Cuvitru（Immune Globulin Subcutaneous (Human) 20% Solution）为治疗：原发性免疫缺陷综合证 近日，免疫球蛋白产品Cuvitru（免疫球蛋白[人]皮下注射液，20%）获得美国食品和药物管理局（FDA）批准，用于2岁及以上儿童及成人原发性免疫缺陷症（Primary Immunodeficiency，PI）的治疗。 Cuvitru是唯一一种浓度高达20%（高浓度）的皮下注射免疫球蛋白产品，不含脯氨酸（proline），在每个注射位点可注射多达60毫升（12g），每个位点每小时可耐受60毫升。与常规的低浓度皮下注射免疫球蛋白产品相比，Cuvitru的注射位点更少，输注持续时间更短。Cuvitru将为原发性免疫缺陷患者提供给药的灵活性，使其根据自身需要定制最适合的治疗。 批准日期：2016年9月14；公司：Shire plc CUVITRU(免疫球蛋白皮下(人)Immune Globulin Subcutaneous (Human), 20% Solution)注射液  供皮下注射 美国初次批准：2016 作用机制 CUVITRU提供一个对一个广泛各种各样细菌和病毒剂广谱的调理作用和中和IgG抗体。CUVITRU还含一个抗体谱能够与和相互作用改变免疫系统的细胞的活性以及抗体能够与细胞例如红细胞反应。这些抗体和IgG的作用机制在CUVITRU的作用尚未完全阐明。 适应证和用途 • CUVITRU是一种免疫球蛋白皮下(人)，20%溶液适用为原发性体液免疫缺陷(PI)在2岁和以上成年和儿童患者替代治疗。 剂量和给药方法 只为皮下输注. 从每天至每2周在定期间隔给药。 根据患者的药代动力学和临床反应个体化剂量。 监视血清IgG谷水平定期地指导随后剂量调整和给药间隔当需要时。 从免疫球蛋白静脉(人)治疗(IGIV)或成年患者转自HYQVIA[免疫球蛋白输注10%(人)与重组人透明质酸[Hyaluronidase]]： •患者的末次IGIV或HYQVIA输注后开始治疗一周。 •通过转换每月IGIV或HYQVIA剂量至等同每周剂量确定初始每周剂量和用一个剂量调整系数增加它。 •频繁给药(2-7倍每周)：计算的每周剂量被除以每周需要次数。 • 每2周给药：每周计算剂量乘以2。 从免疫球蛋白皮下(人)转至治疗(IGSC)： • 每周剂量(克数)应是与以前IGSC治疗(克数)每周的剂量相同。 • 频繁给药(2-7倍每周)：每周计算的剂量除以每周想要的次数。 • 每2周给药：每周计算剂量乘以2。 输注部位：直至同时4个输注部位，部位间至少有4英寸避开骨突起。每次给药轮转部位。 剂型和规格 • 200mg/mL(20%)蛋白溶液为皮下输注 禁忌证 • 对皮下给予的免疫球蛋白(人)过敏样或严重全身性超敏性反应。 • 有抗体对IgA和超敏性病史IgA缺陷患者。 警告和注意事项 •有抗体对IgA处于发生严重超敏性和过敏样反应的更大风险是IgA缺陷患者。 • 处于急性肾功能衰竭的风险患者监视肾功能，包括血尿素氮，血清肌酐，和尿排出量。 • 可能发生血栓形成。对处于高粘血症风险患者监视血栓形成体征和症状和评估血液黏度。 • 可能发生无菌性脑膜炎综合征(AMS) • 监视溶血临床体征和症状。 • 监视患者肺不良反应(输血相关急性肺损伤，TRALI)。 • 产品是由人血制备，它可能携带发送传染性病原体的危险，如，病毒，变异的克-雅氏病e(vCJD)剂，和理论上，Creutzfeldt-Jakob病剂。 不良反应 在≥5%的患者观察到最常见不良反应为：局部不良反应，全身性不良反应包括头痛，恶心，疲乏，腹泻，和呕吐。 药物相互作用 抗体的被动转运可能短暂干扰对活病毒疫苗免疫反应，例如麻疹，腮腺炎，风疹，和水痘。 贮存和处置 •贮存在冰箱温度：2°C至8°C [36°F至46°F]共至36个月或 •室温：不超过25°C [77°F])共至12个月。 如用户将CUVITRU带出至室温不要将它返回冰箱。 •不要冻结。 •不要摇晃。. •为了避光保护保持小瓶在纸盒中。 •遗弃任何未使用产品。 不要使用超过失效期产品 Shire Announces U.S. FDA Approval of CUVITRU[TM] [Immune Globulin Subcutaneous (Human), 20% Solution] Treatment for Primary Immunodeficiency Approval follows positive data from clinical studies on efficacy and tolerability, as well as infusion time and number of injection sites United States Food and Drug Administration (FDA) has granted approval for CUVITRU [Immune Globulin Subcutaneous (Human), 20% Solution] in adult and pediatric patients two years of age and older. CUVITRU is a treatment for patients with primary immunodeficiency (PI), a group of more than 300 genetic disorders in which part of the body's immune system is missing or functions improperly; it affects up to six million people worldwide. With the approval of CUVITRU, Shire now has the broadest portfolio of intravenous and subcutaneous immunolglobulin (IG) products, including the only once-a-month subcutaneous treatment option. CUVITRU is the only 20% subcutaneous IG treatment option without proline and with the ability to infuse up to 60 mL (12 grams) per site and 60 mL per hour, per site as tolerated, resulting in fewer infusion sites and shorter infusion durations compared to other conventional subcutaneous IG treatments. Regardless of infusion rate or volume per site, CUVITRU was generally associated with a low incidence of local adverse and systemic reactions (0.022/infusion and 0.042/infusion, respectively) in the North American clinical study. "In the clinical study, primary immunodeficiency patients tolerated CUVITRU favorably despite the use of higher infusion site volumes and more rapid infusion rates than have been routine in the past," said Richard L. Wasserman, M.D., Ph.D., Medical Director of Pediatric Allergy and Immunology at Medical City Children's Hospital. "The availability of CUVITRU as a high concentration, subcutaneous IG provides primary immunodeficiency patients with the dosing flexibility that allows them to customize their therapy to best fit their individual needs." "As the national patient organization dedicated to people with primary immunodeficiency, the Immune Deficiency Foundation is delighted to see new treatment options that can address patients' clinical priorities as well as their personal preferences, particularly in terms of dosing and administration," said Marcia Boyle, President and Founder of the Immune Deficiency Foundation. "We commend Shire's focus on innovation and continued commitment to supporting the PI community." The approval follows the recent publication of a Phase II/III study of CUVITRU among North American patients in the Journal of Clinical Immunology. Shire expects to launch CUVITRU in the U.S. in the coming weeks. CUVITRU was approved in 17 European countries in June of 2016. The company expects to initiate additional global regulatory submissions for CUVITRU in late 2016 and 2017. "As CUVITRU is now approved in the U.S. following European approval earlier this year, Shire's broad IG treatment portfolio offers a wide range of options to help physicians tailor treatment regimens to meet the priorities of their patients managing these life-long disorders," said Perry Sternberg, Head of U.S. Commercial, Shire. "We look forward to making CUVITRU available to the community this fall." About Primary Immunodeficiency Primary immunodeficiencies (PI) are a group of more than 300 disorders in which part of the body's immune system is missing or does not function properly. Normally, the immune system protects the body from pathogenic microorganisms like bacteria, viruses, and fungi, which can cause infectious diseases. When any part of a person's immune system is absent or dysfunctional, the individuals are susceptible to infections, and it may take longer to recover from infections. When a defect in the immune system is inherited and genetically determined, it is called primary immune deficiency. It is estimated that as many as six million children and adults may be affected by PI worldwide. About CUVITRU [Immune Globulin Subcutaneous (Human), 20% Solution] CUVITRU is an Immune Globulin Subcutaneous (Human) (IGSC), 20% Solution indicated as replacement therapy for primary humoral immunodeficiency (PI) in adult and pediatric patients two years of age and older. CUVITRU is for subcutaneous infusion only.    Detailed Important Risk Information BOXED WARNING: THROMBOSIS Thrombosis may occur with immune globulin products, including CUVITRU. Risk factors may include: advanced age, prolonged immobilization, hypercoagulable conditions, history of venous or arterial thrombosis, use of estrogens, indwelling vascular catheters, hyperviscosity and cardiovascular risk factors. For patients at risk of thrombosis, administer CUVITRU at the minimum dose and infusion rate practicable. Ensure adequate hydration in patients before administration.  Monitor for signs and symptoms of thrombosis and assess blood viscosity in patients at risk of hyperviscosity. CONTRAINDICATIONS CUVITRU is contraindicated in patients who have had an anaphylactic or severe systemic hypersensitivity reaction to the subcutaneous administration of human immune globulin and in IgA-deficient patients with antibodies against IgA and a history of hypersensitivity to human immune globulin treatment. WARNINGS and PRECAUTIONS  Hypersensitivity:  Severe hypersensitivity reactions may occur, even in patients who have tolerated previous treatment with human immune globulin.  IgA-deficient patients with antibodies to IgA are at greater risk of developing potentially severe hypersensitivity and anaphylactic reactions. Renal Dysfunction/Failure:  Monitor renal function and urine output and consider lower, more frequent dosing in patients who are at risk of developing renal dysfunction because of pre-existing renal insufficiency or predisposition to acute renal failure. Thrombosis: Monitor for signs and symptoms of thrombosis and assess blood viscosity for those at risk for hyperviscosity. Aseptic Meningitis Syndrome (AMS):  Monitor for clinical signs and symptoms of AMS. Hemolysis:  Monitor for clinical signs and symptoms of hemolysis and delayed hemolytic anemia. Transfusion-Related Acute Lung Injury (TRALI):  Monitor for pulmonary adverse reactions associated with TRALI. Transmittable Infectious Agents:  Because CUVITRU is made from human plasma, it may carry a risk of transmitting infectious agents, such as viruses and other pathogens.  No confirmed cases of transmission of viral diseases or variant Creutzfeldt-Jakob disease (vCJD) have been associated with CUVITRU. Interference with Laboratory Tests:  False positive serological test results, with the potential for misleading interpretation, may occur as the result of passively transferred antibodies. ADVERSE REACTIONS  The most common adverse reactions observed in clinical trials in ≥ 5% of patients were:  local adverse reactions, systemic adverse reactions including headache, nausea, fatigue, diarrhea, and vomiting. Shire原发性免疫缺陷症新药Cuvitru（免疫球蛋白[人]皮下注射液，20%）获美国FDA批准 2016年9月16日英国制药商Shire的免疫球蛋白产品Cuvitru（免疫球蛋白[人]皮下注射液，20%）获得美国食品和药物管理局（FDA）批准，用于2岁及以上儿童及成人原发性免疫缺陷症（Primary Immunodeficiency，PI）的治疗。 Cuvitru是唯一一种浓度高达20%（高浓度）的皮下注射免疫球蛋白产品，不含脯氨酸（proline），在每个注射位点可注射多达60毫升（12g），每个位点每小时可耐受60毫升。与常规的低浓度皮下注射免疫球蛋白产品相比，Cuvitru的注射位点更少，输注持续时间更短。Cuvitru将为原发性免疫缺陷患者提供给药的灵活性，使其根据自身需要定制最适合的治疗。 Cuvitru的获批，是基于一项II/III期临床研究的数据。该研究显示，Cuvitru与低浓度免疫球蛋白产品相比具有相同的疗效和副作用，但可显著减少皮下注射位点的数量和输注所需的时间。 随着Cuvitru的获批，Shire公司现在拥有了最广泛的静脉注射和皮下注射免疫球蛋白（IG）产品组合，其中包括唯一的每月一次的皮下注射产品。Shire预计将在未来几周内将Cuvitru推向美国市场。 在欧洲，Cuvitru于今年6月获批，作为一种替代疗法，用于2岁及以上儿童及成人原发性免疫缺陷（PI）的治疗中。Shire已计划在2016年底和2017年向全球的其他监管机构提交上市申请文件。 原发性免疫缺陷（PI）是由于遗传原因导致机体免疫系统部分功能缺失或失常。免疫系统担负着保护人体不被细菌、病毒和真菌等病原体感染的功能。当免疫系统的部分功能丧失时，患者会更容易受到感染，并且感染后恢复过程更缓慢。目前原发性免疫缺陷包含了300多种不同遗传因素造成的免疫缺陷。 据估计全世界有600万儿童和成人受到原发性免疫缺陷的困扰。其中53％的患者体内生成抗体的机制出现缺陷，从而可能导致血清中免疫球蛋白水平下降。