下图为根据国家食药监总局公开信息，整理的8月1日重要新药受理信息，按注册类型排序，供参考。
2、Cold Genesys癌症药物CG0070已获Ally Bridge集团1360万美元现金投资发布日期：2014-08-04 来源：fiercebiotech
加州生物技术公司Cold Genesys的癌症治疗药物已斩获1360万美元A轮投资，该药有可能会感染肿瘤细胞并刺激免疫系统加入对肿瘤细胞的战斗。
近日，加州生物技术公司Cold Genesys的癌症治疗药物已斩获1360万美元A轮投资，该药有可能会感染肿瘤细胞并刺激免疫系统加入对肿瘤细胞的战斗。来自Ally Bridge集团现金支持有利于Cold Genesys正在进行的对CG0070的2/3期研究，这是其主要的候选药物。该药物是一种溶瘤病毒可以直接靶向癌细胞，通过双重机制杀死肿瘤细胞，首先在细胞内增殖以干扰肿瘤细胞生长途径，然后表达基因GM-CSF，使体内的树突状细胞和T细胞加入战斗。Cold Genesys从BioSante授权引进该药物，后者已经完成了1期研究，涉及35例非肌肉浸润性膀胱癌（NMIBC）的治疗。现在，该生物技术公司准备推进CG0070上市用于治疗该疾病，在三月已经开始招募2/3期研究和期望于2018年获得最终数据。首席执行官Yeung在声明中称，“Ally Bridge集团作为战略投资者在关键时刻加入了我们的CG0070治疗NMIBC研究项目，不仅使我们能够完成BOND试验的3期阶段，也使我们能够立即开展研究治疗中晚期膀胱癌等的方案，包括其他实体瘤的治疗研究方案。”Ally Bridge集团是一家以香港为总部的投资集团，主要投资对象是美国和中国的药物和医疗设备开发商。该公司的产品组合包括有潜力的IPO公司Otonomy，RestorGenex和mmunGene。信源地址：http://www.fiercebiotech.com/sto ... ng-virus/2014-07-31
3、NICE不推荐来那度胺用于硼替佐米治疗后复发的患者 发布日期：2014-08-04 来源：pmlive
NICE新的草案指南未推荐塞尔基因来那度胺用于治疗使用杨森硼替佐米治疗后复发的多发性骨髓瘤患者。
NICE新的草案指南未推荐塞尔基因来那度胺用于治疗使用杨森硼替佐米治疗后复发的多发性骨髓瘤患者。多发性骨髓瘤发生于骨髓细胞，这种癌症目前尚不能治愈，但一种沙利度胺方案通常作为一种一线治疗用来延缓疾病的进展。硼替佐米在NHS作为一种治疗选择已被推荐用于不能使用沙利度胺的患者，虽然塞尔基因提供了更多的来那度胺相关信息，但NICE仍未推荐这款药物用于这一适应症。NICE目前已发布草案指南征求意见。如果征求意见结束，它将不会推荐来那度胺与地塞米松合并用于治疗症状已出现首次复发及之前接受过硼替佐米治疗的多发性骨髓瘤患者。来那度胺也不会被推荐用于对沙利度胺禁忌或不能耐受的患者及干细胞移植不适合的患者。自2009年以来，没有临床研究证明来那度胺与其它治疗药物的对比效果，塞尔基因的进一步数据“不能减少结果的不确定性”，NICE称。针对这次新的评估，塞尔基因尚未向NICE提交一份患者获取计划，虽然目前该药物的指南基于一项计划，即生产商资助这款药物至少26个周期，通常为两年。推荐的25mg起始剂量的21粒胶囊包装费用为4368欧元，在每一周期的1–21天，每天口服用药一次，28天为一周期。来那度胺在欧洲获批用于治疗罕见血液肿瘤骨髓增生异常综合征(MDS)患者，与地塞米松合并用于治疗多发性骨髓瘤。在美国，这款药物除了用于这两种适应症之外，还获批用于治疗套细胞淋巴瘤。信源地址：
http://www.pmlive.com/pharma_new ... _of_revlimid_589750
4、阿斯利康加快推进癌症免疫治疗药物MEDI4736开发 发布日期：2014-08-04 来源：reuters
阿斯利康正加速推进其癌症免疫治疗药物的开发，该公司计划测试其关键试验药物用于新的肿瘤类型。
阿斯利康正加速推进其癌症免疫治疗药物的开发，该公司计划测试其关键试验药物用于新的肿瘤类型。在新型癌症免疫药物的竞跑中，阿斯利康被认为排在第四位，落后于罗氏、默沙东和百时美施贵宝。但阿斯利康药物MEDI4736仍被认为是一款非常有前景的药物，无论是其单独用药，还是与其它治疗药物合并用药。阿斯利康近日表示，除了正在进行的肺癌试验之外，该公司今年将启动一项MEDI4736用于头颈癌的关键临床试验项目，该公司还在寻求用于其它癌症的扩展试验。“今年底，除了头颈癌与非小细胞肺癌之外，我们有可能会宣布另外一种肿瘤类型，”药物开发主管Morrison在一个结果发布后的电话会议中对分析师表示称。MEDI4736是备受关注的抗PD-L1类治疗药物中的一款药物，这类药物可阻止肿瘤逃避免疫系统防御能力。阿斯利康表示，该公司将在9月份的欧洲医学肿瘤学会年会上发布进一步的MEDI4736用于肺癌的数据，以及用于头颈癌的早期结果。分析师表示，研究MEDI4736用于头颈癌的决定有意义，因为令人鼓舞的结果与6月份报道的默沙东药物用于该疾病的结果类似。MEDI4736的商业前景被阿斯利康在其最近同辉瑞的收购斗争中大肆宣扬，当时该公司认为这款药物单独用药及其与其它药物合并用药可能会产生65亿美元的年销售额。最近几年，阿斯利康旗下的药物主要以瑞舒伐他汀和埃索美拉唑为代表。但该公司在肿瘤领域有很长的历史，数十年前在开发他莫昔芬及其它激素治疗药物方案也开辟了新的天地。该公司还生产吉非替尼，这款药物属于首批靶向性癌症药物。此外，阿斯利康第二季度的销售收入和利润均超过预期，之后，该公司于早些时候提高了其2014年全年的销售收入和利润预测。信源地址：
http://www.reuters.com/article/2 ... USKBN0G01H420140731
阿斯利康正加速推进其癌症免疫治疗药物的开发，该公司计划测试其关键试验药物用于新的肿瘤类型。在新型癌症免疫药物的竞跑中，阿斯利康被认为排在第四位，落后于罗氏、默沙东和百时美施贵宝。但阿斯利康药物MEDI4736仍被认为是一款非常有前景的药物，无论是其单独用药，还是与其它治疗药物合并用药。阿斯利康近日表示，除了正在进行的肺癌试验之外，该公司今年将启动一项MEDI4736用于头颈癌的关键临床试验项目，该公司还在寻求用于其它癌症的扩展试验。“今年底，除了头颈癌与非小细胞肺癌之外，我们有可能会宣布另外一种肿瘤类型，”药物开发主管Morrison在一个结果发布后的电话会议中对分析师表示称。MEDI4736是备受关注的抗PD-L1类治疗药物中的一款药物，这类药物可阻止肿瘤逃避免疫系统防御能力。阿斯利康表示，该公司将在9月份的欧洲医学肿瘤学会年会上发布进一步的MEDI4736用于肺癌的数据，以及用于头颈癌的早期结果。分析师表示，研究MEDI4736用于头颈癌的决定有意义，因为令人鼓舞的结果与6月份报道的默沙东药物用于该疾病的结果类似。MEDI4736的商业前景被阿斯利康在其最近同辉瑞的收购斗争中大肆宣扬，当时该公司认为这款药物单独用药及其与其它药物合并用药可能会产生65亿美元的年销售额。最近几年，阿斯利康旗下的药物主要以瑞舒伐他汀和埃索美拉唑为代表。但该公司在肿瘤领域有很长的历史，数十年前在开发他莫昔芬及其它激素治疗药物方案也开辟了新的天地。该公司还生产吉非替尼，这款药物属于首批靶向性癌症药物。此外，阿斯利康第二季度的销售收入和利润均超过预期，之后，该公司于早些时候提高了其2014年全年的销售收入和利润预测。信源地址：
http://www.reuters.com/article/2 ... USKBN0G01H420140731
5、FDA实权人物Woodcock力促加速批准杜氏肌营养不良症药物 发布日期：2014-08-04 来源：fiercebiotech
Woodcock指出FDA致力于“探索和利用所有可能用于治疗杜氏肌营养不良症的药物，包括采用加速批准的手段（如适用）。
FDA的Woodcock可能是Sarepta首席执行官Garabedia今天最喜欢的人。Woodcock向一些杜氏肌营养不良领域（DMD）的患者发出了一封信，指出FDA致力于“探索和利用所有可能用于治疗杜氏肌营养不良症的药物，包括采用加速批准的手段（如适用），而Sarepta公司在信中被提到。7月30日Sarepta的股价因此大量上涨，而投资者也关注对公司积极的影响，首席执行官一直坚持称，FDA已经转而支持其努力为eteplirsen申请的快速审批。Woodcock是FDA一个有影响力的知情人士，但她的声明事实上没有什么是新的或者真的值得注意的地方。FDA并非在公开反对新型药物，尤其是像DMD这种还没有治疗方法的疾病。患者都需要新的治疗方法，特别是和深受这个致命肌肉消耗性疾病折磨的孩子的父母。因此任何不利于早期批准的行动将都有可能引发不满。但是，这一次可能会有所不同。我们有理由相信，FDA已经真正改变了其关于加快批准DMD药物的态度，Woodcock可能要做的不仅是安抚愤怒的家长等等。这次开放政策明显是在针对一些清晰的迹象，即大西洋两岸的监管机构都在不断改变自己的立场，可能会尽快批准三个相互竞争的实验性疗法。在FDA遭受一次挫折后，Sarepta领导公司快速转换策略，与监管机构会面后准备开始加速批准计划。“在欧洲，我们多次向CHMP提交了我们的全部临床数据，最近收到了推荐Translarna用于无义突变型杜氏肌营养不良症的积极意见，”Peltz在发给媒体FierceBiotech一份声明中表示，“在欧洲委员会批准前，我们预计Translarna将在欧洲获得批准用于DMD治疗的根本原因在于这是第一个该领域的药物。在美国，我们计划近期与美国食品药品管理局接触，以期在可能的途径下获得额外反馈和讨论，让Translarna尽快用于美国患者。”时间会告诉我们，FDA的Woodcock是否只是故作姿态。Sarepta的股票在一天之中经历大幅波动，30日股价飙升13%，但所有这三个DMD药物研发公司的股票价格都在31日下滑5%左右。信源地址：
http://www.fiercebiotech.com/sto ... ated-oks/2014-07-31
6、百特和Halozyme制药免疫缺陷药物HyQiva获BPAC建议批准 发布日期：2014-08-04 来源：新药汇
百特国际（Baxter International）和Halozyme制药7月31日联合宣布，FDA血液制品顾问委员会（BPAC）以15比1的投票结果，认为HyQvia用于治疗原发性免疫缺陷症（PI）具有良好的风险/效益属性，建议批准HyQvia用于原发性免疫缺陷症（PI）的治疗。FDA预计将于2014年第三季度作出审查决定。HyQvia由人正常免疫球蛋白（IGSC，10%）和重组人透明质酸酶（hyaluronidase）组成，透明质酸酶有利于IGSC的分散和吸收，从而提高其生物利用度。FDA曾于2012年因安全性问题拒绝批准HyQiva。之后，百特和Halozyme应FDA的要求，提交了额外的临床前数据，并于2013年12月提交了有关HyQvia的一份修订版生物制品许可申请（BLA），以便重新启动关于HyQvia用于原发性免疫缺陷（PI）成人患者治疗的审查程序。原有BLA的提交，基于一项前瞻性、开放标签、非对照、多中心III期临床试验的数据。该项研究评估了HyQvia用于预防急性严重细菌感染的安全性和有效性，同时与静脉给药型免疫球蛋白的药代动力学参数进行了比较。该项研究的目的是在单一的皮下注射位点注入一剂3或4周剂量的HyQvia。该项研究中，急性严重细菌感染率为0.025/人/年，低于所要求的疗效阈值1.0/人/年。对HyQvia的耐受性评估数据表明，最常见的不良反应为输注部位反应、头痛、疲劳、发热。HyQvia已于2013年5月获欧盟批准，作为一种替代疗法，用于原发性免疫缺陷综合征（primary immunodeficiency syndromes）及伴有继发性低丙种球蛋白血症和反复感染的骨髓瘤或慢性淋巴细胞白血病成人患者（≥18岁）的治疗。英文原文：FDA Advisory Committee Panel Provides Favorable Recommendation on Baxter's HyQvia for Primary Immunodeficiency DEERFIELD, Ill. and SAN DIEGO, Calif., July 31, 2014 - Baxter International Inc. (NYSE:BAX) and Halozyme Therapeutics, Inc., (NASDAQ:HALO) today announced that the Blood Products Advisory Committee (BPAC) of the U.S. Food and Drug Administration (FDA) voted 15-1 that HyQvia [Immune Globulin Infusion 10% (Human) with Recombinant Human Hyaluronidase], Baxter's investigational subcutaneous treatment for patients with primary immunodeficiency (PI), has a favorable risk/benefit profile.HyQvia was approved in Europe in 2013 for adults (≥18 years) with primary immunodeficiency syndromes and myeloma or chronic lymphocytic leukaemia (CLL) with severe secondary hypogammaglobulinaemia and recurrent infections.Data presented at today's advisory committee meeting included a review of the preclinical and clinical data supporting the HyQvia application. The FDA will consider the recommendation from the BPAC in its review of Baxter's amendment to the Biologics License Application (BLA) submitted in December 2013. The company expects the FDA response in the third quarter."We are hopeful that the positive support for HyQvia is the next step toward providing a new treatment option for patients with primary immunodeficiency in the United States. We look forward to working closely with the FDA as it completes its review," said Ludwig Hantson, Ph.D., president of Baxter BioScience."Today's BPAC vote underscores the strength of the HyQvia data and our rHuPH20 platform," commented Dr. Helen Torley, President and Chief Executive Officer of Halozyme. "Our rHuPH20 platform has been studied in thousands of patients spanning multiple disease states and a number of approved products."HyQvia was approved by the European Commission for EU member states in 2013 and is currently being prescribed in several European countries, including Germany, Netherlands, Sweden, Norway, Denmark, Ireland and Italy.about HyQvia
HyQvia is a product consisting of human normal immunoglobulin (IG 10%) and recombinant human hyaluronidase (licensed from Halozyme Therapeutics). The IG provides the therapeutic effect and the recombinant human hyaluronidase facilitates the dispersion and absorption of the IG administered subcutaneously, increasing its bioavailability. The IG is a 10% solution that is prepared from human plasma consisting of at least 98% IgG, which contains a broad spectrum of antibodies.HyQvia is indicated in Europe as replacement therapy in adults (≥18 years) with primary immunodeficiency syndromes and in myeloma or chronic lymphocytic leukaemia (CLL) with severe secondary hypogammaglobulinaemia and recurrent infections.
7、FDA批准Genzyme酶替代疗法Lumizyme用于全年龄段庞贝氏症 发布日期：2014-08-04 来源：新药汇
赛诺菲（Sanofi）旗下健赞（Genzyme）7月31日宣布，FDA已批准扩大Lumizyme（α-葡萄糖苷酶，algucosidase alfa）适应症，用于所有年龄段或表型的庞贝氏症（Pompe）患者的治疗。此前，FDA于2010年5月仅批准Lumizyme用于8岁及以上晚发型庞贝氏症患者的治疗。
庞贝氏症（Pompe）是一种由溶酶体酸性α-葡萄糖苷酶（GAA）基因缺陷或功能障碍导致的渐进性、致衰性且往往致命性的罕见遗传性神经肌肉疾病，全球发病率估计为4万分之一，主要症状为心脏衰弱和骨骼肌无力，逐渐发展为呼吸衰竭，最终因呼吸衰竭死亡。该病因基因突变导致机体无法生成足够的酸性α-葡萄糖苷酶（GAA），而GAA为正常的肌肉功能所必需。心脏和肌肉细胞利用GAA将糖原转化为能量，当GAA缺乏，糖原会在骨骼肌、平滑肌和心肌等组织细胞中蓄积，最终使心脏和肌肉衰竭。Lumizyme是一种水解性溶酶体糖原特异性酶，其作用机理是作为一种酶替代疗法，取代有缺陷的GAA，降低糖原在心脏和骨骼肌中的积累。在美国，α-葡萄糖苷酶以2个不同的生产规模生产。160升规模（初试规模）生产的α-葡萄糖苷酶品牌名为Myozyme，4000升规模（最终生产规模）生产的α-葡萄糖苷酶品牌名为Lumizyme。根据所提供的部分生化和临床数据，FDA得出结论，认为以2种规模生产的α-葡萄糖苷酶（即Myozume和Lumizyme）具有可比性（comparable），α-葡萄糖苷酶的整体安全信息保持不变。在美国以外，Lumizyme以商品名Myozyme上市销售，并已获超过65个国家批准。英文原文：
Genzyme Receives Label Expansion for Lumizyme® (alglucosidase alfa) in the United States for the treatment of Pompe DiseaseCAMBRIDGE, Mass.--(BUSINESS WIRE)--Genzyme, a Sanofi company (EURONEXT: SAN and NYSE: SNY), today announced that the U.S. Food and Drug Administration (FDA) approved a supplement to expand the indication for Lumizyme® (alglucosidase alfa). Lumizyme manufactured at the 4000L scale is now indicated for all Pompe patients of any age or phenotype. The approval of this indication is now consistent with that of the rest of the world, wher alglucosidase alfa manufactured at the 4000L is the only scale available. Previously, in the United States, Lumizyme had been approved only for patients with late onset Pompe disease.“We are thankful to the entire Pompe community who has been on this journey to provide a sustainable, long-term option for Pompe patients in the United States,” said Genzyme President and CEO, David Meeker, M.D. “We are pleased we can now offer alglucosidase alfa produced at the 4000L scale to all patients in the US.”In the United States, alglucosidase alfa is manufactured at two different production scales. Alglucosidase alfa manufactured at the 160L scale (initial pilot scale) has a brand name of Myozyme® (alglucosidase alfa)and alglucosidase alfa manufactured at the 4000L (final manufacturing scale) has a brand name of Lumizyme.based on the biochemical and clinical data provided as part of the submission, FDA concluded that alglucosidase alfa manufactured at both scales in the US (i.e., Lumizyme and Myozyme) are comparable. The overall safety profile of alglucosidase alfa remains unchanged.Specific updates to the Lumizyme product label include:Updated Indication: LUMIZYME® (alglucosidase alfa) is a hydrolytic lysosomal glycogen-specific enzyme indicated for patients with Pompe disease (GAA deficiency).
Inclusion of safety and efficacy data from infantile-onset studies
Removal of the REMS program
updat to the boxed warning to include infantile-onset specific warning regarding fluid overload.
Updated to Pregnancy Category C classification
Lumizyme, marketed as Myozyme outside of U.S., is approved in more than 65 countries. For the complete Lumizyme label, visit
www.lumizyme.com.about Pompe DiseasePompe disease is a progressive, debilitating and often fatal neuromuscular disease caused by a genetic deficiency or dysfunction of the lysosomal enzyme acid alpha-glucosidase (GAA) affecting an estimated 1 in 40,000 people worldwide. This enzymatic defect results in the accumulation of glycogen primarily in muscle tissues that leads to muscle weakness, loss of respiratory function, and often premature death. Absent treatment, when symptoms occur in infancy, babies typically die within the first year of life. When symptoms occur in childhood or adulthood, patients often lose their ability to walk and require wheelchairs to assist with mobility and experience difficulty breathing as well as mechanical ventilation to breathe.important Safety Information for Lumizyme and MyozymeINDICATIONLUMIZYME® (alglucosidase alfa) is a hydrolytic lysosomal glycogen-specific enzyme indicated for patients with Pompe disease (acid α-glucosidase (GAA) deficiency).
8、Vertex囊性纤维化药物Kalydeco获欧盟批准 发布日期：2014-08-04 来源：新药汇
福泰制药（Vertex）7月31日宣布，药物Kalydeco（ivacaftor）获欧盟委员会（EC）批准，用于囊性纤维化跨膜电导调节因子（CFTR）基因中存在8种非G551D门控突变（non-G551D gating mutations）之一的6岁及以上囊性纤维化患者的治疗。此次获批，是基于二部分、随机、双盲、安慰剂对照III期研究的第一部分数据，该研究在39例携带非G551D门控突变的6岁及以上囊性纤维化（CF）患者中开展。第一部分数据表明，Kalydeco使肺功能（FEV1）、汗液氯化物、身体质量指数、CFQ-R得分均取得了统计学意义的显著改善。该项研究的第二部分数据已于2014年6月提交至欧洲囊性纤维化协会会议，数据表明，Kalydeco在研究的第一部分中所取得的改善，在整个治疗24周中均能够维持。该项研究中的安全性与在G551D门控突变患者中开展的III期研究一致。Kalydeco是首个靶向囊性纤维化（CF）根本病因的药物，可使G551D突变患者体内缺陷性CFTR蛋白发挥正常功能。G551D突变是一种最常见的门控突变，该突变损害了ATP介导的通道调节。此次获批的8种非G551D门控突变包括：G178R，S549N，S549R，G551S，G1244E，S1251N，S1255P和G1349D。在欧洲，约有250例患者携带这8种非G551D门控突变。关于
Kalydeco：Kalydeco于2012年首次获FDA及EMA批准，用于治疗CFTR基因存在至少单拷贝G551D突变的6岁及以上囊性纤维化（CF）患者。此外，FDA于2014年6月批准Kalydeco用于携带9种非G551D门控突变中任意一种突变的6岁及以上CF患者，包括：G178R，S549N，S549R，G551S，G1244E，S1251N，S1255P，G1349D或G970R。关于
囊性纤维化（CF）：
囊性纤维化（CF）是由囊性纤维化跨膜电导调节因子（CFTR）基因突变导致CFTR蛋白功能缺陷或缺失所致的罕见遗传性疾病，该病困扰着全球约7万人。CFTR蛋白通常调节细胞膜的离子运输，基因突变能导致蛋白产物功能的破坏或丧失。当细胞膜离子运输被中断，某些器官粘液涂层的粘度将变稠。该病的一个主要特征是呼吸道积聚厚厚的粘液，导致呼吸困难及反复感染。英文原文：
Vertex Receives European Approval for KALYDECO(TM) (ivacaftor) in Eight Non-G551D Gating Mutations-In Europe, approximately 250 people ages 6 and older have one of 8 additional gating mutations--KALYDECO is the first medicine to treat the underlying cause of CF in people with specific non-G551D gating mutations-EYSINS, Switzerland--(BUSINESS WIRE)-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the European Commission has approved KALYDECO™ (ivacaftor) for people with cystic fibrosis (CF) ages 6 and older who have one of eight non-G551D gating mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Today's approval follows the positive opinion from the Committee for Medicinal Products for Human Use (CHMP) in June 2014. KALYDECO was first approved in Europe in July 2012 for people with CF ages 6 and older who have the G551D mutation, which is the most common gating mutation. The eight additional gating mutations included in today's approval are: G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P and G1349D. In Europe, approximately 250 people ages 6 and older have one of these non-G551D gating mutations."Today's approval in people with additional gating mutations marks another step toward our goal of helping more people with this disease," said Simon Bedson, Senior Vice President and International General Manager at Vertex. "We are committed to working closely with the appropriate national authorities to make KALYDECO available for these patients as soon as possible."Today's approval is based on previously announced data from the first part of a Phase 3, two-part, randomised, double-blind, placebo-controlled, cross-over study of 39 people with CF ages 6 and older who have a non-G551D gating mutation. The first part of the study showed statistically significant improvements in lung function (FEV1), sweat chloride, body mass index and CFQ-R scores. Data from the second part of the study were presented at the European Cystic Fibrosis Society Conference in June 2014 and showed that these improvements were maintained through 24 weeks of treatment. The safety profile was similar to prior Phase 3 studies of KALYDECO in people with the G551D mutation."By treating the underlying cause of cystic fibrosis, KALYDECO has changed the way we treat patients with the most common gating mutation, G551D. In general, people with other gating mutations have similarly severe disease to people with the G551D mutation and have an urgent need for new medicines that address the underlying cause of the disease," said Kris De Boeck, M.D., Ph.D., Professor of Pediatric Pulmonology, University Hospital Gasthuisberg, Leuven, Belgium, and President-Elect of the European Cystic Fibrosis Society. "In the Phase 3 study in people with non-G551D gating mutations, KALYDECO led to rapid, significant and sustained improvements in lung function and other measures of disease."In addition, the CHMP approved the inclusion of data from the long-term follow-up PERSIST study in the KALYDECO label. PERSIST is a Phase 3, open-label, 96-week, rollover extension trial that evaluated the long-term safety and durability of treatment with KALYDECO by enrolling people ages 6 and older with at least one copy of the G551D mutation who completed 48 weeks of treatment in the Phase 3 ENVISION and STRIVE studies (placebo and KALYDECO treatment groups) and met other eligibility criteria. Results from PERSIST demonstrated that the safety and efficacy of KALYDECO seen in the Phase 3 STRIVE and ENVISION trials was maintained through nearly three years (144 weeks) in G551D patients.Cystic fibrosis is caused by a defective or missing CFTR protein that results from mutations in the CFTR gene. CFTR proteins act as channels at the cell surface that control the flow of salt and water into and out of the cell. In people with gating mutations, the CFTR protein at the cell surface is defective and does not work properly, causing abnormally thick, sticky mucus to build up in the lungs. The digestive tract and a number of other organs are also affected.KALYDECO, an oral medicine known as a CFTR potentiator, helps the CFTR protein function more normally once it reaches the cell surface. It targets the abnormal CFTR protein channels and opens them to allow chloride ions to move into and out of the cell, which helps thin the mucus so it can hydrate and protect the airways.about KALYDECO™ (ivacaftor)KALYDECO (ivacaftor) is the first medicine to treat the underlying cause of CF in people with specific mutations in the CFTR gene. Known as a CFTR potentiator, KALYDECO is an oral medicine that aims to help the CFTR protein function more normally once it reaches the cell surface, to help hydrate and clear mucus from the airways. KALYDECO (150mg, q12h) was first approved by the U.S. Food and Drug Administration in January 2012 for use in people with CF ages 6 and older who have at least one copy of the G551D mutation and in February 2014 for use in people with CF ages 6 and older who have one of the following additional CFTR mutations: G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P or G1349D. In the European unio, KALYDECO was approved in July 2012 for use in people with CF ages 6 and older who have at least one copy of the G551D mutation and in July 2014 for use in people with CF ages 6 and older who have one of eight additional gating mutations, including G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P or G1349D. In Canada, KALYDECO was first approved in November 2012 for use in people with CF ages 6 and older who have at least one copy of the G551D mutation and in June 2014 for use in people with CF ages 6 and older who have one of the following additional CFTR mutations: G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, G1349D or G970R.KALYDECO is also approved in Australia, New Zealand and Switzerland for use in people with CF ages 6 and older who have at least one copy of the G551D mutation in the CFTR gene.Vertex retains worldwide rights to develop and commercialise KALYDECO.INDICATION AND importANT SAFETY INFORMATION FOR KALYDECO™ (ivacaftor)Ivacaftor (150 mg tablets) is indicated for the treatment of cystic fibrosis (CF) in patients age 6 years and older who have a G551D mutation in the CFTR gene.In the United States and the European unio, ivacaftor is also indicated for the treatment of CF in patients age 6 and older who have one of the following mutations in the CFTR gene: G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, or S549R. In Canada, ivacaftor is indicated for these same mutations and additionally for G970R.Ivacaftor is not effective in patients with CF with 2 copies of the F508del mutation (F508del/F508del) in the CFTR gene. The safety and efficacy of ivacaftor in children with CF younger than 6 years of age have not been established.Elevated liver enzymes (transaminases; ALT and AST) have been reported in patients receiving ivacaftor. It is recommended that ALT and AST be assessed prior to initiating ivacaftor, every 3 months during the first year of treatment, and annually thereafter. Patients who develop increased transaminase levels should be closely monitored until the abnormalities resolve. Dosing should be interrupted in patients with ALT or AST of greater than 5 times the upper limit of normal. Following resolution of transaminase elevations, consider the benefits and risks of resuming ivacaftor dosing.Use of ivacaftor with medicines that are strong CYP3A inducers, such as the antibiotics rifampin and rifabutin; seizure medications (phenobarbital, carbamazepine, or phenytoin); and the herbal supplement St. John's Wort, substantially decreases exposure of ivacaftor and may diminish effectiveness. Therefore, co-administration is not recommended.The dose of ivacaftor must be adjusted when used concomitantly with strong and moderate CYP3A inhibitors or when used in patients with moderate or severe hepatic disease.Ivacaftor can cause serious adverse reactions including abdominal pain and high liver enzymes in the blood. The most common side effects associated with ivacaftor include headache; upper respiratory tract infection (the common cold), including sore throat, nasal or sinus congestion, and runny nose; stomach (abdominal) pain; diarrhea; rash; and dizziness. These are not all the possible side effects of ivacaftor. A list of the adverse reactions can be found in the product labeling for each country wher ivacaftor is approved. Patients should tell their healthcare providers about any side effect that bothers them or does not go away.Please see KALYDECO U.S. Prescribing Information, EU Summary of Product Characteristics, Canadian Product Monograph, Australian Consumer Medicine Information and Product Information, Swiss Prescribing Information and Patient Information, and the New Zealand Datasheet and Consumer Medicine Information.about Cystic FibrosisCystic fibrosis is a rare, life-shortening genetic disease affecting approximately 75,000 people in North America, Europe and Australia. Today, the median predicted age of survival for a person with CF is between 34 and 47 years, but the median age of death remains in the mid-20s.CF is caused by a defective or missing CFTR protein resulting from mutations in the CFTR gene. Children must inherit two defective CFTR genes — one from each parent — to have CF. There are more than 1,900 known mutations in the CFTR gene. Some of these mutations, which can be determined by a genetic, or genotyping test, lead to CF by creating non-working or too few CFTR protein at the cell surface. The defective function or absence of CFTR proteins in people with CF results in poor flow of salt and water into and out of the cell in a number of organs, including the lungs. This leads to the buildup of abnormally thick, sticky mucus that can cause chronic lung infections and progressive lung damage.Collaborative History with Cystic Fibrosis Foundation Therapeutics, Inc. (CFFT)Vertex initiated its CF research program in 1998 as part of a collaboration with CFFT, the nonprofit drug discovery and development affiliate of the Cystic Fibrosis Foundation. This collaboration was expanded to support the accelerated discovery and development of Vertex's CFTR modulators.
9、Alexion重症肌无力药物Eculizumab（Soliris）获欧盟孤儿药资格 发布日期：2014-08-04 来源：firstwordpharma
8月1日，亚力兄制药Alexion Pharmaceuticals (ALXN)宣布欧盟委员会授予其Eculizumab（Soliris）孤儿药资格，用于治疗重症肌无力(MG)患者
重症肌无力是一种罕见的、令人衰弱的神经系统疾病，由不受控制的补体激活引起。针对肌神经接点的抗体引起的不受控制补体激活可最终导致全身各种肌肉群发生深刻而使人衰弱的肌无力。“MG患者发生使人衰弱的肌肉无力，损害了他们行走、清晰说话、吞咽及某些情况下正常呼吸的能力，这可能导致危及生命的肌无力危象，” 亚力兄制药全球研发主管、执行副总裁、哲学博士Mackay称。“通过特定抑制终端补体通路，我们认为Eculizumab有可能会帮助遭受这种灾难性罕见疾病的患者。”Eculizumab是一种新类型的终端补体抑制剂，目前被批准用于阵发性睡眠性血红蛋白尿症(PNH)和非典型溶血尿毒综合征(aHUS)患者治疗，这两种疾病是使人衰弱的、极其罕见并危及生命的疾病，均由慢性不受控制的补体激活引起。Eculizumab未在任何国家获批用于治疗MG。亚力兄制药正为Eculizumab的国际多中心心、安慰剂对照注册试验招募难治性全身性MG患者。欧盟委员会授予的孤儿药资格是为激励开发医疗产品用以治疗、预防或诊断影响欧洲不超过万分之五患者的疾病或症状。如果Eculizumab最终获批用于指定的适应症，孤儿药资格将为亚力兄制药在欧盟提供某些收益和激励，包括一段时期的市场独占权。信源地址：http://www.firstwordpharma.com/node/1228021?tsid=28®ion_id=3#axzz399UVICTO
10、Biogen Idec聚乙二醇干扰素Plegridy获批用于MS治疗 发布日期：2014-08-04 来源：medscape
基于ADVANCE试验确定聚乙烯乙二醇β-1a治疗复发-缓解型多发性硬化症具有疗效性、安全性和耐受性。欧洲委员会（EC）批准Plegridy用于RRMS的治疗。
欧洲委员会（EC）批准Plegridy --- 一种由Biogen Idec公司开发的皮下注射剂型聚乙二醇干扰素β-1a药物，用于成人复发-缓解型多发性硬化症（RRMS）的治疗。聚乙二醇干扰素β-1α在干扰素β基础上经过了结构改良，延长药物半衰期，从而在不影响疗效的情况下，减少给药频率。具体给药方案为皮下注射聚乙二醇干扰素β-1a，2周/次。根据产品特征摘要（SmPC）的建议，起始剂量为63 μg（剂量1），增至94μg（剂量2），最后使用最大剂量125μg（剂量3），继而持续使用最大剂量。5月，Biogen Idec公司获得了欧洲药品管理局人用医药产品委员会（CHMP）的积极意见。随后Biogen Idec公司将聚乙烯乙二醇β-1a的应用申请提交至美国食品和药物管理局（FDA）。阳性结果：公司指出，该药物之所以获得EC的批准，主要基于近期一项大规模的关键性研究——ADVANCE试验（多中心、随机、双盲、安慰剂对照试验）。研究共纳入1516例RRMS成人患者。研究者发现，每2周接受1次治疗的患者，与接受安慰剂的患者相比，前者 1年内年复发率（ARR）显著降低，可减少36%。另外，聚乙二醇干扰素β-1a可降低12周残疾持续进展风险达38%，而相应24周的风险可降低54%。与安慰剂组相比，该药物可以减少86%的钆-增强病灶数量。而研究第二年的数据显示药物疗效和安全性与第一年观察到的结果一致。按照研究方案，在完成2年的ADVANCE研究后，患者可以选择加入开放标签性扩展研究—ATTAIN, 并进行为期4年的随访。药物安全性：ADVANCE试验中聚乙烯乙二醇β-1a的安全性和耐受性结果，与已知的MS干扰素疗效一致。药物治疗与安慰剂对照两组间，出现整体不良事件和严重不良事件的几率相似。感染是最常见的严重不良事件，在两组间发生率均低于1%。与聚乙二醇干扰素β-1α相关的最常见的不良事件为注射部位发红及流感样疾病。本药物应慎用于有抑郁症、癫痫、严重肝功或肾功障碍病史的患者。药物治疗中曾出现细胞减少症状，包括罕见的中性粒细胞减少以及血小板减少。还出现的其它症状包括：转氨酶升高、严重过敏反应、皮下给药部位反应（包括注射部位坏死）以及心脏病恶化。另外，EU SmPC指出干扰素β类药物与肾病综合征、血栓性微血管疾病（具体表现为血栓性血小板减少性紫癜或溶血性尿毒症综合征）、肝炎、自身免疫性肝炎、严重的肝功能衰竭以及包括罕见全血细胞减少在内的外周血细胞计数减少性疾病相关。信源地址：http://www.medscape.com/viewarticle/828766
11、美国药物处方量、销售额排名(2013年7月至2014年6月) 发布日期：2014-08-04 来源：medscape 浏览次数：13 依据艾美仕市场研究公司最新的数据，左旋甲状腺素（Synthroid，艾伯维）仍然是全国处方量最大的药物，而阿立哌唑（Abilify，大冢制药）则继续保持美国销售额最高处方药的桂冠。
治疗甲状腺功能减退药物左旋甲状腺素（Synthroid，艾伯维）仍然是全国处方量最大的药物，而抗精神病药物阿立哌唑（Abilify，大冢制药）则继续保持美国销售额最高处方药的桂冠。
这些数据反映了过去12个月期间（2013年07月至2014年06月）美国处方量和销售额前100名药物情况。作为美国处方量最大药物，过去12个月期间，美国开出了2260万张左旋甲状腺素处方。继左旋甲状腺素钠之后是降胆固醇药物瑞舒伐他汀（Crestor，阿斯利康），大约有2250万张处方；第三名是质子泵抑制剂埃索美拉唑（Nexium，阿斯利康），约有1860万张处方；治疗哮喘药物沙丁胺醇（Ventolin HFA，葛兰素史克）以1750万张处方排名第四；而丙酸氟替卡松/沙美特罗吸入剂（Advair Diskus，葛兰素史克）则以1500万张处方排名第五。美国处方量最大药物排名前10的其他5种药物分别为：抗高血压药缬沙坦（代文，诺华）；降血糖药甘精胰岛素注射液（来得时，赛诺菲安万特）；抗抑郁药度洛西汀（欣百达，礼来）；治疗注意力缺陷药物二甲磺酸赖右苯丙胺（Vyvanse，夏尔公司）；以及抗癫痫药普瑞巴林（Lyrica，辉瑞）。作为美国销售额最高的处方药，2013年07月至2014年06月期间，阿立哌唑销售额高达72亿美元。其次是关节炎治疗药物阿达木单抗（Humira，艾伯维），63亿美元；埃索美拉唑（Nexium，阿斯利康），63亿美元；瑞舒伐他汀（Crestor，阿斯利康），56亿美元；以及关节炎治疗药物依那西普（恩利，安进公司），接近51亿美元。美国销售额最高药物排名前10的其他5种药物分别为：丙酸氟替卡松/沙美特罗吸入剂（Advair Diskus，葛兰素史克），50亿美元；抗病毒药索菲布韦（Sovaldi，吉利德公司），44亿美元；关节炎治疗药物英利昔单抗（类克，山陶克公司），43亿美元；降血糖药甘精胰岛素注射液（来得时，赛诺菲安万特），38亿美元；以及中性粒细胞减少症治疗药物聚乙二醇非格司亭（Neulasta，安进公司），接近37亿美元。表1 美国月处方量前100名药物| 排名 | 药名（商品名） | 2013.7–2014.6期间总处方量 |
| 1 | Synthroid | 22,664,826 |
| 2 | Crestor | 22,557,735 |
| 3 | Nexium | 18,656,464 |
| 4 | Ventolin HFA | 17,556,646 |
| 5 | Advair Diskus | 15,003,169 |
| 6 | Diovan | 11,401,503 |
| 7 | Lantus Solostar | 10,154,739 |
| 8 | Cymbalta | 10,065,788 |
| 9 | Vyvanse | 10,019,178 |
| 10 | Lyrica | 9,684,884 |
| 11 | Spiriva Handihaler | 9,518,849 |
| 12 | Lantus | 9,358,961 |
| 13 | Celebrex | 8,815,391 |
| 14 | Abilify | 8,777,842 |
| 15 | Januvia | 8,758,309 |
| 16 | Namenda | 7,640,319 |
| 17 | Viagra | 7,584,152 |
| 18 | Cialis | 7,555,933 |
| 19 | Zetia | 7,411,629 |
| 20 | Nasonex | 7,304,210 |
| 21 | Suboxone | 7,011,882 |
| 22 | Symbicort | 6,948,403 |
| 23 | Bystolic | 6,722,578 |
| 24 | Flovent HFA | 5,623,533 |
| 25 | Oxycontin | 5,559,330 |
| 26 | Levemir | 5,554,827 |
| 27 | Xarelto | 5,014,364 |
| 28 | Nuvaring | 5,011,966 |
| 29 | Dexilant | 4,866,178 |
| 30 | Thyroid | 4,834,481 |
| 31 | Benicar | 4,725,628 |
| 32 | Voltaren Gel | 4,709,766 |
| 33 | Proventil HFA | 4,494,004 |
| 34 | Tamiflu | 4,149,835 |
| 35 | Novolog | 4,044,310 |
| 36 | Novolog Flexpen | 4,006,690 |
| 37 | Premarin | 3,984,357 |
| 38 | Vesicare | 3,873,046 |
| 39 | Humalog | 3,858,256 |
| 40 | Benicar HCT | 3,633,026 |
| 41 | Lumigan | 3,283,060 |
| 42 | Afluria | 3,242,605 |
| 43 | Lo Loestrin Fe | 3,154,488 |
| 44 | Janumet | 3,089,749 |
| 45 | Ortho-Tri-Cy Lo 28 | 3,053,738 |
| 46 | Toprol-XL | 3,044,003 |
| 47 | Pristiq | 3,023,546 |
| 48 | Combivent Respimat | 2,994,490 |
| 49 | Vytorin | 2,988,460 |
| 50 | Travatan Z | 2,919,358 |
| 51 | Focalin XR | 2,866,278 |
| 52 | Pataday | 2,757,094 |
| 53 | Humalog Kwikpen | 2,626,530 |
| 54 | Lunesta | 2,590,519 |
| 55 | Avodart | 2,527,583 |
| 56 | Pradaxa | 2,442,678 |
| 57 | Seroquel XR | 2,405,130 |
| 58 | Strattera | 2,387,756 |
| 59 | Minastrin 24 Fe | 2,353,282 |
| 60 | Evista | 2,232,555 |
| 61 | Chantix | 2,151,879 |
| 62 | Zostavax | 2,145,562 |
| 63 | Humira | 1,923,427 |
| 64 | Victoza 3-Pak | 1,902,995 |
| 65 | Exelon | 1,877,942 |
| 66 | Exforge | 1,838,730 |
| 67 | Combigan | 1,821,491 |
| 68 | Dulera | 1,790,677 |
| 69 | Onglyza | 1,784,018 |
| 70 | Welchol | 1,778,218 |
| 71 | Premarin Vaginal | 1,718,405 |
| 72 | Enbrel | 1,627,480 |
| 73 | Xopenex HFA | 1,588,895 |
| 74 | Ranexa | 1,567,961 |
| 75 | Truvada | 1,532,734 |
| 76 | Alphagan P | 1,503,194 |
| 77 | Viibryd | 1,447,730 |
| 78 | Tradjenta | 1,443,791 |
| 79 | Effient | 1,434,572 |
| 80 | Azor | 1,433,045 |
| 81 | Norvir | 1,384,306 |
| 82 | Actonel | 1,339,615 |
| 83 | Namenda XR | 1,319,963 |
| 84 | Amitiza | 1,309,980 |
| 85 | Aggrenox | 1,286,826 |
| 86 | Lotemax | 1,276,480 |
| 87 | Patanol | 1,274,050 |
| 88 | Levitra | 1,273,311 |
| 89 | Advair HFA | 1,272,551 |
| 90 | Uloric | 1,244,536 |
| 91 | Detrol La | 1,225,506 |
| 92 | Asmanex Twisthaler | 1,219,112 |
| 93 | Lipitor | 1,212,376 |
| 94 | Atripla | 1,183,636 |
| 95 | Prempro Low Dose | 1,162,513 |
| 96 | Latuda | 1,123,396 |
| 7 | Novolog Flxpen Mix 70/30 | 1,119,855 |
| 98 | Invokana | 1,106,268 |
| 99 | Epiduo | 1,103,330 |
| 100 | Humulin N | 1,071,673 |
表1 美国销售额前100名药物| 排名 | 药名（商品名） | 2013.7–2014.6期间总销售额 |
| 1 | Abilify | $7,240,043,661 |
| 2 | Humira | $6,310,742,887 |
| 3 | Nexium | $6,303,738,580 |
| 4 | Crestor | $5,672,991,435 |
| 5 | Enbrel | $5,097,263,350 |
| 6 | Advair Diskus | $5,064,138,456 |
| 7 | Sovaldi | $4,469,558,675 |
| 8 | Remicade | $4,342,356,359 |
| 9 | Lantus Solostar | $3,829,943,226 |
| 10 | Neulasta | $3,688,450,342 |
| 11 | Copaxone | $3,677,307,402 |
| 12 | Rituxan | $3,361,247,463 |
| 13 | Spiriva Handihaler | $3,270,501,346 |
| 14 | Januvia | $3,124,826,108 |
| 15 | Lantus | $3,011,654,746 |
| 16 | Atripla | $2,898,264,003 |
| 17 | Cymbalta | $2,839,722,673 |
| 18 | Avastin | $2,784,813,413 |
| 19 | Lyrica | $2,782,036,977 |
| 20 | Oxycontin | $2,524,152,087 |
| 21 | Celebrex | $2,435,800,107 |
| 22 | Epogen | $2,378,212,523 |
| 23 | Truvada | $2,351,681,656 |
| 24 | Diovan | $2,177,824,165 |
| 25 | Levemir | $2,121,663,090 |
| 26 | Gleevec | $2,087,891,457 |
| 27 | Herceptin | $2,032,270,434 |
| 28 | Vyvanse | $1,940,313,035 |
| 29 | Lucentis | $1,932,528,073 |
| 30 | Zetia | $1,930,143,528 |
| 31 | Tecfidera | $1,908,407,807 |
| 32 | Symbicort | $1,901,446,790 |
| 33 | Namenda | $1,774,338,290 |
| 34 | Novolog Flexpen | $1,720,294,797 |
| 35 | Xarelto | $1,546,602,955 |
| 36 | Novolog | $1,466,291,783 |
| 37 | Humalog | $1,418,795,789 |
| 38 | Suboxone | $1,388,999,843 |
| 39 | Tysabri | $1,315,848,323 |
| 40 | Viagra | $1,274,239,087 |
| 41 | Seroquel XR | $1,266,841,322 |
| 42 | Victoza 3-Pak | $1,263,623,179 |
| 43 | Cialis | $1,241,580,547 |
| 44 | Stelara | $1,221,329,301 |
| 45 | Alimta | $1,215,044,019 |
| 46 | Nasonex | $1,190,958,069 |
| 47 | Avonex | $1,146,712,504 |
| 48 | Gilenya | $1,143,648,903 |
| 49 | Humalog Kwikpen | $1,140,418,800 |
| 50 | Flovent HFA | $1,091,998,384 |
| 51 | Prezista | $1,079,469,740 |
| 52 | Janumet | $1,071,110,813 |
| 53 | Renvela | $1,063,062,816 |
| 54 | Isentress | $1,045,929,798 |
| 55 | Procrit | $1,028,448,101 |
| 56 | Orencia | $1,017,584,725 |
| 57 | Dexilant | $1,010,512,519 |
| 58 | Vesicare | $1,001,127,504 |
| 59 | Stribild | $962,264,200 |
| 60 | Olysio | $943,547,154 |
| 61 | Synthroid | $927,886,999 |
| 62 | Neupogen | $922,013,084 |
| 63 | Avonex Pen | $920,097,484 |
| 64 | Reyataz | $918,922,508 |
| 65 | Zytiga | $918,603,420 |
| 66 | Xolair | $902,375,287 |
| 67 | Invega Sustenna | $891,518,099 |
| 68 | Benicar | $884,750,288 |
| 69 | Combivent Respimat | $881,992,234 |
| 70 | Pradaxa | $879,463,846 |
| 71 | Sensipar | $878,283,696 |
| 72 | Xgeva | $844,140,641 |
| 73 | Vytorin | $828,020,808 |
| 74 | Betaseron | $824,758,830 |
| 75 | Aranesp | $824,531,232 |
| 76 | Prevnar 13 | $818,701,788 |
| 77 | Latuda | $803,311,039 |
| 78 | Afinitor | $800,598,342 |
| 79 | Lunesta | $778,412,409 |
| 80 | Ventolin HFA | $771,201,081 |
| 81 | Complera | $756,268,942 |
| 82 | Synagis | $754,510,179 |
| 83 | Bystolic | $736,654,176 |
| 84 | Zyvox | $733,964,492 |
| 85 | Gardasil | $717,881,961 |
| 86 | Sandostatin Lar | $715,506,378 |
| 87 | Benicar HCT | $699,679,537 |
| 88 | Treanda | $692,288,820 |
| 89 | Pristiq | $687,192,710 |
| 90 | Zostavax | $674,280,328 |
| 91 | Erbitux | $669,382,439 |
| 92 | Cimzia | $660,599,949 |
| 93 | Strattera | $658,818,959 |
| 94 | Cubicin | $655,269,663 |
| 95 | Xeloda | $652,443,257 |
| 96 | Tarceva | $638,909,500 |
| 7 | Evista | $638,155,000 |
| 98 | Velcade | $636,704,153 |
| 99 | Sprycel | $635,192,399 |
| 100 | Abraxane | $634,678,206 |
信源地址：http://www.medscape.com/viewarticle/829246
12、勃林格殷格翰-礼来糖尿病联盟糖尿病新药Jardiance终获FDA批准
勃林格殷格翰-礼来糖尿病联盟8月1日联合宣布，糖尿病新药Jardiance（empagliflozin）获FDA批准，结合饮食及运动用于2型糖尿病成人患者的治疗，以改善血糖控制。Jardiance不适用于1型糖尿病患者，也不适用于伴有糖尿病酮症酸中毒（血液或尿液中酮体升高）的患者。
Jardiance是勃林格-礼来糖尿病联盟在美国获批的第3个糖尿病产品。Jardiance为每日一次的片剂，获批剂量为10mg和25mg。Jardiance可单独用药，也可与其他降糖药联合用药，包括胰岛素，二甲双胍。Jardiance的获批，是基于一项涵括超过10个跨国III期试验的大型临床项目的数据，涉及超过1.3万例2型糖尿病患者。该项目数据表明，Jardiance 10mg和25mg剂量作为单药疗法或与广泛的背景疗法（包括二甲双胍，磺酰脲，胰岛素，pioglitazone等）联合用药，均显著降低了患者的糖化血红蛋白（HA1c）水平，同时也显著降低了体重、血压。Jardiance属于钠-葡萄糖协同转运蛋白-2（SGLT-2）抑制剂类药物。新兴的SGLT-2抑制剂类药物，已被证实能够阻断肾脏中葡萄糖的再吸收作用，将过多的葡萄糖排泄到体外，从而达到降低血糖水平的效果，而且该降糖效果不依赖于β细胞功能和胰岛素抵抗。Jardiance的获批可谓是迟到了5个月之久。去年春天，FDA曾发出警告信，责骂勃林格未尽职调查莱茵河畔药物工厂出现的大颗粒污染事件，该工厂也同时生产empagliflozin。今年年3月，FDA据此理由拒绝批准勃林格-礼来联盟新药empagliflozin。此次遭拒，给礼来带来了不小的麻烦。今年6月，FDA根据审查结果，撤回了警告信。而勃林格-礼来联盟于2014年6月中旬向FDA重新提交了empagliflozin的新药申请（NDA）。Jardiance的获批，对礼来来说尤其重要。目前，礼来正面临着重磅产品的专利到期，正指望在短期内推出新产品以恢复增长。同时，Jardiance仍然代表着礼来同其他制药公司SGLT-2抑制剂类药物抗衡的最佳人选，如强生的Invokana、阿斯利康和百时美施贵宝的Farxiga（dapagliflozin）。Invokana于2013年11月获FDA批准，Farxiga则在经历长期审查推迟后也最终于2014年1月获FDA批准。（生物谷Bioon.com）英文原文：FDA approves Jardiance® (empagliflozin) tablets for adults with type 2 diabetesThird product from the BI-Lilly Diabetes alliance to be approved by FDARIDGEFIELD, Conn., and INDIANAPOLIS, Aug. 1, 2014 /PRNewswire/ -- Boehringer Ingelheim Pharmaceuticals, Inc. (BIPI) and Eli Lilly and Company (NYSE: LLY) today announced the U.S. Food and Drug Administration (FDA) approved Jardiance® (empagliflozin) tablets as an adjunct to diet and exercise to improve glycemic control, or blood glucose levels, in adults with type 2 diabetes (T2D). JARDIANCE is not for people with type 1 diabetes or for people with diabetic ketoacidosis (increased ketones in the blood or urine).JARDIANCE, a once-daily, 10 mg or 25 mg tablet, is a sodium glucose co-transporter-2 (SGLT2) inhibitor. JARDIANCE works by blocking the reabsorption of glucose in the kidney, increasing glucose excretion and lowering blood glucose levels in adults with T2D who have elevated blood glucose levels.Patients should not take JARDIANCE if they have severe kidney problems or are on dialysis, or if they are allergic to empagliflozin or any ingredient in JARDIANCE. JARDIANCE can cause some people to have dehydration (the loss of body water and salt), which may lead to a dro in blood pressure, and may cause patients to feel dizzy or faint."Many adults with type 2 diabetes still have difficulty controlling their blood sugar levels even with treatment. There is a critical need for new treatment options to help these patients," said Christophe Arbet-Engels, M.D., Ph.D., vice president, metabolic-clinical development and medical affairs, BIPI. "JARDIANCE is a new option that has been shown in clinical trials to reduce blood sugar levels. Although not approved for weight loss, modest weight loss was also observed in these clinical trials."The FDA approval is based on results from a large clinical program comprised of more than 10 multinational clinical trials and more than 13,000 adults with T2D. Phase III studies showed JARDIANCE significantly reduced hemoglobin A1C (a measure of average blood glucose over the past two to three months) and fasting blood sugar after 24 weeks as a stand-alone treatment or in combination with a range of background treatments, including metformin, sulfonylureas, insulin and pioglitazone. Although JARDIANCE is not approved for lowering weight or blood pressure, modest reductions in both weight and systolic blood pressure were observed in clinical trials.The most common adverse reactions associated with JARDIANCE were urinary tract infections and vaginal yeast infections. Hypoglycemia was more commonly reported in patients treated with the combination of JARDIANCE and sulfonylurea or insulin."Today's FDA approval of JARDIANCE provides an exciting new option in the treatment of adults with type 2 diabetes and demonstrates our commitment to these patients, as it marks the third diabetes medicine to emerge from our alliance pipeline," said Enrique Conterno, president, Lilly Diabetes. about Diabetes
Approximately 29 million Americans and an estimated 382 million people worldwide have type 1 or type 2 diabetes. T2D is the most common type, accounting for an estimated 85 to 95 percent of all diabetes cases. Diabetes is a chronic condition that occurs when the body either does not properly produce, or use, the hormone insulin.What is JARDIANCE?JARDIANCE is a prescription medicine used along with diet and exercise to lower blood sugar in adults with type 2 diabetes.JARDIANCE is not for people with type 1 diabetes or for people with diabetic ketoacidosis (increased ketones in the blood or urine).
13、GSK和Genmab制药单抗药Arzerra III期显著改善复发性CLL无进展生存期
葛兰素史克（GSK）和合作伙伴Genmab制药7月31日宣布，独立数据监测委员会（IDMC）针对抗癌药Arzerra（ofatumumab）一项关键性III期PROLONG研究（OMB 112517）进行的一项既定中期分析表明，该项研究达到了改善疾病无进展生存期（PFS）的主要终点。该项研究在复发性慢性淋巴细胞白血病（CLL）患者中开展，评估了Arzerra维持疗法与未进一步治疗（观察）的差异。该项研究中，IDMC未发现任何新的安全性信号，但会继续监测患者的安全，直到所有患者均完成治疗。该项研究的疗效和安全性数据正在进一步的分析中，详细数据将提交至监管机构及未来的科学会议。PROLONG研究是在复发性慢性淋巴细胞白血病（CLL）群体中评估Arzerra维持疗法的首个III期研究，该中期分析数据表明，Arzerra具有治疗复发性CLL的潜力，而目前尚无药物获批用于复发性CLL。PROLONG研究在532例病情复发时对治疗仍有响应的复发性CLL患者中开展，研究中，患者随机接受ofatumumab维持治疗或观察（不进行进一步治疗），ofatumumab治疗组初始剂量为300mg，一周后进行第二次给药（剂量为1000mg），随后每8周给药1000mg，治疗期为2年；而观察组不接受进一步的治疗。该项研究的主要终点是无进展生存期（PFS），次要终点包括评估临床受益、安全性、耐受性、健康相关生活质量、药代动力学。关于慢性淋巴细胞白血病（CLL）：在西方国家，CLL是成人白血病中最常见的类型，约占所有白血病病例的三分之一。在美国，估计目前有10.5万CLL或经治CLL患者，2013年中新增1.568万病例。CLL的平均确诊年龄为72岁，大约有90%的患者年龄超过55岁。大多数患者至少患有一种合并症（comorbidity），如高血压、糖尿病、心血管疾病或慢性阻塞性肺病（COPD）。关于Arzerra（ofatumumab）：Arzerra为单抗药ofatumumab的商品名，目前正由葛兰素史克（GSK）和Genmab制药联合开发，该药是一种创新的全人源化单克隆抗体，靶向于B细胞表面CD20分子的一个抗原表位，该表位包含了CD20分子的胞外大环和小环结构。Arzerra分别于2009年和2010年获FDA和EMA批准，用于对标准药物【阿仑单抗（alemtuzumab，Campath）或氟达拉滨（fludarabine）】治疗无应答的慢性淋巴细胞白血病（CLL）患者的治疗。英文原文：GSK and Genmab announce positive interim result for phase III study of ofatumumab as maintenance therapy for relapsed CLLGlaxoSmithKline plc (LSE/NYSE: GSK) and Genmab A/S (OMX: GEN) announced today that an Independent Data Monitoring Committee (IDMC) interim analysis of a phase III study, PROLONG (OMB 112517), reached the predefined significance level for efficacy (p≤0.001). The interim analysis demonstrated that treatment with ofatumumab (Arzerra™) met the primary endpoint of improving progression free survival (PFS). The study evaluated ofatumumab maintenance therapy versus no further treatment (observation) in patients with relapsed chronic lymphocytic leukaemia (CLL) who responded to treatment at relapse.The IDMC did not identify any new safety signals and will continue to monitor patients for safety until all study patients complete therapy. Further analysis of the safety and efficacy data is underway and will be shared with regulators and the scientific community in the coming months.“This interim result from the PROLONG study demonstrated that maintenance therapy with ofatumumab lowered the risk of disease progression in patients who responded to treatment at relapse. We look forward to sharing the results of the interim analysis with regulatory agencies to evaluate the potential for future regulatory filings,” said Dr. Rafael Amado, Head of oncology R&D, GSK.“We are very pleased that this study of ofatumumab, the first phase III study to evaluate maintenance therapy for relapsed CLL, met the primary endpoint at the interim analysis. This result indicates the potential of ofatumumab in this setting wher there are currently no approved treatments. We look forward to presenting the detailed data from this study at a future medical conference,” said Jan van de Winkel, PhD, Chief Executive Officer of Genmab.about PROLONGThis pivotal phase III study was designed to randomise up to 532 patients with relapsed CLL who have responded to treatment at relapse, to either ofatumumab maintenance treatment or no further treatment (observation). Patients in the ofatumumab arm receive an initial dose of 300 mg of ofatumumab, followed one week later by a second dose of 1,000 mg, then doses of 1,000 mg every 8 weeks for up to two years, while patients in the observation treatment arm receive no further treatment.The primary endpoint of the study is PFS. Secondary objectives will evaluate clinical benefit, safety, tolerability, the health-related quality of life of subjects treated with ofatumumab versus no further treatment, and pharmacokinetics among relapsed CLL patients receiving maintenance therapy with ofatumumab.about CLLCLL, the most commonly diagnosed adult leukaemia in western countries, accounts for approximately one-third of all cases of leukaemia.,[ii],[iii] In the USA, it is estimated that more than 105,000 people currently live with or have been previously treated for CLL and an estimated 15,680 new cases of CLL were diagnosed in the past year.3,[iv] The average age of diagnosis is 72 years, and approximately 90 per cent of patients with CLL are estimated to be over the age of 55 years.3,[v] The majority of patients with CLL have at least one comorbidity such as hypertension, diabetes, cardiovascular disease, or COPD.about ofatumumab (Arzerra)Ofatumumab—a monoclonal antibody that is designed to target the CD20 molecule found on the surface of CLL cells and normal B lymphocytes—is not approved or licensed anywher in the world as maintenance treatment for relapsed CLL.In the USA, ofatumumab is approved for use in combination with chlorambucil for the treatment of previously untreated patients with CLL for whom fludarabine-based therapy is considered inappropriate. In the EU, ofatumumab is approved for use in combination with chlorambucil or bendamustine for the treatment of patients with CLL who have not received prior therapy and who are not eligible for fludarabine-based therapy. Ofatumumab is also approved for first-line use in Russia.In more than 50 countries worldwide, ofatumumab is indicated as monotherapy for the treatment of patients with CLL refractory to fludarabine and alemtuzumab.Ofatumumab is being developed under a co-development and collaboration agreement between Genmab and GSK.Arzerra is a trademark of the GSK group of companies.
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