2019年度新药研究报告（中英文版，112页）

行业报告下载 2020年06月18日 06:52 管理员

2019年夏，FDA加速批准了另一款新型ADC：polatuzumab vedotin（Polivy；基因泰克）。该药物由抗CD79b单克隆抗体与 MMAE组成，两者通过蛋白酶可裂解肽连接子偶联。该药适用于与苯达莫司汀+利妥昔单抗联合用药治疗既往至少接受过 2种治疗的成人复发或难治性弥漫性大B细胞淋巴瘤患者。该药在美国已获得孤儿药认定。Polatuzumab vedotin是首款 CD79b蛋白（在大多数B细胞中均有特异性表达）靶向药物（图 8）。该药物可与CD79b结合，并通过递送抗肿瘤药物MMAE破坏这些B细胞，同时对正常细胞的毒性作用极小。Polatuzumab vedotin获批后不久即在美国上市。去年，Polivy还获得了欧盟的有条件批准，该药物在欧盟获得PRIME称号和孤儿药资格，同时还被认定为高级治疗药物产品。骨髓纤维化被视为一种慢性白血病，是一种不常见的血液系统恶性肿瘤，患者的骨髓会逐渐被纤维瘢痕组织替代。骨髓纤维化可能是原发性疾病，也可能继发于自身免疫性疾病或其他骨髓肿瘤。约50%的原发性骨髓纤维化患者携带JAK2基因突变；骨髓纤维化的首个特异性治疗药物是JAK2抑制剂芦可替尼，已于2011年上市。去年，具有双重作用机制的JAK2/ FLT3抑制剂fedratinib（Inrebic；Celgene）在美国获批上市，增加了该罕见病的治疗选择。Fedratinib适用于治疗伴有中危-2 或高危的原发性或继发性（真性红细胞增多症后或原发性血小板增多症后）成人骨髓纤维化患者。该药物因这个适应症在美国被认定为孤儿药。

In another exciting development last year, the EC in November granted conditional approval for Merck & Co.’s Ebola Zaire Vaccine (Ervebo), the first Ebola vaccine to be approved by any regulatory agency following evaluation in large clinical trials; the FDA followed suit a month later. The vaccine was discovered at Canada’s National Microbiology Laboratory with funding from the U.S. government’s Biomedical Advanced Research and Development Authority (BARDA), and was developed through a public– private partnership with NewLink Genetics and Merck. It has been tested extensively in the last two major Ebola outbreaks in West Africa (2013- 2016) and the DRC (2018-2019). Ervebo is indicated for active immunization of individuals age 18 years or older, to protect against Ebola virus disease caused by Zaire Ebola virus. The approval allows Merck to initiate manufacturing in Germany of licensed doses, which are expected to be available from Q3 2020. Merck is also working with the WHO, the U.S. government and Gavi, the Vaccine Alliance, to ensure uninterrupted access of its vaccine in support of international response efforts in the ongoing outbreak in the DRC. Two other Ebola vaccines had previously been approved, on the basis of phase I and phase II testing, albeit for emergency use only. Russia’s Gamaleya Federal Research Centre for Epidemiology and Microbiology developed Gam Evac Combi, a combined vector vaccine against Ebola fever that was authorized for use in medical practice within the territory of the Russian Federation in 2015. In 2017, the China FDA approved Ad5-EBOV, a vaccine codeveloped by the Institute of Biomedical Engineering, Academy of Military Medical Sciences and Tianjin CanSino Biotechnology.