FDA批准口服MS药物特立氟胺

美国食品药品管理局(FDA)已批准复发性多发性硬化症(MS)最新口服药物特立氟胺(Aubagio)，但附带了一项增加胎儿风险的强烈警告。

该药物由赛诺菲安万特所属健赞公司生产，是FDA批准的第二个用于治疗MS的口服片剂药物。诺华公司的芬戈莫德(Gilenya)已于2010年获得批准。

特立氟胺是来氟米特的活性代谢物。来氟米特是一种低分子量合成药物，于1998年获得FDA批准用于治疗类风湿性关节炎。

FDA在书面声明中指出，特立氟胺最常见副作用为腹泻、肝功检查异常、恶心和脱发。该药物将附带一项有关肝脏毒性风险的黑框警告，建议医生在治疗开始和治疗期间对患者进行肝功检查。

警告还强调了特立氟胺增加出生缺陷的可能性。动物研究表明，该药物属于致畸物，因此被列为妊娠期用药分类的X类，即意味着育龄女性开始接受特立氟胺治疗前的孕检结果必须为阴性，且在治疗期间必须采取有效的避孕措施。

据FDA，在临床试验中服用特立氟胺患者复发率比服用安慰剂者降低30%。2010年底首次报告了特立氟胺多发性硬化症口服(TEMSO)试验的上述结果。

FDA药物评价和研究中心的Russell Katz博士在声明中称：“MS可损害运动、感觉和思维，因此向患者提供不同用药选择非常重要。”

美国多发性硬化症学会首席研究官Timothy Coetzee博士在健赞公司的声明中说：“看到MS患者拥有一种新的口服用药选择，我们倍受鼓舞。”

美国MS患者超过35万人，而全球约250万。症状通常出现在20~40岁，女性患者数量是男性的2倍，多数患者处于该病复发期。

服用特立氟胺的患者将会收到一份有关该药物潜在危害的用药指南。

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By: ALICIA AULT, Clinical Neurology News Digital Network

The Food and Drug Administration has approved the latest oral therapy for relapsing multiple sclerosis, teriflunomide (Aubagio), but it will carry a strong warning that it increases the risk of fetal harm.

The drug, made by the Genzyme division of Sanofi Aventis, is the second oral tablet approved by the agency for MS. Novartis’ fingolimod (Gilenya) was approved in 2010.

Teriflunomide is the active metabolite of leflunomide, a synthetic, low-molecular-weight drug that was approved by the FDA in 1998 for the treatment of rheumatoid arthritis.

The most common side effects with teriflunomide were diarrhea, abnormal liver tests, nausea, and hair loss, the agency noted in a written statement. The drug will carry a boxed warning on the risk of liver toxicity. Clinicians are being advised to conduct liver function tests before initiating treatment and during therapy.

The warning also will highlight teriflunomide’s increased potential for causing birth defects. Animal studies suggested that the drug is a teratogen. Thus, it is being placed in pregnancy category X, which means that women of childbearing age must have a negative pregnancy test before starting teriflunomide therapy, and also must use effective birth control during treatment.

According to the FDA, the relapse rate for patients taking teriflunomide in trials was 30% lower than for those taking a placebo. Those results were first reported in late 2010 in the Teriflunomide Multiple Sclerosis Oral (TEMSO) trial.

"Multiple sclerosis can impair movement, sensation, and thinking, so it is important to have a variety of treatment options available to patients," Dr. Russell Katz, director of the Division of Neurology Products in the FDA’s Center for Drug Evaluation and Research, said in a written statement.

"We are greatly encouraged to see a new oral therapeutic option become available to people living with MS," Dr. Timothy Coetzee, chief research officer at the National Multiple Sclerosis Society, said in a statement provided by Genzyme.

The neurological disorder is thought to affect more than 350,000 Americans, and 2.5 million people worldwide. Symptoms usually begin between ages 20 and 40, and women are affected twice as often as are men. The relapsing phase of the disease is the most common.

Teriflunomide patients will receive a medication guide that outlines the drug’s potential harms.