CSL  announced that the European Commission (EC) has approved Andembry

CSL宣布，欧洲委员会（EC）已批准安第姆布里

(garadacimab), the first and only once-monthly treatment targeting factor XIIa to prevent attacks of hereditary angioedema (HAE) in adult and adolescent patients aged 12 years and older

（garadacimab），第一个也是唯一一个每月一次的针对因子XIIa的治疗方法，用于预防12岁及以上成人和青少年患者的遗传性血管性水肿（HAE）发作

Andembry inhibits plasma protein factor XIIa, which initiates the cascade of events leading to angioedema at various sites of the body.  reinforces CSL's decades-long commitment to delivering innovative treatment modalities to the HAE community and comes with a convenient patient-centric pre-filled pen (auto-injector) enabling subcutaneous self-injection..

胚胎抑制血浆蛋白因子XIIa，这引发了一系列事件，导致身体各个部位的血管性水肿。强化了CSL数十年来为HAE社区提供创新治疗方式的承诺，并配备了一种方便的以患者为中心的预填充笔（自动注射器），可实现皮下自我注射。。

Andembry  is a significant advancement in the management of hereditary angioedema, offering people living with this life-threatening condition long-term control over their disease with a patient-centric, convenient administration method,' said Bill Mezzanotte, MD, Executive Vice President, Head of R&D, CSL.

CSL研发主管、执行副总裁、医学博士比尔·梅扎诺特（BillMezzanotte）说，胚胎移植是遗传性血管性水肿治疗方面的一项重大进步，为患有这种危及生命的疾病的人提供了以患者为中心的方便给药方法，可以长期控制疾病。

'Andembry  CSL's first approved recombinant monoclonal antibody discovered and developed entirely by CSL, underscores our more than 40-year legacy in HAE research and treatment optimization and our decades-long journey to bring this innovation to patients. Thank you to all the colleagues, physicians and patients who contributed to this exciting milestone for HAE patients and CSL.'.

Andembry CSL的第一个完全由CSL发现和开发的经批准的重组单克隆抗体，突显了我们在HAE研究和治疗优化方面40多年的遗产，以及我们为患者带来这一创新的数十年漫长历程。感谢所有为HAE患者和CSL这一激动人心的里程碑做出贡献的同事、医生和患者。”。

HAE

HAE公司

is a rare, chronic, debilitating, and potentially life-threatening genetic disorder characterized by recurrent and unpredictable attacks of angioedema. Attacks of HAE are often painful and can spread to multiple sites of the body, including the abdomen, larynx, face, and extremities. HAE occurs in about 1 in 50,000 people of any ethnic group..

。HAE的发作通常是痛苦的，并且可以扩散到身体的多个部位，包括腹部，喉部，面部和四肢。HAE发生在任何种族的50000人中约有1人。。

'The physical and emotional toll of HAE is substantial, and the true prevalence of the disease could be higher than currently recorded due to patient misdiagnoses,' said Prof. Dr. med Markus Magerl, MD, Head of Clinical Trials, Charité University Hospital Berlin, Institute of Allergology. 'The unpredictable nature of HAE is one of the most daunting aspects for many patients, who never know when an attack might occur.

柏林Charité大学医院变态反应研究所临床试验负责人医学博士MedMarkusMagerl教授说，HAE对身体和情绪的影响很大，由于患者误诊，该疾病的真实患病率可能高于目前的记录HAE的不可预测性是许多患者最令人畏惧的方面之一，他们永远不知道何时可能发生发作。

Current HAE preventive therapies work at various downstream steps in the cascade, but none prevent the cascade at its very start like Andembry.'.

目前的HAE预防疗法在级联反应的各个下游步骤中起作用，但没有一种像胚胎一样在级联反应的一开始就阻止了级联反应。”。

The approval of Andembry is based on the efficacy and safety data from the pivotal international Phase III VANGUARD trial and its open-label extension study. The detailed results of the VANGUARD trial were published in

Andembry的批准是基于关键的国际III期VANGUARD试验及其开放标签扩展研究的有效性和安全性数据。VANGUARD试验的详细结果发表在

The Lancet

刺胳针

in April 2023 and the primary results of the ongoing open-label extension study were published in

2023年4月，正在进行的开放标签扩展研究的主要结果发表在

Allergy

过敏

(October 2024)(previously cited)

（2024年10月）（先前引用）

Citation;.  The Lancet;

引用；。柳叶刀；

Volume 401, Issue 10382

第401卷，第10382期

p1079-1090April 01, 2023. 'Efficacy and safety of garadacimab, a factor XIIa inhibitor for hereditary angioedema prevention (VANGUARD): a global, multicentre, randomised, double-blind, placebo-controlled, phase III trial .

p1079-1090 2023年4月1日。”用于预防遗传性血管性水肿的XIIa因子抑制剂加拉西单抗（VANGUARD）的疗效和安全性：一项全球性，多中心，随机，双盲，安慰剂对照的III期临床试验。

Prof Timothy J Craig, DO

∙

∙

Avner Reshef, MD

医学博士Avner Reshef

∙

∙

H Henry Li, MD

H Henry Li，医学博士

∙

∙

Joshua S Jacobs, MD

医学博士约书亚·S·雅各布斯

∙

∙

Prof Jonathan A Bernstein, MD

医学博士乔纳森·伯恩斯坦教授

∙

∙

Prof Henriette Farkas, PhD

Henriette Farkas教授，博士

∙ et al.

∙等人。

Condition:

条件：

Hereditary Angio-Oedema

遗传性血管水肿

Type:

类型：

drug

药物