2月14日，FDA批准BioMarin制药的Vimizim (elosulfase alfa)，用于治疗黏多醣贮积症，这是首个治疗该症的药物。黏多糖贮积症是一种罕见病（美国800例），患者缺乏N-acetylgalactosamine-6-sulfate sulfatase (GALNS)，Vimizim可以替代该酶。
商品名：Vimizim 通用名：elosulfase alfa 中文名：未知
审批分类：优先审评+孤儿药
药企：BioMarin Pharmaceutical Inc
适应症：ⅣA型黏多糖贮积症（Mucopolysaccharidosis type IVA）。
剂型规格：5 mg/5 mL，推荐剂量为2 mg/kg，静脉注射，每周一次。
活性成分：elosulfase alfa，一种通过DNA重组技术生产的酶。
作用机理：ⅣA型黏多糖贮积症是由于缺乏N-acetylgalactosamine-6 -sulfatase活性，不能有效地水解黏多糖，黏多糖蓄积而导致细胞、组织、器官损伤，elosulfase alfa作为酶替代疗法治疗该症。
临床试验：一项随机双盲安慰剂对照评价了Vimizim的安全性和有效性，176例患者分成Vimizim 2 mg/kg/周组、Vimizim 2 mg/kg/2周组、安慰剂组，经过24周治疗后，Vimizim 2 mg/kg/周组比安慰剂组的6分钟步行距离增加22.5米，Vimizim 2 mg/kg/2周组与安慰剂组相似。
黑框警告：致死性过敏反应。
不良反应：过敏反应（18.7%）、发热（33%）、呕吐（31%）、头痛（26%）、恶心（24%）、腹痛（21%）、发冷（10.3%）、疲乏（10.3%）。
补充说明：Vimizim是FDA批准的首个ⅣA型黏多糖贮积症药物，美国有该症患者800例。
FDA approves Vimizim to treat rare congenital enzyme disorder
First drug to receive Rare Pediatric Disease Priority Review Voucher
The U.S. Food and Drug Administration today approved Vimizim (elosulfase alfa), the first FDA-approved treatment for Mucopolysaccharidosis Type IVA (Morquio A syndrome). Morquio A syndrome is a rare, autosomal recessive lysosomal storage disease caused by a deficiency in N-acetylgalactosamine-6-sulfate sulfatase (GALNS). Vimizim is intended to replace the missing GALNS enzyme involved in an important metabolic pathway. Absence of this enzyme leads to problems with bone development, growth and mobility. There are approximately 800 patients with Morquio A syndrome in the United States.
Vimizim was granted priority review. An FDA priority review provides for an expedited review of drugs for serious diseases or conditions that may offer major advances in treatment. Vimizim is also the first drug to receive the Rare Pediatric Disease Priority Review Voucher - a provision that aims to encourage development of new drugs and biologics for the prevention and treatment of rare pediatric diseases.
“This approval and rare pediatric disease priority review voucher underscores the agency’s commitment to making treatments available to patients with rare diseases,” said Andrew E. Mulberg, M.D., deputy director, Division of Gastroenterology and Inborn Errors Products in the FDA’s Center for Drug Evaluation and Research (CDER). “Prior to today’s approval, patients with this rare disease have had no approved drug treatment options.”
The safety and effectiveness of Vimizim were established in a clinical trial involving 176 participants with Morquio A syndrome, ranging in age from 5 to 57 years. Participants treated with Vimizim showed greater improvement in a 6-minute walk test than participants treated with placebo. On average, patients treated with Vimizim in the trial walked 22.5 meters farther in 6 minutes compared to the patients who received placebo.
The most common side effects in patients treated with Vimizim during clinical trials included fever, vomiting, headache, nausea, abdominal pain, chills and fatigue. The safety and effectiveness of Vimizim have not been established in pediatric patients less than 5 years of age. Vimizim is being approved with a boxed warning to include the risk of anaphylaxis. During clinical trials, life-threatening anaphylactic reactions occurred in some patients during Vimizim infusions.
Vimizim is marketed by Novato, Calif.-based BioMarin Pharmaceutical Inc.
关键词： BioMarin FDA 批准 Vimizim 黏多醣贮积症
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