Manufacturer: Allos Therapeutics Pharmacological Class: Antineoplastic (folate analogue inhibitor) Active Ingredient(s): Pralatrexate 20mg/mL; soln for IV inj; preservative-free. Indication(s): Relapsed or refractory peripheral T-cell lymphoma. Pharmacology: Pralatrexate is a folate analogue metabolic inhibitor that competitively inhibits dihydrofolate reductase. It is also a competitive inhibitor of polyglutamylation by the enzyme folylpolyglutamyl synthetase. This inhibition results in the depletion of thymidine and other biological molecules. Clinical Trials: This product was approved based on the overall response rate observed in a clinical trial. A clinical benefit, such as improvement in progression-free survival or overall survival, hasnotbeendemonstrated. The safety and efficacy of pralatrexate was examined in an open-label, single-arm, multicenter trial that enrolled 115 patients with relapsed or refractory peripheral T-cell lymphoma. Of these, 111 patients were treated with the study drug (dosed at 30mg/m2 once weekly by IV push over 3–5minutes for 6 weeks in 7-week cycles until disease progression or unacceptable toxicity developed). The primary efficacy endpoint was overall responserateasassessedbyInternational Workshop Criteria. A secondary endpoint was the duration of response. Duration of response was measured from the first day of documented response to disease progression or death. In 109 evaluable patients, the response rate (complete response, complete response unconfirmed, and partial response) was 27%. Of the responders, 66% responded within cycle 1. The median time to first response was 45 days, and the median duration of response was 9.4 months in the responders. Legal Classification: Rx Adults: Prior to administration: mucositis should be ≤Grade 1, platelets should be ≥100,000/µL for first dose and ≥50,000/µL for subsequent doses, absolute neutrophil count should be ≥1000/µL. Give by IV push over 3–5min. 30mg/m2 once weekly for 6 weeks in 7-week cycles; may reduce to 20mg/m2 or interrupt treatment to manage toxicity (see literature for adjustment criteria). Continue until disease progression or unacceptable toxicity develops. Supplement with vitamin B12 (1mg IM every 8–10 weeks, starting within 10weeks before first Folotyn dose) and folic acid (1–1.25mg orally daily, beginning 10 days before starting Folotyn and for 30 days after stopping). Children: Not recommended. Precaution(s): Adjust dose to manage toxicities (eg,hematological,mucositis,hepatic impairment); see literature. Monitor CBC and for mucositis weekly. Monitor serum chemistry, renal and hepatic function before the 1st and 4th dose per cycle. Renal or hepatic impairment. Pregnancy (Cat.D) (may cause fetal harm), nursing mothers: not recommended. 适应症：复发性外周T细胞淋巴瘤（Relapsed peripheral T-cell lymphoma） 生产商：Allos Therapeutics 批准日期：9月24日 Folotyn是第一只也是唯一一只针对复发性外周T细胞淋巴瘤的药物，该病具有高复发和预后很差的特点，是血癌中相当罕见的一种，该药被定为“孤儿药”，通过快速审批通道获得批准。