药品信息: 美专家支持批准Folotyn(pralatrexate)用于外周T细胞淋巴瘤 总部设在科罗拉多州威斯敏斯特的制药商Allos Therapeutics Inc。(ALTH)周五宣布，美国食品与药物管理局(FDA)经由快速审核程序，批准该公司的实验性药物Folotyn用于治疗致死率最高的侵入性白血球癌症的药物。受到这条利好消息推动，周五盘前交易中Allos Therapeutics股价一度上涨了8%。 在Allos公司的上述药物获得批准之前，FDA旗下顾问小组在本月初报告称，Allos Therapeutics公司的临床试验数据非常有说服力，清楚的表明Folotyn (pralatrexate)药物对于复发性或者症状难以控制的外周T细胞淋巴瘤(peripheral T-cell lymphoma，PTCL)患者有帮助。 Allos表示，Folotyn是FDA批准的第一种、而且是唯一得到该机构批准的一种外周T细胞淋巴瘤药物。Allos预计将从10月开始向美国患者提供这种药物。 这家公司表示，FDA批准Folotyn药物是基于该药物的总体有效比例数据，并未考虑这种药物的临床有效性——例如患者继续生存并且症状不再恶化、整体生存率提高等等。 Allos公司表示，在Folotyn药物按照快速审批程序获得批准之后，该公司已经同意继续进行临床试验，以进一步验证和描述该药物的临床效果。 FDA Approves Folotyn FDA Approves Folotyn (pralatrexate) for Treatment of Peripheral T-cell Lymphoma SILVER SPRING, Md., Sept. 25 /PRNewswire-USNewswire/ -- The U.S. Food and Drug Administration has approved Folotyn (pralatrexate), the first treatment for a form of cancer known as Peripheral T-cell Lymphoma (PTCL), an often aggressive type of non-Hodgkin's lymphoma. Folotyn was approved under the FDA's accelerated approval process, which allows earlier approval of drugs that meet unmet medical needs. It is approved for patients who haverelapsed,orhavenotrespondedwelltootherformsofchemotherapy. Lymphoma is a cancer of the lymphatic system, which is part of the immune system. There are many types of lymphoma: one type is called Hodgkin's disease, and the rest are called non-Hodgkin's lymphomas. PTCL involves a type of white blood cell called T-cells. It is a relatively rare disease, occurring in less than 9,500 patients each year in the United States. "Folotyn's approval demonstrates FDA's commitment to the rapid approval of drugs for rare and uncommon diseases," said Richard Pazdur, M.D., director of the Office of Oncology Drug Products in the FDA's Center for Drug Evaluation and Research. When studying a new drug, it can take time to learn whether a drug actually provides real improvement for patients - such as living longer or feeling better. This real improvement is known as a "clinical outcome." In 1992 FDA instituted accelerated approvals which allow earlier approval of drugs based on a surrogate endpoint, a laboratory measurement or physical sign that can serve as an indirect or substitute measurement for clinical outcomes. In the case of Folotyn, this meant the FDA approved the drug based on evidence that it reduces tumor size, because tumor shrinkage is considered reasonably likely to predict a clinical benefit such as extending the survival of cancer patients. Tumor shrinkage was seen on imaging scans in one study. Of 109 patients with PTCL in the trial, 27% had reduction in tumor size. To speed the drug's availability, Folotyn was granted priority review, ensuring a review within six months rather than 10 months for a standard review. The drug was also designated as an orphan drug, which provides a variety of financial incentives to manufacturers that develop drugs for a small number of patients with a rare disorder. The most common adverse reactions seen with Folotyn were irritation or sores of the mucous membranes such as the lips, the mouth, and the digestive tract, low platelet cell counts, low white blood cell counts, fever, nausea, and fatigue. Folotyn can harm a fetus. Women should avoid becoming pregnant while being treated with this drug and pregnant women should be informed of the potential risk. Patients treated with Folotyn should take folate and vitamin B12 supplements to reduce mucous membrane irritation. Folotyn is manufactured by Allos Therapeutics Inc. of Westminster, Colo. As a condition of accelerated approval, Allos will conduct studies to confirm that tumor shrinkage actually does predict that patients will live longer.