金吾财讯 | 美国 FDA 正式批准福泰只要(VRTX) 旗下基因编辑疗法 Casgevy (exagamglogene autotemcel)，用于治疗两岁及以上确诊镰状细胞病、β 地中海贫血的儿童患者，拓宽该创新疗法的临床适用年龄区间，为低龄罕见血液病患儿提供全新治疗方案。
本次审批属于专员国家优先代金券试点计划下第八款获批药品，此前该疗法仅获批用于 12 岁及以上适龄患者，本次年龄下限下调后，覆盖更多幼年发病、长期依赖输血的儿童群体，完善全年龄段罕见血液病治疗方案布局。Casgevy 依托 CRISPR 基因编辑技术改造患者自体造血干细胞，从根源改善血红蛋白缺陷问题，减少患者长期输血与血管闭塞危象发作频次，属于一次性治疗型细胞疗法。本次适应症扩容，进一步打开该基因疗法长期商业化空间，覆盖幼年确诊患者群体，扩充药品目标受众规模。
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