美国FDA日前批准了治疗遗传性血管水肿（HAE）的一线治疗药物Berinert（C1酯酶抑制剂）。

    HAE是一种罕见且具有潜在生命威胁的遗传性疾病（一种罕见的潜在威胁生命的遗传病），特征为急性腹痛发作和面部浮肿。Berinert主要用于治疗成人和青少年HAE，这种疾病可能自然发生或于应激、外科手术、感染后出现。腹痛发作症状包括严重的腹痛、恶心、呕吐、绞痛和腹泻。Berinert是来源于人血浆的蛋白产物，主要调节血液凝固和炎症反应。一项纳入了124例C1酯酶抑制因子缺乏的成人或青少年患者的临床试验表明，Berinert对治疗HAE患者出现的急性中重度腹痛发作和颜面部水肿症状有良好效果。

    Berinert使用禁忌证为对C1 酯酶抑制剂有超敏反应的患者，报道的最严重不良反应为HAE所致的严重疼痛，最常见的不良反应包括并发的HAE发作，头痛，腹痛，恶心，肌肉痉挛，疼痛，腹泻和呕吐。Berinert（C1酯酶抑制剂）是由德国的CSL Behring公司生产。

FDA Approves Berinert to Treat Abdominal Attacks, Facial Swelling Associated With Hereditary Angioedema
 
 (Oct. 9, 2009)The U.S. Food and Drug Administration today approved Berinert, the first treatment for acute abdominal attacks and facial swelling associated with a rare and potentially life-threatening genetic disease called hereditary angioedema (HAE).
 
Berinert is approved for adults and adolescents with HAE, which can occur spontaneously or during stress, surgery, or infection in patients diagnosed with HAE. The symptoms during abdominal attacks include severe abdominal pain, nausea, vomiting, cramps, and diarrhea.
 
“Berinert will enhance the treatment options for individuals who experience acute abdominal attacks and facial swelling associated with hereditary angioedema,” said Karen Midthun, M.D., acting director of FDA’s Center for Biologics Evaluation and Research.
 
Berinert is a protein product derived from human plasma. It regulates clotting and inflammatory reactions that, when impaired, can lead to local tissue swelling. In a clinical trial of 124 adults and adolescents with C1 esterase, inhibitor deficiency, Berinert was shown to be effective at treating the symptoms of acute moderate to severe abdominal attacks and facial swelling in patients with HAE.
 
Berinert is contraindicated in patients with a history of life-threatening hypersensitivity reaction to C1 esterase inhibitor preparations.  The most serious adverse reaction reported in clinical studies was an increase in the severity of pain associated with HAE. The most common adverse reactions include subsequent HAE attack, headache, abdominal pain, nausea, muscle spasms, pain, diarrhea and vomiting.
 
Berinert is manufactured by CSL Behring, Inc., Marburg, Germany.