3月19日FDA批准加拿大Paladin公司Impavido (miltefosine)用于治疗12岁以上患者的内脏利什曼病、皮肤利什曼病和黏膜利士曼病。Impavido曾被FDA授予快速通道、优先审评和孤儿药资格，是FDA批准的首个表皮和黏膜利什曼病药物。按照2007年FDA颁布的修正法案，Paladin公司将凭借此项批准获得旨在鼓励开发某些热带病药物的“热带病优先审查凭证”。
4项临床研究评估了患者接受Impavido（n=547）、对照药或安慰剂（n=183）的疗效和安全性。结果显示，Impavido治疗内脏、皮肤和黏膜利士曼病时安全有效。最常见不良反应包括恶心、呕吐、腹泻、头痛、食欲减退、眩晕、腹痛、瘙痒、昏睡和转氨酶水平升高。Impavido药品标签中附有黑框警告内容，警示孕妇禁服。
利什曼病是由利什曼原虫感染引起，经白蛉叮咬传播的一种热带和亚热带寄生虫传染人畜共患病，以皮肤或内脏器官的严重损害、坏死为特征。
FDA approves Impavido to treat tropical disease leishmaniasis
The U.S. Food and Drug Administration today approved Impavido (miltefosine) to treat a tropical disease called leishmaniasis.
Leishmaniasis is a disease caused by Leishmania, a parasite which is transmitted to humans through sand fly bites. The disease occurs primarily in people who live in the tropics and subtropics. Most U.S. patients acquire leishmaniasis overseas.
Impavido is an oral medicine approved to treat the three main types of leishmaniasis: visceral leishmaniasis (affects internal organs), cutaneous leishmaniasis (affects the skin) and mucosal leishmaniasis (affects the nose and throat). It is intended for patients 12 years of age and older. Impavido is the first FDA-approved drug to treat cutaneous or mucosal leishmaniasis.
“Today’s approval demonstrates the FDA’s commitment to making available therapeutic options to treat tropical diseases,” said Edward Cox, M.D., director of the Office of Antimicrobial Products in the FDA’s Center for Drug eva1uation and Research.
The FDA granted Impavido fast track designation, priority review, and orphan product designation. These designations were granted because the drug demonstrated the potential to fill an unmet medical need in a serious disease or condition, the potential to be a significant improvement in safety or effectiveness in the treatment of a serious disease or condition, and is intended to treat a rare disease, respectively. With this approval, Impavido’s manufacturer, Paladin Therapeutics, is awarded a Tropical Disease Priority Review Voucher under a provision included in the Food and Drug Administration Amendments Act of 2007 that aims to encourage development of new drugs and biological products for the prevention and treatment of certain tropical diseases.
Impavido’s safety and efficacy were eva1uated in four clinical trials. A total of 547 patients received Impavido and 183 patients received either a comparator drug or a placebo. Results from these trials demonstrated that Impavido is safe and effective in treating visceral, cutaneous and mucosal leishmaniasis.
The labeling for Impavido includes a boxed warning to alert patients and health care professionals that the drug can cause fetal harm and therefore should not be given to pregnant women. Health care professionals should advise women to use effective contraception during and for five months after Impavido therapy.
The most common side effects identified in clinical trials were nausea, vomiting, diarrhea, headache, decreased appetite, dizziness, abdominal pain, itching, drowsiness and elevated levels of liver enzymes (transaminases) and creatinine.
Paladin Therapeutics is based in Montreal, Canada.
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