2014年12月5日讯 /生物谷BIOON/ --所谓行百里者半九十，医药公司在经历了漫长的时间开发一种新药后，最关键的也是最头痛的一步就是将药物报送FDA审核。这一过程中，FDA往往会要求公司补交各种数据，甚至有时候，经过漫长的审核期之后最终被驳回的药物也不在少数。然而，今天安进公司却让所有人大跌眼镜。FDA决定批准安进公司开发的治疗白血病的新型免疫疗法blinatumomab上市。这一决定耗时仅2个月，比其预计的审核期足足早了5个月之久！
Blinatumomab是一种基于安进公司双特异性T细胞衔接系统（bispecific T cell engager, BiTE）开发而来的免疫疗法。这种疗法能够通过将肿瘤细胞上的CD19蛋白--一种肿瘤细胞通用抗原--呈递给CD3蛋白，进而激活免疫系统识别和杀灭肿瘤细胞（CD3是由免疫系统中的T细胞特异表达的）。Blinatumomab是安进公司在2012年花费12亿美元在Micromet收购案中获得的。
目前FDA批准这一药物用于治疗包括费城染色体阴性B细胞急性白血病在内的急性白血病。安进公司仍在推进利用Blinatumomab治疗慢性白血病等疾病的研究。这也成为了安进公司研发部门取得的又一重大成果。公司目前还有心血管药物evolocumab、肿瘤疫苗T-Vec以及治疗牛皮癣药物brodalumab处于后期临床开发中，预计将于明年报送FDA审批。
Blinatumomab也由此成为了世界上第一种获得FDA批准的CD19药物。此次FDA的提前批准决定无疑是对安进公司的一个巨大利好消息。因为诺华公司、Juno公司和Kite公司都正在加紧开发自己的CD19类肿瘤药物。这五个月的时间将使安进公司有更充裕的时间占领市场。
详细英文报道：
The FDA approved Amgen's ($AMGN) new leukemia treatment more than 5 months ahead of schedule, green-lighting the first contender among a new class of immunotherapies that promise to change the standard of care in blood cancer.
The drug, blinatumomab, is an antibody developed through Amgen's bispecific T cell engager (BiTE) system, designed to direct the body's disease-fighting immune cells to attack cancerous growth. The agency's approval, handed down just two months and change after Amgen's filing, covers Philadelphia chromosome-negative precursor B-cell acute lymphoblastic leukemia (ALL), a rare form of the cancer. The company plans to market its therapy as Blincyto.
Amgen's treatment works by connecting CD19, a protein commonly found on cancer cells, to CD3, which is expressed by the immune system's T cells. By drawing the two proteins together, blinatumomab can redirect the body's natural defenses to home in on malignancies. And, in Phase II data submitted to support its approval, the drug met its primary endpoint of significantly improving rates of complete remission among ALL patients.
Blinatumomab's success marks the first approval for any CD19-targeting agent, a class that includes promising immunotherapies from Novartis ($NVS), Juno Therapeutics, Kite Pharma ($KITE) and others. And the FDA's move, coming well ahead of blinatumomab's May 19 decision deadline, affirms oncology chief Richard Pazdur's willingness to hurry along new treatments that could change the standard of care, cause for optimism among developers of similar drugs.
"Immunotherapies, especially Blincyto with its unique mechanism of action, are particularly promising for patients with leukemia," Pazdur said in a statement. "Recognizing the potential of this novel therapy, the FDA worked proactively with the sponsor under our breakthrough therapy designation program to facilitate the approval of this novel agent."
Amgen has moved quickly with blinatumomab since picking it up in a $1.2 billion buyout of Micromet in 2012, and the immunotherapy is also in development as a treatment for chronic lymphocytic leukemia, B-cell lymphoma and other blood and marrow cancers. Beyond blinatumomab, the company is investigating a slew of new BiTE candidates, and Amgen believes its technology has applications in a wide range of cancers.
The drug is a major pillar of Amgen's late-stage pipeline, standing alongside the much-heralded cardio treatment evolocumab, the cancer vaccine T-Vec and the psoriasis drug brodalumab, all of which could be up for FDA approval next year.