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2024年FDA批准的50款新药概览 患者 治疗 药物 新药 领域 -健康界189.00 US$Animals Amla (Gifu) January 14, 2026与此前FDA批准的另一款SMA基因疗法Spinraza相比，Zolgensma（AVXS-101）无论在疗效还是成本效益上都具有优势。 Givosiran（Givlaritm）用于治疗成人急性肝卟啉症，这是一种导致有毒卟啉分子积聚的遗传性疾病。 Givosiran是一个双链siRNA，由Alnylam开发的。 2017年，FDA批准Keytruda作为"不限癌种"（也被称为广谱抗癌药）疗法治疗微卫星不稳定性高（MSI-H）或错配修复缺陷（dMMR）实体瘤患者，这是FDA批准的首款不依照肿瘤来源，...