美国FDA批准诺华基因疗法治疗罕见肌肉疾病

格隆汇
Nov 25, 2025

诺华制药公司周一表示,美国食品和药物管理局已批准该公司为 一种罕见肌肉疾病患者开发基因疗法。该疗法被命名为Itvisma,获准用于治疗脊髓性肌萎缩症患者,这些患者年龄在两岁及两岁以上,且确诊存在存活运动神经元1基因突变。Itvisma的活性成分与这家瑞士制药商的旧疗法 Zolgensma 相同,后者在美国被批准用于治疗两岁以下的SMA患者。新疗法的批发价为259万美元,而Zolgensma的批发价为210万美元。诺华公司说, Itvisma是 第一种也是唯一一种适用于广大人群的基因替代疗法。

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