2009年9月，阿罗斯治疗公司的抗肿瘤新药—普拉曲沙注射液(商品名：Folotvn)获美国FDA批准上市，用于单一用药治疗复发性和顽固性周围T细胞淋巴瘤。剂量规格：普拉曲沙20mg／mL。辅料：氯化钠0．6％(280～300mOsm)。普拉曲沙注射液是按美国FDA加速审批程序批准的，供复发或对其它化疗药物治疗无效的患者使用。

普拉曲沙注射液获准上市是基于对115例复发性和顽固性T细胞淋巴瘤一国际多中心开放式、单组的临床研究结果，患者在7周疗程中一周1次静脉注射普拉曲沙30mg／m2直至出现疾病恶化或患者不能接受的药物毒性。此外，患者每8～10周肌肉注射维生素B12-1mg和一日1次口服叶酸1．0～1．25mg。按临床研究报告判断其中109例有效。

按国际工作组1999年发表的淋巴瘤疗效评价标准(IWC)主要疗效标准是总有效率(完全有效、未确定的完全有效率和部分有效率)。第二关键的疗效标准是疗效的持续时间。临床研究显示，109例中29例(27％)周围T细胞淋巴瘤患者的肿瘤缩小，疗效平均持续时间为287日(9．4个月)。109例中13例患者持续有效14周。

普拉曲沙最常见的不良反应是血小板减少症(33％)。黏膜炎(对唇、口腔和消化道黏膜的刺激，2l％)，发热，恶心和乏力。普拉曲沙对胎儿有害，欲怀孕和孕妇不应服用。患者服用普拉曲沙期间应加服叶酸盐和维生素B12以减少该药对黏膜的刺激。

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 have relapsed, or have not responded well to other forms of chemotherapy.
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.