Crizanlizumab: First Approval
Crizanlizumab: First Approval
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DOI：
10.1007/s40265-019-01254-2
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发表时间：
2020-01-01
期刊：
影响因子：
11.5
通讯作者：
Blair, Hannah A.
中科院分区：
文献类型：
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作者：
Blair, Hannah A.
Crizanlizumab (Adakveo)；crizanlizumab-tmca是诺华制药公司开发的一种静脉给药单克隆抗体，用于预防镰状细胞病患者的血管闭塞危像（VOCs）。Crizanlizumab结合p -选择素，从而阻断其与p -选择素糖蛋白配体-1的相互作用。2019年11月，crizanlizumab在美国获得首个全球批准，用于降低成人和16岁镰状细胞病儿童患者的VOCs频率。该药还在欧盟接受监管审查，用于预防镰状细胞病患者的挥发性有机化合物。在澳大利亚、西班牙、德国和匈牙利，也正在评估crizanlizumab（联合ruxolitinib）在骨髓纤维化中的应用。本文总结了crizanlizumab在镰状细胞病患者中首次批准减少VOCs的发展里程碑。
Crizanlizumab (Adakveo (R); crizanlizumab-tmca) is an intravenously administered monoclonal antibody developed by Novartis Pharmaceuticals for the prevention of vaso-occlusive crises (VOCs) in patients with sickle cell disease. Crizanlizumab binds to P-selectin, thereby blocking its interaction with P-selectin glycoprotein ligand-1. In November 2019, crizanlizumab received its first global approval in the USA, where it is indicated to reduce the frequency of VOCs in adults and paediatric patients aged >= 16 years with sickle cell disease. The drug is also under regulatory review in the EU for the prevention of VOCs in patients with sickle cell disease. The use of crizanlizumab (in combination with ruxolitinib) in myelofibrosis is also being evaluated in Australia, Spain, Germany and Hungary. This article summarizes the milestones in the development of crizanlizumab leading to this first approval for the reduction of VOCs in patients with sickle cell disease.