Soliris获欧盟批准用于治疗难治性全身性重症肌无力(gMG)的儿童和青少年

This website version was updated on 24 August 2023. This release has been updated to reflect the p value of p=0.0004.

First and only targeted therapy approved for paediatric patients with gMG in the EU
showed sustained improvements in disease severity and function over 26 weeks

Soliris (eculizumab)已在欧盟(EU)批准扩大使用范围，包括治疗6至17岁抗乙酰胆碱受体(AChR)抗体阳性(Ab+)的难治性全身性重症肌无力(gMG)的儿童和青少年。. This is 这是欧盟批准的首个也是唯一一个用于治疗儿科患者的靶向疗法.

The approval by the European Commission follows the positive opinion 人用药品委员会(CHMP)的批准，并基于第三期临床试验的结果 Soliris in paediatric patients with refractory gMG.¹

In the trial, 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 is a rare, debilitating, chronic, autoimmune neuromuscular disease that leads to a loss of muscle function and severe weakness.²

John F. Brandsema, MD, 费城儿童医院和儿科患者III期试验的主要研究者, 他说:“此次批准代表了难治性gMG患儿护理的重大进展, who previously had no targeted treatment options to help manage their condition. Soliris 在26周的III期试验中显示出临床益处和疾病严重程度的持续改善, 为患有这种罕见神经疾病的儿童和青少年提供改善生活质量和重新定义疾病管理的潜力.”

Marc Dunoyer, Chief Executive Officer, Alexion, said: “The impact of gMG on children can be devastating, and families have long been awaiting solutions. This approval of our first-in-class C5 inhibitor Soliris 为欧盟难治性gMG患儿提供变革性药物，帮助解决罕见病社区未满足的医疗需求，这体现了澳门第一赌城在线娱乐的努力. Soliris 为受gMG影响的儿童和青少年提供了改善结果的希望，澳门第一赌城在线娱乐致力于尽快增加这些家庭的获得机会.”

The efficacy and safety of Soliris in paediatric patients aged six years and older 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 该药物于2017年首次在欧盟获批用于治疗某些成人gMG患者，并在美国获批用于治疗某些成人gMG患者, China and Japan. Regulatory submissions for 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.

Notes

gMG
gMG is a rare autoimmune disorder characterised by loss of muscle function and severe muscle weakness.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 can occur at any age, 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, double vision, droopy eyelids and lack of balance; these can often lead to more severe symptoms as the disease progresses such as, impaired swallowing, choking, extreme fatigue and respiratory failure.^12,13

Phase III Trial in Paediatric Patients with Refractory gMG
A Phase III open-label, 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, younger than 18, 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标签延长期，评估药物的安全性和有效性 Soliris, which is ongoing.

Soliris
Soliris (eculizumab) is a first-in-class C5 complement inhibitor. The medication works by inhibiting the C5 protein in the terminal complement cascade, a part of the body’s immune system. 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 is approved in the US, 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.

Additionally, Soliris is approved in the EU for the treatment of certain paediatric patients with gMG, and in the US, Japan and China for certain adults with gMG.

Further, Soliris is approved in the US, EU and Japan 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, AstraZeneca Rare Disease, is the group within AstraZeneca focused on rare diseases, created following the 2021 acquisition of Alexion Pharmaceuticals, Inc. 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, development 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, nephrology, neurology, metabolic disorders, cardiology and ophthalmology. Headquartered in Boston, Massachusetts, Alexion has offices around the globe and serves patients in more than 50 countries.

AstraZeneca
AstraZeneca (LSE/STO/Nasdaq: AZN) is a global, science-led biopharmaceutical company that focuses on the discovery, development, and commercialisation of prescription medicines in Oncology, Rare Diseases, and BioPharmaceuticals, including Cardiovascular, Renal & Metabolism, and Respiratory & Immunology. Based in Cambridge, 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. Please visit 3mr.net and follow the Company on Twitter @AstraZeneca.

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