Soliris approved in the EU for children and adolescents with refractory generalised myasthenia gravis (gMG)

本网站于2023年8月24日更新. 此版本已更新，以反映p=0的p值.0004.

欧盟首个也是唯一一个批准用于小儿gMG患者的靶向治疗
在26周内疾病严重程度和功能持续改善

Soliris (eculizumab) has been approved in the European Union (EU) for expanded use to include the treatment of refractory generalised myasthenia gravis (gMG) in children and adolescents aged six to 17 years who are anti-acetylcholine receptor (AChR) antibody-positive (Ab+). 这是 the first and only targeted therapy approved for the treatment of paediatric patients with the disease in the EU.

欧盟委员会的批准是在 积极的观点 of the Committee for Medicinal Products for Human Use (CHMP) and is based on results from the Phase III trial of Soliris 治疗难治性gMG的儿科患者.¹

在审判中， Soliris demonstrated clinical benefit 治疗难治性gMG的儿科患者 who previously failed immunosuppressive treatment and continued to experience significant unresolved disease symptoms. Soliris showed significant improvement in the primary endpoint of change from baseline in Quantitative 重症肌无力 (QMG) total score at week 26, 医生报告的评估疾病严重程度和功能的量表(-5).8 [95% CI -8.4, -3.13], p=0.0004).¹

gMG是罕见的, 使人衰弱的, 慢性, 一种自身免疫性神经肌肉疾病，导致肌肉功能丧失和严重无力.²

约翰F. Brandsema, MD, Children’s Hospital of Philadelphia and primary investigator in the Phase III trial of paediatric patients, said: “This approval represents a significant advance in care for paediatric patients with refractory gMG, 谁以前没有针对性的治疗选择来帮助控制他们的病情. Soliris showed clinical benefit and sustained improvements in disease severity through 26 weeks in a Phase III trial, offering potential to improve quality of life and redefine disease management for children and adolescents living with this rare neurological disease.”

Marc Dunoyer, 首席执行官, Alexion, 他说:“gMG对儿童的影响可能是毁灭性的, 家属们一直在等待解决方案. 澳门第一赌城在线娱乐的首个同类C5抑制剂获批 Soliris for paediatric patients with refractory gMG in the EU exemplifies our efforts to deliver transformative medicines that help address unmet medical needs for rare disease communities. Soliris offers hope for improved outcomes for children and adolescents impacted by gMG and we are committed to increasing access for these families as quickly as possible.”

的有效性和安全性 Soliris 在6岁及以上的儿科患者中，与已建立的概况一致 Soliris 在成人难治性gMG的临床试验中.^1,3,4 在儿科患者的III期临床试验中, 大多数报告的不良事件被认为是轻度或中度的. 最常见的不良反应是头痛和鼻咽炎.¹

Soliris was first approved the EU in 2017 for the treatment of certain adults with gMG and is also approved for certain adults with gMG in the US, 中国和日本. 规管意见书 Soliris for the treatment of paediatric patients with gMG are currently ongoing or planned with multiple health authorities.

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

Eighty percent of people with gMG are AChR antibody positive meaning they produce specific antibodies (anti-AChR) that bind to signal receptors at the neuromuscular junction (NMJ), 神经细胞和它们控制的肌肉之间的连接点.^2,5-8 这种结合激活了补体系统, 哪一种对人体抵御感染至关重要, 导致免疫系统攻击NMJ.² 这会导致炎症和大脑与肌肉之间的沟通中断.²

gMG可以发生在任何年龄, 但它最常见于40岁之前的女性和60岁之后的男性.^9-11 最初的症状可能包括口齿不清, 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, 极度疲劳和呼吸衰竭.^12,13

难治性gMG患儿的III期临床试验
一项开放tags、多中心、为期26周的III期临床试验评估了该药的安全性和有效性 Soliris 11例12至17岁的患者. 参与者的年龄必须大于6岁, 18岁以下, have a confirmed refractory myasthenia gravis diagnosis with a positive serologic test for anti-AChR antibodies, prior failure after a year or more on immunosuppressive therapy or required maintenance plasma exchange (PE) or intravenous immunoglobulin (IVIg) to control symptoms, Quantitative 重症肌无力 (QMG) score of at least 12 at trial entry and 重症肌无力 Foundation of America Clinical Classification Class II to IV at screening.^1,14

The primary endpoint of change from baseline in QMG total score at Week 26 was assessed along with multiple secondary endpoints evaluating improvement in disease-related and quality-of-life measures.

Patients who completed the 随机 control period were eligible to continue into an 非盲 extension period evaluating the safety and efficacy of Soliris，该项目正在进行中.

Soliris
Soliris (eculizumab)是一流的C5补体抑制剂. 该药物通过抑制终末补体级联中的C5蛋白起作用, 免疫系统:身体免疫系统的一部分. 当以不受控制的方式激活时, 终末补体级联反应过度, 导致身体攻击自己的健康细胞. Soliris 是否在初始给药期后每两周静脉注射一次.

Soliris 在美国被批准了吗, EU, Japan and China for the treatment of patients with paroxysmal nocturnal haemoglobinuria and atypical haemolytic uraemic syndrome.

此外, Soliris 是否在欧盟被批准用于治疗某些患有gMG的儿科患者, 在美国, 日本和中国的某些成年人有gMG.

此外, Soliris 在美国被批准了吗, 欧盟和日本对某些成人视神经脊髓炎谱系障碍的治疗.

Soliris 不适合用于治疗志贺毒素E. 与大肠杆菌相关的溶血性尿毒综合征.

Alexion
Alexion, 澳门在线赌城娱乐罕见病, 澳门在线赌城娱乐内部的团队专注于罕见疾病吗, 在2021年收购Alexion Pharmaceuticals后创建, 公司. 作为罕见病领域三十多年的领导者, Alexion is focused on serving patients and families affected by rare diseases and devastating conditions through the discovery, 改变生活的药物的开发和商业化. Alexion focuses its research efforts on novel molecules and targets in the complement cascade and its 发展 efforts on haematology, 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在全球设有办事处，为50多个国家的患者提供服务.

澳门在线赌城娱乐
澳门在线赌城娱乐(LSE/STO/Nasdaq: AZN)是一家全球性制药公司, 以科学为主导的澳门第一赌城在线娱乐公司，专注于发现, 发展, 以及肿瘤学处方药的商业化, 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 operates in over 100 countries and its innovative medicines are used by millions of patients worldwide. 请访问 澳门在线赌城娱乐.com 并在推特上关注公司 @澳门在线赌城娱乐.

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