美国FDA在参考了Kalydeco(ivacaftor)的体外试验数据以及早期临床数据后,批准Kalydeco可用于治疗更多的囊肿性纤维化(CF)患者,该决定使Kalydeco的适应证从10个基因突变,扩展到33个。Kalydeco由总部位于波士顿的Vertex制药公司开发,可用于治疗2岁以上携带有CFTR基因突变的患者。(源自:药品资讯网) The US FDA approved Kalydeco for the treatment of more patients with cystic fibrosis (CF) after referring to in vitro data from Kalydeco (ivacaftor) as well as early clinical data. This decision led to the extension of Kalydeco’s indications from 10 mutations To 33. Developed by Vertex Pharmaceuticals, a Boston-based company, Kalydeco can be used to treat patients older than 2 years who carry mutations in the CFTR gene. (From: Drug Information Network)