瑞士罗氏集团昨日宣布，美国食品药品监督管理局（FDA）接受 baloxavir marboxil 的新药上市申请，并授予其优先审评资格。Baloxavir marboxil 是一种单剂口服药物，用于治疗 12 岁及以上的急性、无并发症流感患者。FDA 有望在 2018 年 12 月 24 日前做出审批决定。
优先审评资格的授予，意味着 FDA 认为该药有望为某一疾病的治疗、预防或诊断带来重大改善。如果获批，baloxavir marboxil 将成为首个口服单剂抗病毒药物，同时也是近 20 年来首个具有创新作用机制的抗流感药物 1。
流行性感冒（influenza），简称流感，是对公众健康的严重威胁。全球每年因流感导致大约 300－500 万患者病情严重，数百万患者住院治疗，65 万患者死亡 2,3,4,5,6。
罗氏首席医学官兼全球产品开发负责人 Sandra Horning 博士表示：
近年来流感季节的严峻形势，表明我们亟需现有疗法以外的创新治疗选择。如果获批，baloxavir marboxil 将成为近 20 年来首个具有创新作用机制的抗流感药物。临床试验表明，baloxavir marboxil 仅需服用一次，就能减少流感症状的持续时间，并在一天内显著减少病毒脱落。我们期待与 FDA 继续合作，推进审评进程。
Baloxavir marboxil 是同类首个单剂试验性口服药物，其创新的作用机制能够有效针对流感病毒，包括对奥司他韦具有耐药性的菌株、禽流感菌株（H7N9, H5N1）7,8,9。与其它现有抗病毒药物不同的是，baloxavir marboxil 通过抑制流感病毒中的 cap-依赖型核酸内切酶蛋白，起到抑制病毒复制的作用。
此次新药上药申请，是基于 III 期试验 CAPSTONE-1 的研究结果。研究中，流感患者分为两组，一组服用单剂 baloxavir marboxil，对照组则服用安慰剂或奥司他韦 75mg，每日两次，连续五天。此外，一项 II 期安慰剂控制研究的结果也用作此次新药上市申请的支持数据。
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参考资料：
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