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(/databases/db_11_1.html)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(/brand/zs.asp?bigclassname=%E8%A5%BF%E8%8D%AF%E4%BA%A7%E5%93%81smallclassname=%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，沙利度胺）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 U.S. FDA granted carfilzomib fast-track status, allowing Onyx to initiate a rolling submission of its new drug application for carfilzomib.[4] In December 2011, the FDA granted Onyx standard review designation, [5][6] for its new drug application submission based on the 003-A1 study, an open-label, single-arm Phase 2b trial. The trial evaluated 266 heavily-pretreated patients with relapsed and refractory multiple myeloma who had received at least two prior therapies, including bortezomib and either thalidomide or lenalidomide.[7] Carfilzomib was approved by the FDA for use in patients with relapsed and refractory multiple myeloma on 20 July 2012.[8] Onyx expects to launch the drug in the U.S. on 1 August 2012. When it launches, it will cost $10,000 p