2011年2月17日，美国食品与药物管理局（FDA）发布公告，批准Corifact用于预防先天性凝血因子XIII缺乏症患者的出血症。 先天性凝血因子XIII缺乏症是一种罕见的基因缺陷病。Corifact是首个被FDA批准用于治疗该病的制剂。 凝血因子XIII是一种存在于血液循环并参与正常凝血过程的重要物质。先天性凝血因子XIII缺乏症患者体内无法生成足够量的凝血因子XIII。如果不予以治疗，这些患者便有威及生命的出血危险。 先天性凝血因子XIII缺乏症是一种罕见病，美国每300万到500万人中有1人得此病。这种凝血因子的缺乏可能会导致软组织瘀伤、粘膜出血和致命性颅内出血。患有凝血因子XIII缺乏症的新生儿可能在出生时有脐带出血。 “该制剂有助于填补一项重要需求”，FDA生物制品评价和研究中心主任Karen Midthun博士说。 Corifact获得FDA罕用药资格，因为其适用于一种罕见病或罕见状态。Corifact虽然根据FDA加速批准规定被批准上市，但它仍需完成一项持续研究，以证明患者实际的临床获益与迄今取得研究数据所预期的一致。 FDA基于1项对先天性凝血因子XIII缺乏症患者的临床研究结果批准了Corifact。该研究纳入14例患者，其中包括儿童。研究中，被观察到的最常见副作用是过敏反应（过敏、皮疹、瘙痒和红斑）、寒战、发烧、关节痛、头痛、凝血酶-抗凝血酶水平升高以及肝酶升高。 Corifact，第十三因子浓缩物（人） 静脉使用。冻干浓缩的重构 初始美国批准：2011   适应症 Corifact被用于日常的先天性因子XIII（FXIII）缺乏预防性治疗。该Corifact有效性是基于维持一个低谷FXIII约5％至20％的活性水平。有没有显示出对出血Corifact（1）发作的治疗直接受益对照试验。 剂量和用法 对于静脉注射使用。重组后才能使用。 初始剂量 •40国际单位（单位）每公斤体重 •管理的速度不超过每分钟4毫升 后续计量 •计量应遵循最新的低谷FXIII活动的水平，与剂量每28天（4周）维持约5％至20％谷底水平。推荐剂量为每公斤± 5个单位的调整应根据槽FXIII <5％或> 20％，病人的临床状况活动水平。 •计量调整应遵循基于一个特定的检测来确定FXIII水平。一个剂量使用Berichrom ®活性测定调整的例子是列于表1所示。 调整剂量使用Berichrom活性测定（2.2，表1） FXIII活动槽级别（％）用量变化 其中<5％提高槽每公斤级5个单位 海槽水平为5％至20％没有变化 两个> 20％减少谷底水平由每公斤5个单位 其中> 25％，减少槽每公斤级5个单位 制备与重构 •在原纸箱和冰箱商店在2-8小瓶° C（36-46 ° F）。不要冻结。 •复溶Corifact使用前用无菌水20毫升的注射液，USP。 管理 •不要与其他药物混合产品。管理通过一个单独的输液线。   剂型和规格 •Corifact作为一次性使用含有作为重建冻干浓缩1000-1600小瓶的FXIII单位提供。 禁忌 •Corifact禁用于已知过敏性或与人血浆来源的产品，或在Corifact任何组件严重的全身反应的患者。   警告和注意事项 •可能发生过敏反应。如有必要，应停止给药并制定适当的治疗。 •抑制抗体已被发现患者在接受Corifact。如果预期FXIII活动水平没有达到，或者如果突破性出血发生在接受预防治疗，一个试验的措施FXIII抑制抗体浓度应执行（5.2）。 •血栓性事件的报道在接受Corifact患者。称取与血栓形成的风险的管理带来的好处。 •Corifact是由人类的血液和可能携带传染性病原体的传播风险，例如，病毒和理论上，克雅氏病（CJD）代理。 不良反应 •最常见的不良报道Corifact临床研究，超过一科（频率> 1％），反应是过敏反应（包括过敏，皮疹，瘙痒和红斑），寒战/升温度，关节痛，头痛，高架凝血酶 - 抗凝血酶水平，以及肝酶增加。 •中和抗体对FXIII报告了一项临床研究。 特殊族群使用 •小儿：短半衰期和通关速度较成人（后为每公斤体重调整）已被观察到。这些结果是因为受试者数量有限难以解释（N = 5）。 修订：02/2011 FDA：批准首个先天性凝血因子XIII缺陷症新药 2011年2月17日，美国食品药品监督管理局批准了Corifact，是首个用于防止先天性凝血因子XIII缺乏症患者出血的药物。 此类患者凝血因子XIII产生不足，该物质随血液循环且有凝血作用，如果没有妥善的治疗，其出血可能危及生命。 先天性凝血因子XIII缺乏症是一种极为罕见的疾病，在美国约每300到500万人中有一名患者。该缺乏症可能引起软组织挫伤、粘膜出血甚至是致命性颅内出血，XIII因子缺乏的新生儿可能有脐带出血。 FDA生物制剂评价及研究中心负责人Karen Midthun医学博士说：该药物的获批弥补了一项重要的空白。 Corifact是通过FDA的罕见药程序获批的，因为其用于治疗罕见疾病，通过快速审批程序，但是需要在上市后对继续进行临床试验并提交研究结果。 FDA是在一项对14名(包括儿童)凝血因子XIII缺乏患者的临床研究结果作出此项批准的。该药物的常见不良反应有过敏反应(皮疹、瘙痒、红斑)、发冷、发烧、关节痛、头疼、凝血酶-抗凝血酶水平提高以及肝酶升高等。 Corifact由健康捐赠者的血浆制得。患者使用可能会产生凝血因子XIII抗体而使药物失效。如果用量超过说明书标准可能产生不正常凝血现象。 Corifact由位于德国马堡的CSL Behring生产。 FDA批准首个治疗先天性凝血因子XIII缺乏症的制剂 Important Safety Information Corifact is indicated for congenital factor XIII lack of routine preventive treatment. Effectiveness is based on the Corifact maintain trough FXIII about 5% to 20% of the level. Do not show the treatment of bleeding episodes controlled study Corifact direct benefit. Corifact must be administered intravenously. Corifact known allergies or contraindications with human plasma-derived products or any part of Corifact part or severe systemic response to the individual. Allergic reactions may occur Corifact. If you have allergies or allergic reactions (including urticaria, rash, chest tightness, wheezing, hypotension, etc.) sign, immediately stop the management and the establishment of appropriate treatment. Inhibition antibody has been found in patients receiving FXIII Corifact. Monitoring during the treatment of patients with trough FXIII activity levels. If the expected level of plasma FXIII activity or breakthrough bleeding is not achieved, the implementation of an experimental measurement of FXIII inhibition antibody concentration. Thromboembolic events have been reported Corifact. Balance and risk of thrombosis, particularly pregnant women, who are at increased risk administrative efficiency. Corifact from human plasma. Infectious disease personnel, including viruses, and theoretically transfer the risk of Creutzfeldt-Jakob disease (CJD) agent, can not be completely eliminated. In clinical trials, the most common adverse reactions, more than one subject (frequency> 1%) and after treatment were Corifact allergic reaction (including allergies, rashes, itching and erythema) report, chills / temperature, joint pain, increased headache, elevated thrombin antithrombin levels, the increase in liver enzymes. CSL Behring received FDA congenital factor XIII deficiency Corifact ™ certification CSL Behring announced today that the U.S. Food and Drug Administration (FDA) has granted marketing Corifact ™, factor VIII concentrate (human), the approval of congenital factor XIII (FXIII) lack of routine preventive treatment. Corifact, has been used in 12 countries around the world called Fibrogammin ® products provide - P, is the first and only FXIII concentrate approved in the U.S. Congenital FXIII deficiency, also known as fibrin stabilizing factor deficiency is a rare potentially life-threatening bleeding disorder, in which the normal blood clot, but the instability of blood clots, leading to recurrent bleeding. It is estimated that conditions affecting the incidence in the United States about 150 people, a hundred million people. "The U.S. FDA approved Corifact is a very special opportunity to people who need our security, high-quality therapy, positive changes in life, while continuing to strengthen and hemorrhagic diseases of our products, broad product portfolio," Powell said Lynne, CSL Behring, senior vice president, North American business operations. "Corifact is the fourth new product, CSL Behring has brought to the U.S. market over the past two years. It represents our development of non-rare and serious disease treatment products has always been committed." Congenital FXIII deficiency symptoms include bleeding from the birth, poor wound healing, miscarriage, subcutaneous bleeding, umbilical cord, joints and muscles, and excessive bleeding after trauma. Lack of FXIII in patients with high protein but also the risk of intracranial hemorrhage (ICH), and inside the skull, can be life-threatening bleeding. Studies show that 25 to 60 FXIII deficient patients are in their life will experience at least one of the intangible cultural heritage. For an accurate diagnosis of FXIII deficiency, doctors, blood tests and detailed and specific family history is necessary. About Corifact ™ Corifact, intravenous (IV) infusion about every 28 days, to be used for routine preventive treatment of congenital FXIII deficiency. There is no controlled trials show the treatment of bleeding episodes Corifact direct benefit. Corifact is a fibrin stabilizing factor concentrates provide A-and B - subunit to prevent the FXIII deficiency. Clinical studies have shown that, Corifact an immediate increase in blood levels of FXIII. Concentrated dose of the formula, based on the patient's body weight for 5 to 20 years per cent of the normal level of FXIII slot. Corifact packaged as freeze-dried in a high stability and long shelf life, single-use vials of powder. Concentrate is stable for up to 6 months at room temperature (25 ° C) and 24 months refrigerated (2-8 ℃). Corifact also has a low fluid volume, which will help to reduce management time. Corifact also be used as Fibrogammin ® - phosphorus in Argentina, Austria, Belgium, Brazil, France, Germany, Britain, Indonesia, Israel, Japan, Luxembourg and Switzerland. Corifact not who's personal experience with known hypersensitivity or allergy to human plasma-derived products or components in the development of symptoms of any serious systemic reactions. In clinical studies, the most common adverse reactions reported include the following and Corifact allergies (including allergies, skin rash, itching, rash), chills / temperature, joint pain, headache, increased by elevated levels of thrombin antithrombin in the liver enzyme increases. The physician should monitor the patient's course of treatment slots FXIII activity levels. If the expected FXIII level was not reached, or breakthrough bleeding occurs, an investigation to determine the presence of antibodies FXIII inhibitors, should be implemented. Corifact security guarantees after virus inactivation and removal. Corifact from human plasma. Infectious disease personnel, including viruses, and theoretically transfer the risk of Creutzfeldt-Jakob disease (CJD) agent, can not be completely eliminated. For more information, including full prescribing information, please visit. About CSL Behring CSL Behring is a leader in plasma protein therapeutics industry. Committed to saving lives and improving with rare and serious disease quality of life, production and marketing of the company and restructuring of various blood-borne treatment worldwide. CSL Behring therapies are indicated for hemophilia coagulation disorders and vascular diseases such as hemophilia, primary immune deficiency, hereditary angioedema and inherited respiratory disease. The company's products are heart surgery, organ transplantation use, burn treatment and prevention of hemolytic disease of newborns. CSL Behring operates the world's largest plasma collection networks, CSL is one of the plasma. CSL Behring is a CSL Limited (ASX: CSL's) subsidiary, a biopharmaceutical company headquartered in Melbourne, Australia. For more information, visit the important safety information Corifact is indicated for congenital factor XIII lack of routine preventive treatment. Effectiveness is based on the Corifact maintain trough FXIII about 5% to 20% of the level. Do not show the treatment of bleeding episodes controlled study Corifact direct benefit. Corifact must be administered intravenously. Corifact known allergies or contraindications with human plasma-derived products or any part of Corifact part or severe systemic response to the individual. Allergic reactions may occur Corifact. If you have allergies or allergic reactions (including urticaria, rash, chest tightness, wheezing, hypotension, etc.) sign, immediately stop the management and the establishment of appropriate treatment. Inhibition antibody has been found in patients receiving FXIII Corifact. Monitoring during the treatment of patients with trough FXIII activity levels. If the expected level of plasma FXIII activity or breakthrough bleeding is not achieved, the implementation of an experimental measurement of FXIII inhibition antibody concentration. Thromboembolic events have been reported Corifact. Balance and risk of thrombosis, particularly pregnant women, who are at increased risk administrative efficiency. Corifact from human plasma. Infectious disease personnel, including viruses, and theoretically transfer the risk of Creutzfeldt-Jakob disease (CJD) agent, can not be completely eliminated. In clinical trials, the most common adverse reactions, more than one subject (frequency> 1%) and after treatment were Corifact allergic reaction (including allergies, rashes, itching and erythema) report, chills / temperature, joint pain, increased headache, elevated thrombin antithrombin levels, the increase in liver enzymes. Please see the full prescribing information.