5月5日（当地），美国FDA宣布批准了Radicava（edaravone）用于治疗肌萎缩侧索硬化（ALS），这也是美国FDA22年来批准的首款ALS新疗法。这种药物必须静脉输注，每年花费约145524美元。而另一个药物riluole是在1995年批准的。

ALS是一种罕见的未知病因的神经退行性疾病，该病患者身体中动用肌肉所必需的运动神经元被损坏，导致了像舌头、脖子和四肢之类里的所有肌肉的瘫痪。在美国，大约有12000-15000名ALS患者。这种疾病具有渐进的特性，患者的症状会不断恶化，大部分患者会在症状产生后的3到5年内会由于呼吸衰竭而死亡。

原文如下（以下转自公众号渐冻人关爱在线）：

FDA News Release

FDA approves drug to treat ALS

For Immediate Release

May 5, 2017

Release

The U.S. Food and Drug Administration today approved Radicava (edaravone) to treat patients with amyotrophic lateral sclerosis (ALS), commonly referred to as Lou Gehrig’s disease.

“After learning about the use of edaravone to treat ALS in Japan, we rapidly engaged with the drug developer about filing a marketing application in the United States,” said Eric Bastings, M.D., deputy director of the Division of Neurology Products in the FDA’s Center for Drug Evaluation and Research. “This is the first new treatment approved by the FDA for ALS in many years, and we are pleased that people with ALS will now have an additional option.”

“听说edaravone在日本上市治疗ALS后，我们迅速与药物的开发者接洽，讨论递交美国上市申请事宜，”FDA药物评估与研究中心神经学产品部副主任Eric Bastings博士说道：“这是多年以来美国FDA批准的首个ALS新疗法，我们很高兴能让ALS患者有另外一个选择。”

ALS is a rare disease that attacks and kills the nerve cells that control voluntary muscles. Voluntary muscles produce movements such as chewing, walking, breathing and talking. The nerves lose the ability to activate specific muscles, which causes the muscles to become weak and leads to paralysis. ALS is progressive, meaning it gets worse over time. The Centers for Disease Control and Prevention estimates that approximately 12,000-15,000 Americans have ALS. Most people with ALS die from respiratory failure, usually within three to five years from when the symptoms first appear. 

Radicava is an intravenous infusion given by a health care professional. It is administered with an initial treatment cycle of daily dosing for 14 days, followed by a 14-day drug-free period. Subsequent treatment cycles consist of dosing on 10 of 14 days, followed by 14 days drug-free.

依达拉奉需要静脉注射，初始周期为注射14天，观察14天（无注射），之后的14天中注射10天，然后再观察14天不用药。

The efficacy of edaravone for the treatment of ALS was demonstrated in a six-month clinical trial conducted in Japan. In the trial, 137 participants were randomized to receive edaravone or placebo. At Week 24, individuals receiving edaravone declined less on a clinical assessment of daily functioning compared to those receiving a placebo.

依达拉奉治疗ALS的疗效在日本进行了为期6个月的临床试验。 在试验中，137名参与者随机接受依达拉奉或安慰剂治疗。 在第24周的观察中，与接受安慰剂的人相比，接受依达拉奉的患者的日常功能的临床评估指标下降较少。

The most common adverse reactions reported by clinical trial participants receiving edaravone were bruising (contusion) and gait disturbance.

接受依达拉奉的临床试验参与者报告的最常见的不良反应是瘀伤（挫伤）和步态障碍。

Radicava is also associated with serious risks that require immediate medical care, such as hives, swelling, or shortness of breath, and allergic reactions to sodium bisulfite, an ingredient in the drug. Sodium bisulfite may cause anaphylactic symptoms that can be life-threatening in people with sulfite sensitivity.

依达拉奉也可能存在严重的需要紧急处理的不良反应，如荨麻疹，水肿或呼吸短促，以及药物中亚硫酸氢钠的过敏反应。 亚硫酸氢钠可能引起过敏性症状，对亚硫酸盐敏感的人来说可能危及生命。

The FDA granted this drug orphan drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases.

美国食品和药物管理局批准了这种药物孤儿药物名称，此举为协助和鼓励开发稀有疾病药物提供了激励。

The FDA granted approval of Radicava to Mitsubishi Tanabe Pharma America, Inc.

多说几句： 依达拉奉在我国已有10余年的应用，之前在急性脑血管病的应用中显示出了安全性，这是一种神经保护剂，能强效清理自由基，使神经免于氧化应激（oxidative stress）和神经元凋亡（neuronal apoptosis），因此有望为ALS患者带来益处。