2015年7月2日由美国食品和药品监督管理局(FDA)批准由鲁玛卡托(lumacaftor)和依伐卡托(ivacaftor)组成的复方药物在美国上市。该药由Vertex制药公司研发,商品名为Orkambi。Orkambi是第一个批准用于治疗囊性纤维化的药物,主要用于治疗有F508del突变的12岁以上患者的囊性纤维化[1]。囊性纤维化(CF)是一种慢性、渐进、通常致命的隐性 July 2, 2015 Approved by the U.S. Food and Drug Administration (FDA) for the listing of compounded drugs consisting of lumacaftor and ivacaftor in the United States. The drug developed by Vertex Pharmaceutical Company, under the trade name Orkambi. Orkambi is the first drug approved for the treatment of cystic fibrosis and is mainly used to treat cystic fibrosis in patients over the age of 12 with the F508del mutation [1]. Cystic fibrosis (CF) is a chronic, progressive, often fatal recessive