III trial of Soliris in paediatric patients with refractory gMG.¹

在审判中， Soliris 在先前免疫抑制治疗失败并持续经历显著未解决疾病症状的难治性gMG儿科患者中证明了临床益处. Soliris 第26周时，定量重症肌无力(QMG)总分的主要终点较基线变化有显著改善, a physician-reported scale assessing disease severity and function (-5.8 [95% CI -8.4, -3.13], p=0.0004).¹

gMG是罕见的, 使人衰弱的, 慢性, autoimmune neuromuscular disease that leads to a loss of muscle function and severe weakness.²

Keiko石垣岛, MD, 博士学位, 儿科, 东京女子医科大学, 医学院, 东京, 日本, said: “gMG is challenging to manage in paediatric patients, as current therapies available to this population, 比如免疫抑制剂, may not offer adequate control as the disease progresses. 今天扩大批准 Soliris in 日本 demonstrates the impact of C5 complement inhibition in treating gMG, 为儿科患者提供有针对性的选择，有可能保持肌肉功能并降低疾病严重程度.”

Marc Dunoyer, 首席执行官, Alexion, 他说:“患有gMG的儿科患者可能对标准治疗没有反应，并继续出现影响其活动能力的症状, 语言和呼吸. 澳门第一赌城在线娱乐首创的C5抑制剂 Soliris 是否有可能改善儿科患者及其家属的预后和生活质量, 澳门第一赌城在线娱乐为日本儿科gMG社区提供首个也是唯一一个靶向治疗而感到自豪.”  

的有效性和安全性 Soliris in paediatric patients is consistent with the established profile of Soliris in clinical trials involving adults with refractory gMG.^1,3,4 In the Phase III clinical trial of paediatric patients, the majority of reported adverse events were considered mild or moderate. The most common adverse events were headache and nasopharyngitis.¹

Soliris 于2017年首次在日本被批准用于治疗某些成人gMG，并在美国被批准用于治疗某些成人gMG, 中国和欧盟(EU). Soliris 也是最近 在欧盟获得批准 扩大使用范围，包括治疗6至17岁AChR Ab+的难治性gMG儿童和青少年. 规管意见书 Soliris 多个卫生当局目前正在或计划开展治疗小儿gMG患者的项目.

笔记

gMG
gMG是一种罕见的自身免疫性疾病，其特征是肌肉功能丧失和严重的肌肉无力.²

80%的gMG患者是AChR抗体阳性，这意味着他们产生特异性抗体(抗AChR)，与神经肌肉接点(NMJ)的信号受体结合。, the connection point between nerve cells and the muscles they control.^{2, 5-8} This binding activates the complement system, which is essential to the body’s defence against infection, causing the immune system to attack the NMJ.² This leads to inflammation and a breakdown in communication between the brain and the muscles.²

gMG可以发生在任何年龄, but it most commonly begins for women before the age of 40 and for men after the age of 60.^9-11 Initial symptoms may include slurred speech, 复视, droopy eyelids and lack of balance; these can often lead to more severe symptoms as the disease progresses such as, 吞咽障碍, 令人窒息的, extreme fatigue and respiratory failure.^12,13

Phase III Trial in Paediatric Patients with Refractory gMG
A Phase III 非盲, multicentre 26-week trial evaluated the safety and efficacy of Soliris in eleven patients aged 12 to 17 years old. Participants were required to be older than six years of age, 18岁以下, 是否有难治性重症肌无力且抗achr抗体血清学检测阳性, 经过一年或一年以上的免疫抑制治疗或需要维持血浆置换(PE)或静脉注射免疫球蛋白(IVIg)来控制症状失败, 试验开始时重症肌无力(QMG)定量评分至少12分，筛查时重症肌无力美国临床分类基金会II至IV级.^1,14

评估第26周时QMG总分较基线变化的主要终点，以及评估疾病相关和生活质量措施改善的多个次要终点.

完成随机对照期的患者有资格继续进入开放tags延长期，评估药物的安全性和有效性 Soliris，该项目正在进行中.

Soliris
Soliris (eculizumab) is a first-in-class C5 complement inhibitor. The medication works by inhibiting the C5 protein in the terminal complement cascade, 免疫系统:身体免疫系统的一部分. When activated in an uncontrolled manner, the terminal complement cascade over-responds, leading the body to attack its own healthy cells. Soliris is administered intravenously every two weeks, following an introductory dosing period.

Soliris 在美国被批准了吗, EU, 日本和中国对阵发性夜间血红蛋白尿和非典型溶血性尿毒综合征患者的治疗.

此外, Soliris 在日本和欧盟被批准用于治疗某些成人和儿童gMG患者, and in the US and China for certain adults with gMG.

此外, Soliris 在美国被批准了吗, EU and 日本 for the treatment of certain adults with neuromyelitis optica spectrum disorder.

Soliris is not indicated for the treatment of patients with Shiga-toxin E. coli-related haemolytic uraemic syndrome.

Alexion
Alexion, 澳门在线赌城娱乐罕见病, is the group within 澳门在线赌城娱乐 focused on rare diseases, created following the 2021 acquisition of Alexion Pharmaceuticals, 公司. As a leader in rare diseases for more than 30 years, Alexion专注于通过这一发现为受罕见疾病和毁灭性疾病影响的患者和家庭提供服务, 发展 and commercialisation of life-changing medicines. Alexion的研究重点是补体级联中的新分子和靶点，以及血液学方面的开发工作, 肾脏学, 神经学, 代谢紊乱, 心脏病学和眼科. 总部设在波士顿, 麻萨诸塞州, Alexion has offices around the globe and serves patients in more than 50 countries.

澳门在线赌城娱乐
澳门在线赌城娱乐 (LSE/STO/Nasdaq: AZN) is a global, science-led biopharmaceutical company that focuses on the discovery, 发展, and commercialisation of prescription medicines in 肿瘤学, 罕见疾病, 和澳门第一赌城在线娱乐, 包括心血管, 肾 & 新陈代谢和呼吸 & 免疫学. 总部设在剑桥, UK, 澳门在线赌城娱乐在100多个国家开展业务，其创新药物被全球数百万患者使用. 请访问 澳门在线赌城娱乐.com 并在社交媒体上关注公司 @澳门在线赌城娱乐.

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