Vertex新的三联组合治疗囊性纤维化,3期临床大获成功

2019-06-02 不详 MedSci原创

自2012年推出Kalydeco以来,总部位于马萨诸塞州波士顿的Vertex公司在囊性纤维化(CF)治疗方面一直处于领先地位。全球CF患者约75000人,其目前的Kalydeco,Orkambi和Symkevi组合只能治疗这一总数的一半,Vertex公司的最终目标是治疗所有的CF患者。

自2012年推出Kalydeco以来,总部位于马萨诸塞州波士顿的Vertex公司在囊性纤维化(CF)治疗方面一直处于领先地位。全球CF患者约75000人,其目前的Kalydeco,Orkambi和Symkevi组合只能治疗这一总数的一半,Vertex公司的最终目标是治疗所有的CF患者。

Vertex近日宣布新的三联组合:VX-445、Symkevi(tezacaftor)和Kalydeco(ivacaftor)结合,用于治疗具有一个F508del突变和一个最小功能突变的患者,试验数据显示这些患者有显着改善。

最新数据来自一项为期24周的3期研究,与接受三重安慰剂的患者相比,用力呼气量在一秒内达到百分比(ppFEV1)的平均绝对改善率相对于基线为14.3%,并且肺部恶化的年化率降低63%。

Vertex三重组合指导委员会联合主席Steven M. Rowe 表示:"在这些3期研究中看到的肺功能实质性改善是前所未有的,这代表了向绝大多数CF患者提供新的联合治疗药物的决定性时刻。"

Vertex计划在2019年第三季度向FDA提交申请,预计将在2020年上半年获得批准。

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    2020-03-27 wolongzxh
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    2019-06-04 liuli5080

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