Imfinzi and Tremelimumab Granted Orphan Drug Designation in the US for Liver Cancer

Imfinzi 和 Tremelimumab 在美国被批准用于肝癌的孤儿药物指定

2020-02-14 12:40:11 Drugs

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20 January 2020 -- AstraZeneca’s Imfinzi (durvalumab) and tremelimumab, an anti-CTLA4 antibody and potential new medicine, have both been granted Orphan Drug Designation (ODD) in the US for the treatment of hepatocellular carcinoma (HCC), the most common type of liver cancer. The US Food and Drug Administration (FDA) grants ODD to medicines intended for the treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the US. Liver cancer is the third leading cause of cancer death worldwide and for patients with unresectable or advanced disease, only 13% are alive five years after diagnosis.1-3 José Baselga, Executive Vice President, Oncology R&D, said: “Many patients with liver cancer are diagnosed and treated only after the disease is advanced, and there is an urgent need for new effective and tolerable treatments. We are eager to bring new potential options to these patients and look forward to the results of our ongoing Phase III HIMALAYA trial later this year.” The Phase III HIMALAYA trial is testing Imfinzi and the combination of Imfinzi plus tremelimumab in patients with unresectable, advanced HCC who have not been treated with prior systemic therapy and are not eligible for locoregional therapy (treatment localised to the liver). HIMALAYA is the first trial to test dual immune checkpoint blockade in the 1st-line advanced HCC setting. Imfinzi is not currently approved to treat HCC in any country, alone or in combination with tremelimumab. Hepatocellular carcinoma (HCC) Liver cancer is the third leading cause of cancer death and the sixth most commonly diagnosed cancer worldwide.1 HCC represents about 80% of all primary liver cancers.4 Approximately 700,000 people were diagnosed with HCC around the world in 2018, and an estimated 42,000 people were diagnosed in the US last year.1,2 Between 80-90% of all patients with HCC also have chronic liver disease, which is primarily caused by infection with the hepatitis B or C viruses.5,6 Chronic liver disease is associated with inflammation that, over time, results in immunosuppression and can lead to the development of HCC.7,8 The unique immune environment of liver cancer provides clear rationale for researching medicines that harness the power of the immune system to treat HCC.9 A critical unmet need exists for patients with HCC who face limited treatment options.10 More than half of patients are diagnosed at advanced stages of the disease, often when symptoms first appear.11,12 HIMALAYA HIMALAYA is a randomised, open-label, multicentre, global Phase III trial of Imfinzi monotherapy and the combination of Imfinzi and tremelimumab vs. the standard-of-care medicine sorafenib, a multi-kinase inhibitor, in patients with unresectable, advanced HCC who have not been treated with prior systemic therapy and are not eligible for locoregional therapy. The trial is being conducted in 189 centres across 16 countries including in the US, Canada, Europe, South America and Asia. The primary endpoint is overall survival and key secondary endpoints include objective response rate and progression-free survival. Imfinzi Imfinzi (durvalumab) is a human monoclonal antibody that binds to PD-L1 and blocks the interaction of PD-L1 with PD-1 and CD80, countering the tumour's immune-evading tactics and releasing the inhibition of immune responses. Imfinzi is approved in the curative-intent setting of unresectable, Stage III non-small cell lung cancer (NSCLC) after chemoradiation therapy in 54 countries, including the US, Japan, China and across the EU, based on the Phase III PACIFIC trial. Imfinzi is also approved for previously treated patients with advanced bladder cancer in 11 countries, including the US. Imfinzi is under Priority Review with FDA for the treatment of patients with previously untreated extensive-stage small cell lung cancer (SCLC) in combination with chemotherapy. A Prescription Drug User Fee Act date is set for the first quarter of 2020. As part of a broad development programme, Imfinzi is also being tested as a monotherapy and in combination with tremelimumab, an anti-CTLA4 monoclonal antibody and potential new medicine, as a treatment for patients with NSCLC, SCLC, bladder cancer, head and neck cancer, liver cancer, biliary tract cancer, cervical cancer and other solid tumours. Tremelimumab Tremelimumab is a human monoclonal antibody and potential new medicine that targets the activity of cytotoxic T-lymphocyte-associated protein 4 (CTLA-4). Tremelimumab blocks the activity of CTLA-4, contributing to T cell activation, priming the immune response to cancer and fostering cancer cell death. Tremelimumab is being tested in a clinical trial programme in combination with Imfinzi in NSCLC, SCLC, bladder cancer, head and neck cancer and liver cancer. AstraZeneca’s approach to Immuno-Oncology (IO) Immuno-oncology (IO) is a therapeutic approach designed to stimulate the body’s immune system to attack tumours. The Company’s IO portfolio is anchored by immunotherapies that have been designed to overcome anti-tumour immune suppression. AstraZeneca believes that IO-based therapies offer the potential for life-changing cancer treatments for the clear majority of patients. The Company is pursuing a comprehensive clinical-trial programme that includes Imfinzi as a monotherapy and in combination with tremelimumab in multiple tumour types, stages of disease, and lines of therapy, using the PD-L1 biomarker as a decision-making tool to define the best potential treatment path for a patient. In addition, the ability to combine the IO portfolio with radiation, chemotherapy, small targeted molecules from across AstraZeneca’s Oncology pipeline, and from research partners, may provide new treatment options across a broad range of tumours. AstraZeneca in Oncology AstraZeneca has a deep-rooted heritage in oncology and offers a quickly growing portfolio of new medicines that has the potential to transform patients' lives and the Company's future. With at least six new medicines to be launched between 2014 and 2020, and a broad pipeline of small molecules and biologics in development, the Company is committed to advance oncology as a key growth driver for AstraZeneca focused on lung, ovarian, breast and blood cancers. In addition to AstraZeneca's main capabilities, the Company is actively pursuing innovative partnerships and investments that accelerate the delivery of our strategy, as illustrated by the investment in Acerta Pharma in haematology. By harnessing the power of four scientific platforms – Immuno-Oncology, Tumour Drivers and Resistance, DNA Damage Response and Antibody Drug Conjugates – and by championing the development of personalised combinations, AstraZeneca has the vision to redefine cancer treatment and one day eliminate cancer as a cause of death. AstraZeneca AstraZeneca (LSE/STO/NYSE: AZN) is a global, science-led biopharmaceutical company that focuses on the discovery, development and commercialisation of prescription medicines, primarily for the treatment of diseases in three therapy areas - Oncology, Cardiovascular, Renal and Metabolism, and Respiratory. AstraZeneca operates in over 100 countries and its innovative medicines are used by millions of patients worldwide. Please visit astrazeneca.com and follow the Company on Twitter @AstraZeneca. Source: AstraZeneca Posted: January 2020
2020年1月20日----阿斯利康(AstraZeneca)公司的 Imfinzi ( durvalumab )和抖动单抗(一种抗 CTLA4抗体和潜在的新药)均已在美国获得用于治疗最常见的肝癌( HCC )的孤儿药物指定( ODD )。 美国食品药品监督管理局(Food and Drug Administration)( FDA )授予 ODD 用于治疗、诊断或预防影响美国不到20万人的罕见疾病或疾病的药物。 肝癌是全球第三大癌症死亡原因,对于无法切除或晚期疾病的患者来说,只有13%的人在确诊后五年内存活。1-3 肿瘤研究与开发执行副总裁 Jos é Basegga 表示:“许多肝癌患者只有在疾病进展后才被诊断和治疗,迫切需要新的有效和可容忍的治疗方法。我们渴望为这些患者带来新的潜在选择,并期待今年晚些时候我们正在进行的 III 期 HIMALAYA 试验的结果。” 第三期 HIMALAYA 试验正在对 Imfinzi 和 Imfinzi 加震颤联合疗法进行检测,这些患者是无法切除的晚期 HCC 患者,他们之前没有接受全身治疗,也没有资格接受局部治疗(治疗地点在肝脏)。HIMALAYA 是第一个在一线先进 HCC 设置中测试双重免疫检查点阻断的试验。 Imfinzi 目前未被批准单独或与震颤联合治疗任何国家的 HCC 。 肝癌. 肝癌是全球第三大癌症死亡原因和第六大最常见的癌症确诊。1肝癌约占所有原发性肝癌的80%。42018年全球约有70万人被诊断为 HCC ,去年美国约有4.2万人被确诊。1,2丙肝患者中80-90%也有慢性肝病,主要由乙肝或丙肝病毒感染引起。5,6慢性肝病与炎症有关,随着时间的推移,炎症会导致免疫抑制,并可能导致 HCC 的发展。7,8肝癌独特的免疫环境为研究利用免疫系统的力量治疗肝癌的药物提供了明确的依据。对于面临有限治疗选择的 HCC 患者,存在着严重的未得到满足的需求。超过一半的病人在疾病的晚期阶段被诊断出来,通常是在症状首次出现时。11,12 HIMALAYA HIMALAYA 是 Imfinzi 单药治疗的随机、开放标签、多中心、全球 III 期试验,与 Imfinzi 和抖动联合应用。标准的护理药物索拉非尼,多激酶抑制剂,适用于不能切除的,先进的肝癌患者谁没有事先全身治疗,没有资格进行局部治疗。该试验正在美国、加拿大、欧洲、南美和亚洲等16个国家的189个中心进行。主要终点是整体生存,关键的次要终点包括客观反应率和无进展生存。 Imfinzi Imfinzi ( durvalumab )是一种人用单克隆抗体,与 PD-L1和 CD80结合,阻断 PD-L1与 PD-1和 CD80的相互作用,对抗肿瘤的免疫逃避策略,释放免疫应答的抑制作用。 Imfinzi 是根据 III 期 PACIFIC 试验,在包括美国、日本、中国和整个欧盟在内的54个国家的化学放射治疗后,在不可切除的 III 期非小细胞肺癌( NSCLC )的治疗意图设定中获得批准的。Imfinzi 还被批准用于包括美国在内的11个国家先前治疗的晚期膀胱癌患者。 Imfinzi 正在接受 FDA 的优先审查,以治疗以前未治疗的大细胞肺癌( SCLC )合并化疗的患者。处方药用户费用法案的日期定为2020年第一季度。 作为一项广泛发展方案的一部分, Imfinzi 也正在作为单一疗法进行试验,并与一种抗 CTLA4单克隆抗体和潜在的新药木乃伊联合进行试验,作为治疗非小细胞非小细胞肺癌(NSCLC)、小细胞肺癌、膀胱癌、头颈癌、肝癌、胆道癌、宫颈癌和其他实体肿瘤的药物。 Tremelimumab Tremelimumab 是一种针对细胞毒性 T 淋巴细胞相关蛋白4( CTLA-4)活性的单克隆抗体和潜在新药。Tremelimumab 阻断 CTLA-4的活性,促进 T 细胞活化,激发肿瘤免疫应答,促进癌细胞死亡。Tremelimumab 正在与 Imfinzi 联合在非小细胞肺癌(NSCLC)、 SCLC 、膀胱癌、头颈癌和肝癌进行临床试验。 阿斯利康(AstraZeneca)治疗免疫肿瘤的方法( IO ) 免疫肿瘤学( IO )是一种旨在刺激机体免疫系统攻击肿瘤的治疗方法。该公司的 IO 产品组合以免疫疗法为基础,旨在克服抗肿瘤免疫抑制。阿斯利康(AstraZeneca)认为,基于 IO 的治疗为绝大多数患者提供了改变生命的癌症治疗的潜力。 本公司正在进行一项全面的临床试验计划,其中包括以 Imfinzi 作为单一疗法,并结合多种肿瘤类型、疾病阶段和治疗方案中的震颤,使用 PD-L1生物标志物作为决策工具,为患者确定最佳的潜在治疗路径。此外, IO 组合与辐射、化疗、来自阿斯利康(AstraZeneca)肿瘤管道的小目标分子以及来自研究合作伙伴的能力,可能在广泛的肿瘤范围内提供新的治疗选择。 阿斯利康(AstraZeneca)肿瘤学 阿斯利康(AstraZeneca)在肿瘤领域有着深厚的传统,并提供了快速增长的新药组合,有可能改变患者的生活和公司的未来。2014年至2020年期间,公司将推出至少六种新药,并开发一大批小分子和生物制剂,公司致力于推进肿瘤学作为阿斯利康(AstraZeneca)专注于肺癌、卵巢癌、乳腺癌和血癌的关键增长动力。除阿斯利康(AstraZeneca)的主要能力外,本公司正积极寻求创新的合作伙伴关系和投资,以加速我们战略的实施,如在血液学方面对 Aceta Pharma 的投资所示。 通过利用免疫肿瘤学、肿瘤驱动和抵抗、 DNA 损伤反应和抗体药物共轭四个科学平台的力量,并通过支持个性化组合的开发,阿斯利康(AstraZeneca)有重新定义癌症治疗的愿景,有一天消除癌症作为死亡原因。 阿斯利康(AstraZeneca)公司 阿斯利康(AstraZeneca)( LSE / STO / NYSE : AZN )是一家全球性的、以科学为主导的生物制药公司,专注于处方药的发现、开发和商业化,主要用于治疗肿瘤、心血管、肾代谢和呼吸三个治疗领域的疾病。阿斯利康(AstraZeneca)在100多个国家开展业务,其创新药物被全球数百万患者使用。请访问阿斯利康。在 Twitter @ AstraZeneca 上跟踪公司。 资料来源:阿斯利康(AstraZeneca) Posted :2020年1月

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