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FDA批准首个纳米抗体,治疗获得性血栓性血小板减少性紫癜

医药魔方 医药魔方 来源:医药魔方
2019-02-07
Cablivi注射液 纳米抗体
原文

2月6日,FDA批准赛诺菲子公司Ablynx开发的Cablivi (caplacizumab-yhdp) 注射液上市,联合血浆置换和免疫抑制疗法用于成人获得性血栓性血小板减少性紫癜(aTTP)。2018年9月3日,欧盟已经针对该适应症批准了Cablivi的上市申请。Cablivi 是FDA针对该适应症批准的首个新药,也是全球首个上市的纳米抗体新药。


aTTP是一种罕见但会威胁生命的凝血障碍疾病,表现为患者全身的小血管出现广泛性凝血,阻碍体内主要器官的正常氧气和血液供应,引发卒中和心脏病,进而导致脑损伤或死亡。癌症、艾滋病、怀孕、狼疮、感染、术后、骨髓移植或化疗患者均有可能进展出现aTTP。


caplacizumab是Ablynx公司开发的一种抗血管性血友病因子(vWF)纳米抗体,可抑制超大vWF多聚体和血小板间的相互作用,避免血小板和凝聚和凝血块的出现。纳米抗体是只有重链可变区的单域抗体,含有最小功能的抗原结合片段,与普通抗体相比具有分子量小、结构简单易改造、稳定性好等优点。


caplacizumab的疗效在一项涉及145例aTTP患者的随机研究中得到证实。患者接受现有的标准治疗、血浆置换和免疫抑制药物,在此基础上随机分组并分别给予caplacizumab或安慰剂。


结果显示,接受caplacizumab治疗的患者相比安慰剂组可以快速改善血小板计数,并且aTTP相关死亡患者的数量、治疗期间aTTP复发的次数、至少1次治疗相关重大血栓事件的患者数量均低于安慰剂组,在整个研究期间(给药期+停药后28天随访期)发生aTTP复发的患者比例显著低于安慰机组(13% vs 38%)。caplacizumab常见的不良反应包括鼻子或牙龈出血、头痛。


赛诺菲在2018年1月连续进行了两笔大收购,先是116亿美元收购从Biogen拆分出来的血友病和血液疾病专科药物公司Bioverativ,紧跟着以39亿欧元收购Ablynx,将caplacizumab收入囊中。


机器翻译

On February 6, the FDA approved Cablivi (caplacizumab-yhdp) Injection, developed by Sanofi subsidiary Ablynx, in combination with plasmapheresis and immunosuppressive therapy for acquired thrombotic thrombocytopenic purpura (aTTP) in adults.On September 3, 2018, the European Union has approved the marketing application of Cablivi for this indication.Cablivi is the first new drug approved by the FDA for this indication and the first new nanobody drug to be marketed worldwide.

aTTP is a rare but life-threatening blood clotting disorder characterized by extensive clotting of small blood vessels throughout the patient's body that impedes normal oxygen and blood supply to major organs in the body, causing stroke and heart disease, leading to brain damage or death.Cancer.AIDS.Pregnancy.Lupus.Infection.After surgery.Patients with bone marrow transplantation or chemotherapy are likely to progress to aTTP.

Caplacizumab is an anti-von Willebrand factor (vWF) nanobody developed by Ablynx that inhibits the interaction between large vWF multimers and platelets, avoiding platelets and the appearance of clots and clots.Nanobodies are single-domain antibodies with only the heavy chain variable region, containing the smallest functional antigen-binding fragment, and have a lower molecular weight than common antibodies.The structure is simple and easy to reform. The stability is good and so on.

The efficacy of caplacizumab was confirmed in a randomized study involving 145 patients with aTTP. Patients received existing standard treatments. Plasma exchange and immunosuppressive drugs were randomized and given caplacizumab or placebo, respectively.

The results showed that patients receiving caplacizumab were able to rapidly improve platelet counts compared with placebo, and the number of aTTP-related deaths. The number of aTTP relapses during treatment. The number of patients with at least one treatment-related major thrombotic event was lower than placebo. In the group, the proportion of patients with aTTP recurrence during the entire study period (dose period + 28 days follow-up period) was significantly lower than that of the placebo group (13% vs 38%). Common side effects of caplacizumab include nose or gum bleeding.

Sanofi made two major acquisitions in January 2018, first to acquire the hemophilia and blood disease specialist drug company Bioverativ, which was split from Biogen, and then to acquire Ablynx for 3.9 billion euros, which will be caplacizumab. In the pocket of income.

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