近日：美国食品和药品监管局今天批准Radicava(edaravone 中文暂译名 依达拉奉)治疗有肌萎缩性侧索硬化症[amyotrophic lateral sclerosis (ALS)]，常被称为Lou Gehrig’s病患者。
FDA药品评价和研究中心中神经学产品部副主任Eric Bastings，M.D.说：“在了解edaravone在日本治疗ALS使用后，我们迅速地与药物开发者接洽关于在美国提交一个上市申请文件，”这是在多年来被FDA对ALS批准的第一个新治疗，而我们很高兴有ALS人们现将有一个另外选择。”
Radicava是一种被一位卫生保健专业人员静脉输注给予。给予有一个初始治疗疗程每天给药共14天，接着为一个14-天无药阶段。随后治疗疗程由14天的10给药，接着14天无药。
在日本进行的一个6-个月临床试验证实edaravone为ALS治疗的疗效。在试验中，137例参加者被随机至接受edaravone或安慰剂。在周24，接受edaravone个体与接受一个安慰剂参加者比较对一个每天功能的临床评估下降较少。
批准日期: 2017年5月 公司: Mitsubishi Tanabe Pharma
RADICAVA(依达拉奉[edaravone])注射液，为静脉使用
美国初次批准：2017
作用机制
不知道RADICAVA在有ALS患者中发挥其治疗作用的机制
适应证和用途
RADICAVA是适用为肌萎缩性侧索硬化症[amyotrophic lateral sclerosis(ALS)]的治疗
剂量和给药方法
推荐剂量是60mg作为一个静脉输注历时60分钟给药如下：
● 初始治疗疗程：每天给药共14天接着14-天无药阶段
● 随后治疗疗程：14天阶段的每天给药共10天，接着14-天无药阶段。
剂型和规格
注射液：30mg/100mL在一个一次性-剂量聚丙烯袋
禁忌证
有对edaravone或在RADICAVA 中任何无活性成分超敏性史患者
警告和注意事项
●超敏性反应：建议患者寻求立即医疗护理
● 亚硫酸盐过敏反应：RADICAVA含亚硫酸氢钠，它可能致过敏性型反应。
不良反应
最常见不良反应(至少10%和大于安慰剂)是挫伤，步态不稳，和头痛
特殊人群中使用
● 妊娠：根据动物数据，可能致胎儿危害。
包装规格
RADICAVA注射液是被供应为一个30mg/100mL(0.3mg/mL)清晰，无色，无菌溶液为静脉输注在一剂量聚丙烯袋，每个用聚乙烯醇(PVA)包住二次包装含一个氧吸收剂和氧指示剂，它应是粉红色一反映适当的氧水平[见剂量和给药方法(2.2)和如何供应/贮存和处置]。这些是供应在指纸盒如列出如下:‘
NDC 70510-2171-1 30mg/100mL(0.3mg/mL)一次性-剂量袋
NDC 70510-2171-2 2袋每纸盒
贮存和处置
贮存至25°C(77°F)。外出允许从15°C至30°C(59°F至86°F)[见USP控制室温]。避光保护。贮存在包住的包装内保护来自氧降解直至使用时间。指示剂将转转蓝或字如氧已超过可接受水平。一旦外包装被打开，在24小时内使用.

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.”
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.
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.
The most common adverse reactions reported by clinical trial participants receiving edaravone were bruising (contusion) and gait disturbance.
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.
The FDA granted this drug orphan drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases.
The FDA granted approval of Radicava to Mitsubishi Tanabe Pharma America, Inc.
https://www.mt-pharma-america.com/wp-content/uploads/2017/05/Approval-NDA209176-edaravone-final-PI.pdf