商品名称：KYPROLIS
通用名称：CARFILZOMIB(来那度胺)
英文名字:lenalidomide
英文别名:3-(7-amino-3-oxo-1h-isoindol-2-yl)piperidine-2,6-dione
中文别名:3-(7-氨基-3-氧代-1H-异吲哚-2-基)哌啶-2,6-二酮
生产商：ONYX PHARMS INC
规格：60MG
剂型：注射剂
用途：新型免疫调节药,主要用于治疗多发性骨髓瘤和骨髓增生异常综合征。
类风湿性关节炎，不同于与年龄相关的骨关节炎，它是一种使人日益衰弱的自身免疫性疾病，这意味着人的身体在攻击自己的组织。这种疾病的特点是广泛的组织、滑膜、关节炎症。
新药tofacitinib，也称JAK抑制剂，可阻止参与疾病的炎性免疫反应激活。
美国食品和药物管理局7月20日批准Kyprolis（来那度胺）用于此前至少经过两个优先疗法包括万珂（硼替佐米）和免疫调节剂治疗的多发性骨髓瘤患者的治疗。
多发性骨髓瘤是发生在血浆细胞中的血癌，它通常生长在骨髓、大多数的骨骼海绵软组织内。血细胞由骨髓生产。根据美国癌症协会预测，2012年将有21700人被诊断出患有多发性骨髓瘤，10710将死于这种疾病。
通过对266例事先接受了硼替佐米和沙利度胺治疗的多发性骨髓瘤复发患者进行的临床研究，对Kyprolis的安全性和有效性进行了评估。
此项研究的目的是测定治疗后肿瘤完全或部分消失患者比例（总响应率）。总响应率23％。响应时间中位数为7.8个月。
在超过30％的参加研究者中观察到的最常见副作用有疲劳、低血细胞计数和血小板水平、气短、腹泻、发烧。观察到的与Kyprolis有关的严重副作用包括心脏衰竭和呼吸急促。如果发生这些严重副作用，应密切监测患者并予治疗。
Kyprolis是通过FDA加速审批程序获批的，加速审批程序允许FDA依据临床试验数据显示出的能够合理预测临床效益的替代终点审批治疗严重疾病的药物。加速审批程序旨在为患者更早地提供充满希望的新药物。在该药物批准上市后，公司还须提交确认药物临床效益的更多的临床资料。
Krypolis由加利福尼亚州南旧金山的Onyx制药公司负责销售。

Generic Name: carfilzomib
Date of Approval: July 20, 2012
Company: Onyx Pharmaceuticals, Inc.
Treatment for: Multiple Myeloma

FDA Approves Kyprolis

The U.S. Food and Drug Administration (FDA) has granted accelerated approval of Kyprolis (carfilzomib) for Injection, a proteasome inhibitor, 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. The indication for Kyprolis is based on response rate. Currently, no data are available for Kyprolis that demonstrate an improvement in progression-free survival or overall survival.
The approval was based on the results of the Phase 2b 003-A1 study, a single-arm, multicenter clinical trial that enrolled 266 patients with multiple myeloma who had received a median of five prior anti-myeloma regimens. The primary efficacy endpoint was overall response (ORR) and determined by an Independent Review Committee using the International Myeloma Working Group (IMWG) criteria. ORR was 22.9% and median response duration was 7.8 months.
Safety data were evaluated in 526 patients with relapsed and/or refractory multiple myeloma who received single-agent carfilzomib. There were 37 deaths on study, or 7% of patients. The most common causes of death, other than disease progression, were cardiac (5 patients), end-organ failure (4 patients), and infection (4 patients).
Important warnings and precautions include:
•cardiac arrest
•congestive heart failure
•myocardial ischemia
•pulmonary hypertension
•pulmonary complications
•infusion reactions
•infusion reactions
•tumor lysis syndrome
•thrombocytopenia
•hepatic toxicity
•embryo-fetal toxicity.
The most common serious adverse reactions were pneumonia, acute renal failure, pyrexia, and congestive heart failure.
The most common adverse reactions (incidence of 30% or greater) observed in clinical trials of patients with multiple myeloma were fatigue, anemia, nausea, thrombocytopenia, dyspnea, diarrhea, and pyrexia.
Serious adverse reactions were reported in 45% of patients.
Important Safety Information Regarding Kyprolis (carfilzomib) for Injection
Death due to cardiac arrest has occurred within a day of Kyprolis administration. Patients with New York Heart Association Class III and IV heart failure, myocardial infarction in the preceding 6 months, and conduction abnormalities uncontrolled by medications were not eligible for the clinical trials. These patients may be at greater risk for cardiac complications.
Pulmonary arterial hypertension (PAH) was reported in 2% of patients treated with Kyprolis and was Grade 3 or greater in less than 1% of patients. Dyspnea was reported in 35% of patients enrolled in clinical trials. Grade 3 dyspnea occurred in 5%; no Grade 4 events, and 1 death (Grade 5) was reported.
Infusion reactions, characterized by a spectrum of systemic symptoms including fever, chills, arthralgia, myalgia, facial flushing, facial edema, vomiting, weakness, shortness of breath, hypotension, syncope, chest tightness, or angina can occur immediately following or up to 24 hours after administration of Kyprolis. Administration of dexamethasone prior to Kyprolis reduces the incidence and severity of reactions. Tumor lysis syndrome (TLS) occurred following Kyprolis administration in < 1% of patients. Patients with multiple myeloma and a high tumor burden should be considered to be at greater risk for TLS.
Thrombocytopenia following Kyprolis administration resulted in a dose reduction in 1% of patients and discontinuation of treatment with Kyprolis in < 1% of patients.
Cases of hepatic failure, including fatal cases, have been reported (< 1%). Kyprolis can cause elevations of serum transaminases and bilirubin.
There are no adequate and well-controlled studies in pregnant women using Kyprolis. Females of reproductive potential should be advised to avoid becoming pregnant while being treated with Kyprolis.

KYPROLIS Rx

Generic Name and Formulations:
Carfilzomib 60mg/vial; lyophilized pwd for IV inj after reconstitution; preservative-free.

Company:
Onyx Pharmaceuticals