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20 年来首个创新抗流感药物!罗氏 baloxavir marboxil 获 FDA 优先审评资格


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。



(向上滑动,查看完整参考资料)

参考资料:

1.Portsmouth S et al. Cap-Dependent Endonuclease Inhibitor S-033188 for the Treatment of Influenza: Results from a Phase 3, Randomized, Double-Blind, Placebo- and Active-Controlled Study in Otherwise Healthy Adolescents and Adults with Seasonal Influenza. ID Week 2017.

2.World Health Organization. Up to 650 000 people die of respiratory diseases linked to seasonal flu each year [Internet; cited 2018 May 29]. Available from: http://www.who.int/mediacentre/news/releases/2017/seasonal-flu/en/.

3.World Health Organization. Influenza (Seasonal) [Internet; cited 2018 May 29]. Available from: http://www.who.int/mediacentre/factsheets/fs211/en.

4.Baxter D. Evaluating the case for trivalent or quadrivalent influenza vaccines. Hum Vaccin Immunother. 2016; 12(10):2712-2717.

5.Centers for Disease Control and Prevention. Estimated Influenza Illnesses, Medical Visits, Hospitalizations, and Deaths Averted by Vaccination in the United State. [Internet; cited 2018 June 06]. Available from: https://www.cdc.gov/flu/about/disease/2015-16.htm

6.Nair H, et al. Global burden of respiratory infections due to seasonal influenza in young children: a systematic review and meta-analysis. Lancet. 2011 Dec 3;378(9807):1917-30.

7.T. Noshi et al. S-033447/S-033188, a Novel Small Molecule Inhibitor of Cap-dependent Endonuclease of Influenza A and B Virus: In Vitro Antiviral Activity against Laboratory Strains of Influenza A and B Virus in Madin-Darby Canine Kidney Cells. Poster presentation at OPTIONS IX, August 2016

8.K.Taniguchi et al. Inhibitory Effect of S-033188, a novel inhibitor of influenza virus cap-dependent endonuclease, against avian influenza A/H7N9 virus in vitro and in vivo. Poster presentation at ESWI, September 2017.

9.K.Taniguchi et al. Inhibitory Effect of S-033188/S-033447, a novel inhibitor of influenza virus cap-dependent endonuclease, against highly pathogenic avian influenza virus A/H5N1. Poster presentation at ECCMID, April 2017.


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