美国食品药品监督管理局（FDA）已批准福泰制药的基因疗法Casgevy的适应症拓展，将可用患者年龄下限扩展至2岁及以上 1。这标志着Casgevy成为全球首个可用于低龄儿童的CRISPR基因编辑疗法 1。该药物通过靶向BCL11A基因的红细胞特异性增强子区域重启胎儿血红蛋白，无需引入外源基因序列，用于治疗镰状细胞病和输血依赖型β地中海贫血 1。
临床试验数据支持该批准决定。在CLIMB系列研究的12-35岁患者队列中，32名患者实现了连续12个月无需输血，占比约91.4% 1。更低龄段的5-11岁儿童队列中，9名可评估患儿有8人达成12个月输血独立，有效比例接近89% 1。患者中位无需输血时长达到20.1个月 1。FDA从提交相关材料到做出批准决定仅用时53天，该项目已纳入FDA局长国家优先凭证试点项目 1。
为进一步了解治疗的长期安全性，所有接受治疗的患者被邀请参与CLIMB-131长期随访研究，计划追踪长达15年 1。
Casgevy, a gene therapy developed by Vertex Pharmaceuticals, has received FDA approval to treat patients as young as 2 years old, marking the first CRISPR gene-editing therapy available for young children with blood disorders 1. The therapy works by editing the BCL11A gene to reactivate fetal hemoglobin, addressing sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT) 1.
Clinical trial data demonstrated strong efficacy across different age groups: among 32 patients aged 12 to 35 years in the CLIMB trial series, all achieved at least 12 consecutive months without needing blood transfusions, representing a 91.4 percent transfusion independence rate 1. In the younger cohort of children aged 5 to 11 years, 8 out of 9 evaluable patients achieved 12-month transfusion independence, corresponding to an effectiveness rate of approximately 89 percent 1. The median duration without requiring transfusions reached 20.1 months across evaluated patients 1.
The FDA expedited its review process, approving Casgevy in just 53 days after submission, incorporating the application into the FDA Commissioner's Breakthrough Therapy Designation pilot program 1. The therapy employs a base-editing approach that targets the red blood cell-specific enhancer region of the BCL11A gene without introducing foreign genetic sequences 1. All patients who have received the treatment have been invited to participate in the CLIMB-131 long-term follow-up study, which is designed to track patients for up to 15 years 1.
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