—新型降胆固醇药REPATHA(evolocumab)injection获FDA批准上市 2015年8月27日,美国食品和药品监管局(FDA)批准Repatha(evolocumab)injection为有些患者胆固醇用当前治疗选择他们的低密度脂蛋白(LDL)不能控制。 Repatha，在新类型药物被称为PCSK9抑制剂的第二个皮批准药物，被批准在有杂合子家族超胆固醇血症(HeFH)，纯合子家族性超胆固醇血症(HoFH)，或临床动脉粥样硬化心血管疾病成年患者，例如心脏发作或卒中，需要另外LDL胆固醇的降低患者使用除了饮食和最大耐受他汀类药物治疗。 家族性超胆固醇血症(包含HeFH和HoFH二者)是一种遗传条件致LDL胆固醇的高水平。在血中LDL胆固醇的高水平是连接至心血管病或心脏病。心脏病对美国是死亡第一原因，男性和女性二者都是。根据疾病控制和预防中心，在美国每年死于心脏病约610,000人– 等同于每死亡四例中一例。 药品评价和研究中心新药室主任John Jenkins，M.D说：“Repatha提供在这个新类型另一个治疗心脏药物为患者有家族性超胆固醇血症或已知心血管病用他汀类不足以减低其LDL胆固醇，” “心血管病是严重威胁美国人健康，和FDA承诺促进发展和批准有效和安全药物解决这个重要的公共健康问题。” Repatha是一种抗体靶向已知特异性蛋白，被称为PCSK9。PCSK9减低在肝中从血液去除LDL胆固醇受体数。通过阻断PCSK9的能力起作用，可得到更多受体以从血液摆脱LDL胆固醇而，结果较低LDL胆固醇水平。 在一项52-周安慰剂-对照试验和八项12-周安慰剂-对照试验在参加者有原发性高脂血症，包括两项专门纳入有HeFH参加者和一项纳入有HoFH参加者评价Repatha的疗效和安全性。在2-周研究之一，329例有HeFH参加者，需要另外降低胆固醇尽管有或无他汀类其它脂质降低治疗，被随机化接受Repatha或安慰剂共12周。用Repatha参加者与安慰剂比较有平均减低LDL胆固醇约60%。 Repatha的最常见副作用包括鼻咽炎，上呼吸道感染，流感，背痛，和给予注射处的反应例如发红，疼痛，或瘀伤。使用Repatha曾报道过敏反应例如皮疹和荨麻疹。如果他们出现了严重的过敏反应症状，患者应停止使用Repatha和得到医疗帮助。 多个临床试验曾显示 他汀类减低心脏发作和卒中。正在进行一项试验评价添加Repatha至他汀类对减低心血管风险的影响。 Repatha由总部在加州Thousand OaksAmgen公司上市。 Important U.S. Product Information Repatha is indicated as an adjunct to diet and: •Maximally tolerated statin therapy for treatment of adults with heterozygous familial hypercholesterolemia (HeFH) or clinical atherosclerotic cardiovascular disease (ASCVD), who require additional lowering of low-density lipoprotein cholesterol (LDL-C) •Other LDL-lowering therapies (e.g., statins, ezetimibe, LDL apheresis) in patients with homozygous familial hypercholesterolemia (HoFH) who require additional lowering of LDL-C Limitations of Use The effect of Repatha on cardiovascular morbidity and mortality has not been determined. Important Safety Information Repatha™ is contraindicated in patients with a history of a serious hypersensitivity reaction to Repatha. Hypersensitivity reactions (e.g. rash, urticaria) have been reported in patients treated with Repatha, including some that led to discontinuation of therapy. If signs or symptoms of serious allergic reactions occur, discontinue treatment with Repatha, treat according to the standard of care, and monitor until signs and symptoms resolve. The most common adverse reactions (>5% of Repatha-treated patients and more common than placebo) were: nasopharyngitis, upper respiratory tract infection, influenza, back pain, and injection site reactions.  In a 52-week trial, adverse reactions led to discontinuation of treatment in 2.2% of Repatha-treated patients and 1% of placebo-treated patients. The most common adverse reaction that led to Repatha treatment discontinuation and occurred at a rate greater than placebo was myalgia (0.3% versus 0% for Repatha and placebo, respectively). Adverse reactions from a pool of the 52-week trial and seven 12-week trials, included: Local injection site reactions that occurred in 3.2% and 3.0% of Repatha-treated and placebo-treated patients, respectively. The most common injection site reactions were erythema, pain, and bruising. The proportions of patients who discontinued treatment due to local injection site reactions in Repatha-treated patients and placebo-treated patients were 0.1% and 0%, respectively.  Allergic reactions occurred in 5.1% and 4.6% of Repatha-treated and placebo-treated patients, respectively. The most common allergic reactions were rash (1.0% versus 0.5% for Repatha and placebo, respectively), eczema (0.4% versus 0.2%), erythema (0.4% versus 0.2%), and urticaria (0.4% versus 0.1%). Neurocognitive events were reported in less than or equal to 0.2% in Repatha-treated and placebo-treated patients. In a pool of placebo- and active-controlled trials, as well as open-label extension studies that followed them, a total of 1609 patients treated with Repatha had at least one LDL‑C value < 25 mg/dL. Changes to background lipid-altering therapy were not made in response to low LDL-C values, and Repatha dosing was not modified or interrupted on this basis. Although adverse consequences of very low LDL-C were not identified in these trials, the long-term effects of very low levels of LDL-C induced by Repatha are unknown. Musculoskeletal adverse reactions were reported in 14.3% of Repatha-treated patients and 12.8% of placebo-treated patients. The most common adverse reactions that occurred at a rate greater than placebo were back pain (3.2% versus 2.9% for Repatha and placebo, respectively), arthralgia (2.3% versus 2.2%), and myalgia (2.0% versus 1.8%). In 49 patients with homozygous familial hypercholesterolemia studied in a 12-week, double-blind, randomized, placebo-controlled trial, 33 patients received 420 mg of Repatha subcutaneously once monthly. The adverse reactions that occurred in at least 2 (6.1%) Repatha-treated patients and more frequently than in placebo-treated patients, included upper respiratory tract infection (9.1% versus 6.3%), influenza (9.1% versus 0%), gastroenteritis (6.1% versus 0%), and nasopharyngitis (6.1% versus 0%). Repatha is a human monoclonal antibody. As with all therapeutic proteins, there is a potential for immunogenicity with Repatha. About Amgen Cardiovascular Building on more than three decades of experience in developing biotechnology medicines for patients with serious illnesses, Amgen is dedicated to addressing important scientific questions to advance care and improve the lives of patients with cardiovascular disease, the leading cause of morbidity and mortality worldwide.9 Amgen’s research into cardiovascular disease, and potential treatment options, is part of a growing competency at Amgen that utilizes human genetics to identify and validate certain drug targets. Through its own research and development efforts, as well as partnerships, Amgen is building a robust cardiovascular portfolio consisting of several approved and investigational molecules in an effort to address a number of today’s important unmet patient needs, such as high cholesterol and heart failure. http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/default.htm