FDA委员会支持批准Vanda制药昼夜节律紊乱药物tasimelteon

Vanda制药11月15日宣布，FDA外周和中枢神经系统药物顾问委员会（PCNSDAC）以压倒性票数，建议批准tasimelteon（ 他司美琼，拟用商品名HETLIOZ），用于完全失明患者，治疗非24小时睡醒障碍（非-24，Non-24）。

该委员会认为，相关临床研究已证实了tasimelteon治疗Non-24的疗效，同时具有良好的安全性。

此前，FDA已授予tasimelteon新药申请（NDA）优先审查资格，同时指定该药的处方药用户收费法（PDUFA）目标日期为2014年1月31日。

目前，还没有药物获批用于治疗Non-24。如果获批，tasimelteon有望解决这一远未满足的医疗需求。

关于Non-24：

Non-24是一种严重、罕见、慢性昼夜节律紊乱，其特点是无法将人体生物钟与24小时昼夜节律（24-hour day-night cycle）同步。Non-24影响着大多数全盲（totally blind）个体，在美国约有6.5万-9.5万患者。

英文原文：FDA panel backs Vanda's circadian rhythm drug

(Reuters) - An advisory panel to the Food and Drug Administration said on Thursday that an experimental drug designed to help regulate the internal body clocks of blind patients appears safe and effective.

The drug, tasimelteon, is designed to treat Non-24-Hour Sleep-Wake Disorder, or Non-24, a condition that is most commonly found in the totally blind and can cause disrupted nighttime sleep patterns and excessive daytime sleepiness.

If approved it would be marketed under the proposed brand name Hetlioz.

The FDA is due to make a decision on whether to approve the drug by January 31, 2014. The agency is not bound to follow the advice of its advisory panels but typically does so.

Non-24 is a rare disorder that affects between 65,000 and 95,000 people, according to Vanda. It occurs almost exclusively in people who are deprived of light, which is needed to synchronize the body's internal clock.

Tasimelteon affects melatonin, a hormone produced by the body's pineal gland that plays a key role in regulating the body's master circadian clock. When taken before bed it resets the circadian clock by replacing the normal resetting triggered by light.

In two clinical trials, Washington-based Vanda said the drug offered improvements to patients across a variety of sleep and wake parameters including measures of total sleep time and daytime nap duration.

The most common side effects included back pain, vivid dreams, diarrhea, dry mouth, headache, sleepiness and upper respiratory tract infections.

The drug has been given Orphan Drug status by the FDA. Orphan drugs are designed to treat diseases that affect fewer than 200,000 people. If approved, the drug would receive seven years of U.S. market exclusivity.

In January the company said tasimelteon failed to improve symptoms in a clinical trial of patients with major depressive disorder.

Vanda's only marketed product is its schizophrenia drug Fanapt, which is commercialized in the United States and Canada by the Swiss drugmaker Novartis AG under a licensing agreement with Vanda.