FDA批准多发性骨髓瘤新药.doc

FDA批准多发性骨髓瘤新药
发布时间:2012-7-30 来源:药品资讯网信息中心
FDA(abases/)于7月20日批准Kyprolis(carfilzomib)治疗先前至少已经尝试过两种疗法,包括Velcade(国内注册名万珂;硼替佐米)和免疫(nd/?bigclassname=%E8%A5%BF%E8%8D%AF%E4%BA%A7%E5%93%81&smallclassname=%E5%85%8D%E7%96%AB%E7%B3%BB%E7%BB%9F)治疗的多发性骨髓瘤患者。
 
在一项有266名先前至少接受过两种疗法,包括Velcade和Thalomid(thalidomide,沙利度***)的复发的多发性骨髓瘤患者参与的临床(/)研究(/),对Kyprolis的安全性和效果作了评价。
 
在这项研究(/)中,超过30%的参与者中观察到的最常见的副作用有疲劳、低血细胞计数和血小板水平、气急、腹泻、发烧。
Carfilzomib (CFZ) is a tetrapeptide epoxyketone and a selective proteasome inhibitor. It is an analog of epoxomicin.[1]
Discovery, early development and regulatory approval
Carfilzomib is derived from epoxomicin, a natural product that was shown by the laboratory of Craig Crews at Yale University to inhibit the proteasome.[2] The Crews laboratory subsequently invented a more specific derivative of epoxomicin named YU101,[3] which was licensed to Proteolix, Inc.. Scientists at Proteolix modified YU101 to create carfilzomib, which they advanced to multiple Phase 1 and 2 clinical trials, including a pivotal Phase 2 clinical trial designed to seek accelerated approval. Clinical trials for carfilzomib continue under Onyx Pharmaceuticals, which acquired Proteolix in 2009. In January 2011, the . FDA granted carfilzomib fast-track status, allowing Onyx to initiate a rolling submission of its new drug application for carfilzomib.[4] In December 2011, t