FDA已经批准蛋白酶抑制剂carfilzomib(Kyprolis)用于接受过其他疗法但效果不佳的多发性骨髓瘤患者的治疗。批准该药用于至少两种已有疗法(其中之一必须为硼替佐米,Velcade)无效的复发性或难治性骨髓瘤患者的治疗。获得FDA加速审批程序1个月后，carfilzomib在肿瘤药物咨询委员会（ODAC）投票表决中得到了11:0（其中1票弃权）的高度认同，成功获得批准。
“Kyprolis的获批为那些现有疗法无法控制病情发展的多发性骨髓瘤患者提供了一种新的选择，”FDA血液及肿瘤药品办公室负责人、医学博士Richard Pazdur在一份报告中如是说。“过去十年来多发性骨髓瘤治疗药物领域取得的进展让我们倍受鼓舞，治疗方法得到了极大改善”。
临床前及临床试验证实Carfilzomib是一种可以有效治疗对硼替佐米产生耐药的骨髓瘤的二代蛋白酶抑制剂。此外，它避免了硼替佐米等一代药物的外周神经毒性。然而，正如ODAC成员在他们投票表决前的讨论中提到的那样，Carfilzomib也并非没有副作用。实际上，FDA提交给ODAC的评核报告中也提到了carfilzomib的副作用（包括心、肝及肺毒性以及可致死）会影响它的治疗效果。由于目前缺少靶向的治疗手段，咨询委员会最终决定同意推荐此药通过审核。
FDA在批准报告中声明，30%以上的患者使用carfilzomib后出现副作用,常见疲劳、嗜中性粒细胞减少、血小板降低、呼吸困难、腹泻及发热。加速审批程序下FDA可以在临床数据有限的情况下批准临床上确有疗效的药物。Carfilzomib便是由于良好的临床效果获得快速审批的。
如此快速获批甚至出乎药物生产者（加利福尼亚州南旧金山Onyx制药）的意料，他们甚至已经开始进行更深入的研究以证实之前的结果。在Onyx的一份声明中，公司官员称已有两项III期临床试验在进行之中，其中一项就是为了证实在快速审批获得通过中起关键作用的结果。
“它的获批为其他疗法无法达到预期效果的骨髓瘤患者提供了一种新的选择，”新泽西哈肯萨克大学医疗中心医学博士David Siegel在Onyx的报告中如是说。Kyprolis具有单一制剂活性，使得临床医生有望更好地治疗那些尚无有效治疗方法的骨髓瘤患者。

INDICATIONS
KYPROLIS is indicated for the treatment of patients with multiple myeloma who have received at least two prior therapies including bortezomib and an immunomodulatory agent and have demonstrated disease progression on or within 60 days of completion of the last therapy. Approval is based on response rate [see Clinical Studies]. Clinical benefit, such as improvement in survival or symptoms, has not been verified.
SIDE EFFECTS
The following adverse reactions are discussed in greater detail in other sections of the labeling:
•Cardiac Arrest, Congestive Heart Failure, Myocardial Ischemia [see WARNINGS AND PRECAUTIONS]
•Pulmonary Hypertension [see WARNINGS AND PRECAUTIONS]
•Pulmonary Complications [see WARNINGS AND PRECAUTIONS]
•Infusion Reactions [see WARNINGS AND PRECAUTIONS]
•Tumor Lysis Syndrome [see WARNINGS AND PRECAUTIONS]
•Thrombocytopenia [see WARNINGS AND PRECAUTIONS]
•Hepatic Toxicity and Hepatic Failure [see WARNINGS AND PRECAUTIONS]
The most common adverse reactions (incidence of 30% or greater) to KYPROLIS observed in clinical trials of patients with multiple myeloma were fatigue, anemia, nausea, thrombocytopenia, dyspnea, diarrhea, and pyrexia.
Dosage Forms And Strengths
KYPROLIS single-use vial contains 60 mg of carfilzomib as a sterile, white to off-white lyophilized cake or powder.
KYPROLIS (carfilzomib) for Injection is supplied as an individually cartoned single-use vial containing a dose of 60 mg of carfilzomib as a white to off-white lyophilized cake or powder.
Clinical Trials Safety Experience
Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared with rates in the clinical trials of another drug, and may not reflect the rates observed in medical practice.