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Osimertinib, a third-generation tyrosine kinase inhibitor targeting non-small cell lung cancer with EGFR T790M mutations. 奥西替尼,第三代酪氨酸激酶抑制剂,靶向EGFR T790M突变的非小细胞肺癌。
Pub Date : 2016-10-01 DOI: 10.1358/DOT.2016.52.10.2541343
C. McCoach, A. Jimeno
Oncogenic driver mutations in the epidermal growth factor receptor (EGFR) gene have provided a focus for effective targeted therapy. Unfortunately, all patients eventually develop resistance to frontline therapy with EGFR tyrosine kinase inhibitors (TKIs). The majority of patients develop a large subclonal population of tumor cells with a T790M mutation that renders these cells resistant to first-generation TKIs. Osimertinib is a third-generation EGFR TKI that was designed to overcome resistance from T790M mutations. This agent has demonstrated strong preclinical activity, and in the clinic it has demonstrated a high objective response rate and progression-free survival in patients with EGFR double mutations (L858R/T790M and exon 19 deletion/T790M). It is now approved by the FDA for patients who have a documented T790M mutation and who have progressed on a prior TKI. Osimertinib is also approved in the E.U. and Japan.
表皮生长因子受体(EGFR)基因的致癌驱动突变为有效的靶向治疗提供了一个焦点。不幸的是,所有患者最终都会对EGFR酪氨酸激酶抑制剂(TKIs)的一线治疗产生耐药性。大多数患者发展出大量具有T790M突变的肿瘤细胞亚克隆群体,使这些细胞对第一代TKIs具有耐药性。奥西替尼是第三代EGFR TKI,旨在克服T790M突变的耐药。该药物已显示出很强的临床前活性,在临床中,它已在EGFR双突变(L858R/T790M和外显子19缺失/T790M)患者中显示出较高的客观缓解率和无进展生存率。目前,FDA已批准该药物用于有T790M突变记录且在先前TKI治疗中取得进展的患者。奥西替尼也在欧盟和日本获得批准。
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引用次数: 9
Stem Cell and Regenerative Medicine Global Conference (SCRGC) 2016 (August 23-24, 2016 - Gyeonggi-do, Korea). 2016年干细胞与再生医学全球会议(SCRGC)将于2016年8月23日至24日在韩国京畿道举行。
Pub Date : 2016-10-01 DOI: 10.1358/dot.2016.52.10.2544830
A. Vertès
In its third edition, the Stem Cell and Regenerative Medicine Global Conference (SCRGC) organized by the Global Stem Cell & Regenerative Medicine Acceleration Center (GSRAC) was focused on breaking barriers to accelerate the pace of innovation and development of the regenerative medicine industry. GSRAC is both a think tank and a global network of key opinion leaders from the public and the private sectors. GSRAC was commissioned in 2011 by the Ministry of Health and Welfare (MOHW) of Korea. GSRAC's primary mission is to enable and accelerate the delivery of innovative technologies to patients who are affected by currently untreatable diseases. This goal is notably achieved by resolving hurdles in the field of regenerative medicine. With a total of 30 speakers and panelists from 8 different countries and more than 400 attendees from an array of institutions including hospitals, clinics, biotechnology companies, pharmaceutical companies, scientists, as well as policy makers, the 2-day SCRGC highlighted critical challenges and paths to resolving them in policy and regulatory, and industrial-scale manufacturing of gene-based and cell-based therapies, comprising plenary lectures and sessions covering strategic policy, regulatory, reimbursement and business development, and business of manufacturing, and production technologies. Several of these presentations are summarized in this report.
由全球干细胞与再生医学加速中心(GSRAC)主办的第三届干细胞与再生医学全球会议(SCRGC)聚焦于打破障碍,加快再生医学产业的创新和发展步伐。GSRAC既是一个智库,也是一个由来自公共和私营部门的关键意见领袖组成的全球网络。GSRAC于2011年由韩国卫生和福利部(MOHW)委托。GSRAC的主要使命是促进并加速创新技术向目前无法治愈疾病的患者提供服务。这一目标主要是通过解决再生医学领域的障碍来实现的。来自8个不同国家的30位演讲者和小组成员,以及来自医院、诊所、生物技术公司、制药公司、科学家和政策制定者等一系列机构的400多名与会者,为期两天的scgc强调了在政策和监管方面的关键挑战和解决这些挑战的途径,以及基于基因和细胞的疗法的工业规模制造,包括全体会议和会议,涵盖战略政策,监管,报销和业务发展,以及制造和生产技术的业务。本报告总结了其中的一些介绍。
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引用次数: 0
Lifitegrast: First LFA-1/ICAM-1 antagonist for treatment of dry eye disease. Lifitegrast:首个用于治疗干眼病的LFA-1/ICAM-1拮抗剂
Pub Date : 2016-09-01 DOI: 10.1358/DOT.2016.52.9.2542066
D. Paton
Dry eye disease is an extremely common condition affecting millions worldwide. The underlying pathophysiological mechanism is thought to be localized inflammation of the ocular surface resulting in the localization of T cells at this surface followed by their activation and subsequent liberation of cytokines. This effect on T cells results from the binding of lymphocyte function-associated antigen-1 (LFA-1) located on T cells to intercellular adhesion molecule 1 (ICAM-1) expressed on inflamed epithelium and endothelium, and on T cells. Lifitegrast is a T-cell integrin antagonist designed to mimic ICAM-1, thus blocking the interaction of LFA-1 and ICAM-1. Lifitegrast enters the systemic circulation to a limited extent thus reducing the likelihood of unwanted systemic reactions. Clinical trials in over 2,500 subjects with dry eye disease have shown that 5.0% lifitegrast given by ocular instillation causes a significant reduction in objective and subjective signs and symptoms of the disease. These beneficial effects are associated with a relatively low incidence of unwanted effects, almost all local in nature. In light of these findings, lifitegrast was approved by the Food and Drug Administration (FDA) in 2016 for the treatment of dry eye disease, the first drug with this mechanism of action to be so approved.
干眼病是一种极其常见的疾病,影响着全世界数百万人。潜在的病理生理机制被认为是眼表的局部炎症导致T细胞在该表面的定位,随后它们被激活并随后释放细胞因子。这种对T细胞的影响是由于位于T细胞上的淋巴细胞功能相关抗原-1 (LFA-1)与炎症上皮和内皮以及T细胞上表达的细胞间粘附分子1 (ICAM-1)结合。lifitgrast是一种t细胞整合素拮抗剂,旨在模拟ICAM-1,从而阻断LFA-1和ICAM-1的相互作用。lifitgrast进入体循环的程度有限,从而减少了不必要的全身反应的可能性。在2500多名干眼病患者中进行的临床试验表明,通过眼滴注给予5.0%立替格司特,可显著减少干眼病的客观和主观体征和症状。这些有益的影响与不良影响的发生率相对较低有关,几乎都是局部性的。鉴于这些发现,美国食品和药物管理局(FDA)于2016年批准了lifitegrast用于治疗干眼症,这是第一个获得批准的具有这种作用机制的药物。
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引用次数: 15
Zabofloxacin for chronic bronchitis. 沙星治疗慢性支气管炎。
Pub Date : 2016-09-01 DOI: 10.1358/DOT.2016.52.9.2530595
B. Kocsis, D. Szabo
Treatment of lower respiratory tract infection poses as an ongoing challenge among respiratory tract diseases. Bacterial infections are causes of acute exacerbations in chronic bronchitis and indications for antibacterial therapy. Several antibiotics were applied to treat bacterial infections in chronic bronchitis, among them fluoroquinolones are considered potent, broad-spectrum agents with excellent tissue penetration. This monograph focuses on zabofloxacin, a novel fluoroquinolone agent recently approved and launched in South Korea, and summarizes the drug's antibacterial efficacy, pharmacokinetic properties and toxicity. Recent advances concerning fluoroquinolones in chronic bronchitis will be discussed, along with a comparison between zabofloxacin and moxifloxacin. Zabofloxacin has proved to be noninferior to moxifloxacin against major community-acquired Gram-positive and Gram-negative respiratory tract pathogens and found to be well tolerated in both oral and parenteral administrations. These features can make it a potential antimicrobial agent in therapy of chronic bronchitis and other lower respiratory tract infections.
下呼吸道感染的治疗是呼吸道疾病中一个持续的挑战。细菌感染是慢性支气管炎急性加重的原因,也是抗菌治疗的适应症。几种抗生素被用于治疗慢性支气管炎细菌感染,其中氟喹诺酮类药物被认为是有效的,广谱的药物,具有良好的组织穿透性。本专著重点介绍了最近在韩国获批上市的新型氟喹诺酮类药物扎氟沙星的抗菌功效、药代动力学特性和毒性。本文将讨论氟喹诺酮类药物治疗慢性支气管炎的最新进展,并对扎氟沙星和莫西沙星进行比较。扎氟沙星已被证明对主要社区获得性革兰氏阳性和革兰氏阴性呼吸道病原体的治疗效果不逊于莫西沙星,并且在口服和肠外给药中均具有良好的耐受性。这些特点使其成为治疗慢性支气管炎和其他下呼吸道感染的潜在抗菌剂。
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引用次数: 8
GTCBio's Precision Medicine Conference (July 7-8, 2016 - Boston, Massachusetts, USA). GTCBio精准医学会议(2016年7月7-8日,美国马萨诸塞州波士顿)。
Pub Date : 2016-09-01 DOI: 10.1358/DOT.2016.52.9.2550577
P. Cole
GTCBio's Precision Medicine Conference met this year to outline the many steps forward that precision medicine and individualized genomics has made and the challenges it still faces in technological, modeling, and standards development, interoperability and compatibility advancements, and methods of economic and societal adoption. The conference was split into four sections, 'Overcoming Challenges in the Commercialization of Precision Medicine', 'Implementation of Precision Medicine: Strategies & Technologies', 'Integrating & Interpreting Personal Genomics, Big Data, & Bioinformatics' and 'Incentivizing Precision Medicine: Regulation & Reimbursement', with this report focusing on the final two subjects.
今年GTCBio的精准医学会议概述了精准医学和个性化基因组学所取得的许多进步,以及它在技术、建模和标准开发、互操作性和兼容性进步以及经济和社会采用方法方面仍然面临的挑战。会议分为四个部分,“克服精准医疗商业化的挑战”、“精准医疗的实施:战略与技术”、“整合与解读个人基因组学、大数据和生物信息学”和“激励精准医疗:监管与报销”,本报告重点关注最后两个主题。
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引用次数: 0
Combination therapies for improved management of lower urinary tract symptoms/benign prostatic hyperplasia. 改善下尿路症状/良性前列腺增生管理的联合疗法
Pub Date : 2016-09-01 DOI: 10.1358/DOT.2016.52.9.2525739
C. de Nunzio, F. Presicce, A. Tubaro
Several urological and non-urological conditions can contribute to the onset of lower urinary tract symptoms (LUTS), including benign prostatic hyperplasia (BPH), which is one of the main underlying causes in male patients. Six pharmacological classes (alpha-adrenoceptor blockers [ABs], 5alpha-reductase inhibitors [5ARIs], phytotherapeutics, antimuscarinics [AMs], beta3-adrenoceptor agonists and phosphodiesterase type 5 inhibitors [PDE5Is]) are available, alone or in combination, for the treatment of male LUTS. The aim of this review is to summarize the latest evidence on combination medical treatments for male patients with LUTS/BPH. Standard combinations include AB + 5ARI (for patients with increased prostate volume who are at risk for BPH progression); AB + PDE5I (for patients with concomitant erectile dysfunction); and AB + AM or beta3 agonist (for patients with persistent storage symptoms and not at risk for acute urinary retention). Other possible multidrug treatments have been proposed in preliminary studies, but further randomized controlled trials are needed to determine whether these putative strategies will eventually be considered a new standard for patients with LUTS/BPH. The possibility of tailoring BPH treatment according to different patient characteristics and expectations, using two or more drugs, seems a promising path in the field of LUTS/BPH management; however, physicians should consider the risk of increasing costs without proven long-term efficacy with most of these combination treatments.
一些泌尿系统和非泌尿系统疾病可导致下尿路症状(LUTS)的发生,包括良性前列腺增生(BPH),这是男性患者的主要潜在原因之一。6类药物(α -肾上腺素能受体阻滞剂[ABs]、α -还原酶抑制剂[5ARIs]、植物疗法、抗uscarins [AMs]、β -肾上腺素受体激动剂和磷酸二酯酶5型抑制剂[PDE5Is])可单独或联合用于治疗男性LUTS。本文综述了男性LUTS/BPH联合治疗的最新证据。标准组合包括AB + 5ARI(适用于前列腺体积增大且有前列腺增生进展风险的患者);AB + PDE5I(用于伴有勃起功能障碍的患者);和AB + AM或β 3激动剂(适用于有持续性尿潴留症状且无急性尿潴留风险的患者)。其他可能的多药治疗已在初步研究中提出,但需要进一步的随机对照试验来确定这些假定的策略最终是否会被视为LUTS/BPH患者的新标准。根据不同患者的特点和期望,使用两种或两种以上的药物来定制BPH治疗的可能性,似乎是LUTS/BPH管理领域的一个有前途的途径;然而,医生应该考虑到大多数这些联合治疗在没有证实长期疗效的情况下增加费用的风险。
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引用次数: 10
Drug Information Association (DIA) - 52nd Annual Meeting (June 26-30, 2016 - Philadelphia, Pennsylvania, USA). 药物信息协会(DIA)第52届年会(2016年6月26-30日,美国宾夕法尼亚州费城)。
Pub Date : 2016-08-01 DOI: 10.1358/DOT.2016.52.8.2539366
R. Ballinger, M. Spence, W. Chalkley
The Drug Information Association's Annual Meeting is the largest global event which crosses all disciplines involved in the discovery development and life cycle management of healthcare products. The 2016 meeting, its 52nd occurrence, brought experts from regulatory and government agencies, industry, academia and health and patient organizations together to discuss novel therapies in development and how to use these to enhance health and well-being in patient groups, with the hope of increasing knowledge across all areas involved.
药物信息协会年会是全球最大的活动,涉及医疗保健产品的发现、开发和生命周期管理的所有学科。2016年的第52次会议汇集了来自监管和政府机构、工业界、学术界以及卫生和患者组织的专家,共同讨论正在开发的新疗法以及如何利用这些疗法来增强患者群体的健康和福祉,希望增加所有相关领域的知识。
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引用次数: 0
Notable deals in the pharmaceutical industry in the third quarter of 2016. 2016年第三季度医药行业值得关注的交易。
Pub Date : 2016-08-01 DOI: 10.1358/DOT.2016.52.8.2540100
E. Cruces
During the third quarter of 2016, Cortellis Competitive Intelligence had 865 new deals added as part of its ongoing coverage of pharmaceutical licensing activity. This figure shows similar deal activity compared with the same quarter of 2015 (934), and 21% less than the previous quarter, although they had similar disclosed financial sizes in comparison with the first half of 2016. This article will focus on a number of the most valuable and notable deals forged during the quarter, as well as a selection of deals from some of the most prolific deal makers. An update on milestones, options and terminated deals of significance will also be presented, along with an early outlook on the last quarter of 2016's pharmaceutical licensing activity.
2016年第三季度,Cortellis Competitive Intelligence新增了865笔交易,这是其持续覆盖的药品许可活动的一部分。该数据显示,与2015年同期(934笔)相比,交易活动相似,比上一季度减少了21%,尽管与2016年上半年相比,它们披露的财务规模相似。本文将重点介绍本季度达成的一些最有价值、最引人注目的交易,以及一些最多产的交易撮合者的精选交易。会议还将介绍里程碑、备选方案和已终止的重要交易的最新情况,以及2016年最后一个季度药品许可活动的早期展望。
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引用次数: 0
Notable deals in the pharmaceutical industry in the second quarter of 2016. 2016年第二季度医药行业值得关注的交易。
Pub Date : 2016-08-01 DOI: 10.1358/dot.2016.52.8.2545018
P. D'Souza
During the second quarter of 2016, Cortellis Competitive Intelligence had 1,014 new deals added as part of its ongoing coverage of pharmaceutical licensing activity. This was on par with the last quarter (1,011) and a substantial increase on the same quarter for the previous 1 year (659). This article will focus on highlighting a number of the most valuable and notable deals forged during the quarter, as well as a selection of deals from some of the most prolific deal makers. An update on milestone, options and terminated deals of significance will also be presented, along with an early outlook on the next quarter's pharmaceutical licensing activity.
2016年第二季度,Cortellis Competitive Intelligence新增了1,014笔交易,这是其持续覆盖的药品许可活动的一部分。这与上一季度(1011人)持平,比前一年同期(659人)大幅增加。本文将重点介绍本季度达成的一些最有价值、最引人注目的交易,以及一些最多产的交易撮合者的精选交易。会议还将介绍里程碑、可选方案和已终止的重要交易的最新情况,以及对下一季度药品许可活动的初步展望。
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引用次数: 0
Rolapitant hydrochloride: prophylactic treatment for chemotherapy-induced nausea and vomiting. 盐酸罗拉匹坦:预防化疗引起的恶心和呕吐。
Pub Date : 2016-08-01 DOI: 10.1358/DOT.2016.52.8.2525738
R. Navari
Chemotherapy-induced nausea and vomiting (CINV) is a significant clinical issue which affects patients' quality of life as well as treatment decisions. Significant improvements in the control of CINV have occurred in the past 15 years with the introduction of new antiemetic agents: 5-HT3 receptor antagonists, tachykinin NK1 receptor antagonists and olanzapine. Aprepitant was the first NK1 receptor antagonist introduced (2003) for the prevention of CINV in combination with a 5-HT3 receptor antagonist and dexamethasone. Two additional NK1 receptor antagonists, netupitant and rolapitant, were approved by the FDA in 2014 and 2015, respectively. A description of rolapitant and its role in CINV will be presented, along with a comparison to the other NK1 receptor antagonists, aprepitant and netupitant.
化疗引起的恶心和呕吐(CINV)是一个重要的临床问题,影响患者的生活质量和治疗决策。在过去的15年里,随着新的止吐剂的引入,CINV的控制有了显著的改善:5-HT3受体拮抗剂、速激肽NK1受体拮抗剂和奥氮平。阿瑞吡坦是2003年引入的第一个用于预防CINV的NK1受体拮抗剂,与5-HT3受体拮抗剂和地塞米松联合使用。另外两种NK1受体拮抗剂netupitant和rolaptant分别于2014年和2015年获得FDA批准。将介绍罗拉匹坦及其在CINV中的作用,并与其他NK1受体拮抗剂阿瑞匹坦和尼图匹坦进行比较。
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引用次数: 1
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Medicamentos de actualidad. Drugs of today
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