△至，美国的湿性AMD（年龄相关性黄斑变性）患者很可能超过150万人，而AMD是工业化国家致盲的主要原因
△此次申报数据主要是来自于Brolucizumab的HAWK和HARRIER III期研究的数据
△诺华使用了优先审查通路获得了Brolucizumab在美国的快速评审。若获得FDA批准，预计该药将于底上市
昨日，诺华公司在巴塞尔宣布，美国食品药品监督管理局（FDA）已接受诺华公司用于治疗湿性年龄相关性黄斑变性（AMD）的brolucizumab（RTH258）的生物药许可证申请（BLA）。该种AMD亦称为新生血管性AMD（nAMD）。为加速brolucizumab上市，诺华公司使用了优先审查通路以期获得快速审评。若得到FDA的批准，诺华预计将于底上市brolucizumab。
据估计，至，美国将有150万至175万人罹患湿性AMD，而湿性AMD是全球致盲的主要原因，也是一个日益严峻的公共卫生问题1。随着病情发展，患者可能会失去中心视力，导致无法进行日常活动。若不治疗，视力会迅速恶化，可致失明2。
诺华制药（美国）总裁Fabrice Chouraqui认为：“我们致力于重塑湿性AMD患者及其照护者的治疗历程。实现这一里程碑是其中重要一步。湿性AMD患者需要面对视力下降导致的巨大身心困扰，我们希望能够为患者提供新的治疗选择。”
“湿性AMD剥夺了人们的宝贵视力，对数百万人的生活造成了巨大负担。这些人不仅面临视力丧失，而且还要承受对频繁眼内注射药物的痛苦，”视力支持基金会执行主任Dawn Prall George认为，“我们对这种前景光明的新治疗方案十分期待，希望它们能帮助患者更好地控制这种严重损害性疾病。”
Brolucizumab（RTH258）是一种人源化单链抗体片段（scFv），是目前达到开发阶段的临床上最先进的人源化单链抗体片段。由于单链抗体片段体积小、组织渗透性强、从全身循环里快速清除及其药物释放特性，brolucizumab在药物开发中受到高度关注7-9。
该专利创新结构使得药物分子更小（26kDa），对所有血管内皮生长因子VEGF-A异构体有强大抑制作用及高度亲和力7-10。临床前研究中，brolucizumab通过阻止配体-受体相互作用，抑制VEGF受体的激活7,9,10。VEGF信号通路增强的信号转导与病理性眼部血管生成及视网膜水肿有关11。抑制VEGF通路已被证明能抑制脉络膜视网膜血管疾病患者的新生血管病灶的生长，缓解视网膜水肿，改善视力12。
nAMD是北美、欧洲、澳大利亚和亚洲65岁以上人群严重失明和法定盲的主要原因，全球患者约2000万至2500万人2,13。nAMD是由异常血管形成于黄斑（视网膜中负责敏锐的中央视力的区域）下方所导致的。这些血管非常脆弱，有漏液现象，会破坏正常视网膜结构，最终导致黄斑的损害1,14,15。
nAMD的早期症状包括视物扭曲变形以及清晰视物的困难16。及时诊断及干预至关重要。随着疾病发展，细胞损伤增加，视力进一步下降。如此下去，可致中央视力的完全丧失，使患者无法阅读、驾驶或识别熟悉面孔14。若不治疗，视力会迅速恶化2。
诺华眼科正在重新构建视觉损伤及失明的治疗与预防。我们致力于突破医学和技术的界限，以开发颠覆性基因疗法、下一代药物以及覆盖眼前节到眼后节各部位疾病的变革性性治疗技术为使命。
*Brolucizumab（RTH258）尚未在中国递交新药审批
Disclaimer
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