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FDA批准Hemlibra用于减少或防止某些A型血友病的出血频率

2017-11-19 MedSci MedSci原创

HemlibraA型血友病患者
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美国FDA批准了Hemlibra (emicizumab - kxwh)用于防止或减少成人和儿童A型血友病患者出血的频率，这些患者体内有一种叫做因子VIII(FVIII)抑制剂的抗体。"降低出血频率或防止出血事件是血友病患者疾病管理的重要组成部分。这一批准提供了一种新的预防治疗方法，它已经被证明可以显着减少因子VIII抑制剂的A型血友病患者出血的次数。" FDA药物评估和研究中心血液和肿瘤产品办公


美国FDA批准了Hemlibra (emicizumab - kxwh)用于防止或减少成人和儿童A型血友病患者出血的频率，这些患者体内有一种叫做因子VIII(FVIII)抑制剂的抗体。

"降低出血频率或防止出血事件是血友病患者疾病管理的重要组成部分。这一批准提供了一种新的预防治疗方法，它已经被证明可以显着减少因子VIII抑制剂的A型血友病患者出血的次数。" FDA药物评估和研究中心血液和肿瘤产品办公室主任Richard Pazdur博士说道。"另外，接受Hemlibra治疗的患者的生理功能也有所改善。"

A型血友病是一种遗传性的凝血障碍，主要影响男性。据美国国立卫生研究院(National Institutes of Health)称，在美国，每5000个男性中就有一个患有血友病，其中大约80%的人为A型血友病。A型血友病患者缺少一种能产生凝血因子VIII的基因。患者可能会经历反复的严重出血，主要发生在关节内，结果可能会使关节严重受损。一些患者会产生FVIII抑制剂或抗体的免疫反应。这种抗体干扰了现有血友病治疗的有效性。

Hemlibra是一线疗法，通过桥接其他因子来恢复这些患者的凝血功能。Hemlibra是一种预防性的治疗，每周通过皮下注射。

Hemlibra的安全性和有效性基于两个临床试验的数据。第一个试验包括109名年龄在12岁及12岁以上的男性含FVIII抑制剂A型血友病患者。该试验的随机部分将Hemlibra治疗组和53名未接受预防性治疗的患者进行了比较。他们在入组之前曾接受过旁路治疗。服用Hemlibra的患者每年经历约2.9次出血事件，而未接受预防性治疗的患者每年约有23.3次出血事件。这意味着治疗出血率降低了87%。该试验还包括患者口述的身体健康指标。与未接受预防性治疗的患者相比，接受Hemlibra治疗的患者与血友病相关的症状（疼痛性肿胀和关节疼痛）和身体机能（运动疼痛和走路困难）得到了改善。

第二项试验是对23名12岁以下的FVIII抑制剂A型血友病男性患者进行单臂试验。在试验期间，服用Hemlibra的患者中87％没有经历需要治疗的出血事件。

Hemlibra的常见副作用包括注射部位反应，头痛和关节痛（关节痛）。

Hemlibra标签有一个警告框，提醒医务人员和患者，在抢救治疗(活化的凝血酶原复合物浓缩物)的患者同时服用Hemlibra治疗出血时，24小时或更长时间后观察到严重的血凝块（血栓性微血管病和血栓栓塞）。

Hemlibra曾获得FDA颁发的优先审评资格和突破性疗法认定。Hemlibra还获得孤儿药物的名称，这为激励协助和鼓励罕见疾病药物的开发提供了动力。

该药属于基因泰克公司所有。


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    2018-04-26 仁医06

    #FDA批准#

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    2018-10-05 仁者大医

    #A型血#

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    2017-11-21 changfy

    #A型血友病#

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    2017-11-21 wfang546

    #Hemlibra#

    0

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    2017-11-19 三生有幸9135

    学习一下谢谢分享

    0