近日，美国食品和药品监管局FDA批准Coagadex，凝血因子X（人），为遗传性因子X（10）缺乏。直至今天的孤儿药物批准，对有遗传性因子X缺乏患者不能得到特异性凝血因子替代治疗。
FDA的生物制品评价和研究中心主任Karen Midthun，M.D.说：“Coagadex的批准对患有这个罕见但严重疾病患者是一个重要进展。”
批准日期：2015年10月20日；公司：Bio Products Laboratory有限公司
COAGADEX®(凝血因子X(人)凝血因子X(人))为静脉注射溶液/冰冻干燥粉
美国初次批准：[2015]
作用机制
COAGADEX暂时地取代丢失的对有效止血需要的因子X。因子X是一种无活性的酶原，它可被因子IXa (通过内在通路)或被因子VIIa(通过外在通路)激活。因子X从其无活性形式转换为活性形式(因子Xa)通过一个52-残基肽从重链的裂解。因子Xa与因子Va在一个磷脂表面上结合形成凝血酶原酶复合物，它在存在钙离子时激活凝血酶原为凝血酶。然后凝血酶作用与可溶性纤维蛋白和因子XIII形成一个交联纤维蛋白凝块。
适应证和用途
COAGADEX，凝血因子X(人)，是一种血浆来源人血凝血因子适用在成年和儿童(年龄12岁和以上)有遗传因子X缺乏为：
• 按需治疗和出血发作的控制
• 有轻度遗传性因子X缺乏患者中出血的围手术处理.
使用的限制
未曽在有中度和严重遗传性因子X缺乏患者研究重大手术出血的围手术处理。
剂量和给药方法
只为重建后静脉使用
⑴ 每小瓶COAGADEX含以国际单位(IU)的标记量因子X。
⑵ 治疗的剂量和时间依赖于因子X缺乏的严重程度，出血的位置和程度和患者的临床情况。
⑶ 为出血发作的治疗：每kg体重使用25IU，间隔24小时重复直至出血停止。
⑷ 为围手术处理：
① 手术前，升高血浆因子X水平至70-90IU/dL。
② 手术后，维持血浆因子X水平在最低50IU/dL直至患者不再处于由于手术出血风险。
③ 所需剂量(IU)=体重(kg)×想要因子X升高(IU/dL或正常的%)×0.5
剂型和规格
COAGADEX是可得到为冰冻干燥粉为重建在一次性使用小瓶含名义上(约) 250 IU或500 IU的因子X活性。当用药盒供应的无菌注射用水重建，最终浓度是约100 IU/mL。
禁忌证
在患者有对COAGADEX或组分的任何有威胁生命超敏性反应不要使用。
警告和注意事项
⑴ 超敏性反应，包括过敏性反应，是可能的。症状发生，终止COAGADEX和给予适当治疗。
⑵ 可能发生中和抗体的发展(抑制剂)。如没有达到预期血浆因子X活性水平，或如用适当剂量不能控制出血，进行分析测定因子X抑制剂浓度。
⑶ COAGADEX是从人血制造和所以携带传播传染病原体，如病毒，变异型克雅氏病[variant Creutzfeldt-Jakob disease(vCJD)]病原体和，理论上，克雅氏病(CJD)病原体。
不良反应
在临床试验中观察到最常见不良药物反应(频数 ≥5%受试者)是输注部位红斑，输注部位疼痛，疲乏和背痛。
贮存和处置
供应
COAGADEX是supplied在一次性使用玻璃小瓶中供应含一个名义上(约)250IU或500IU(重建后约100IU/mL)的因子X活性，分别用注射用无菌水2.5mL或5mL包装，和一个Mix2小瓶转运装置。.
小瓶是用一种合成橡皮塞密封。塞子被铝与翻转聚丙烯帽确保顶封。
规格            药  盒
250IU    64208-7752 1
500IU    64208-7753-1

贮存和处置
● 避光保护贮存COAGADEX其原始包装内。
● 贮存COAGADEX包装在冰箱或在室温(36°F至86°F)。不要冻结。
● 不要使用小瓶和纸盒标签印有是失效日期后的COAGADEX或无菌注射用水。失效日期是指该月最后1天。
● 在重建的一小时内使用重建的COAGADEX。
●如重建溶液是云雾状或含任何颗粒不要使用COAGADEX
COAGADEX (Coagulation Factor X, Human)
Brand name: Coagadex
Generic name: coagulation factor X (human)
Company: Bio Products Laboratory Limited
Treatment for: Hereditary Factor X Deficiency
Coagadex (coagulation factor X (human)) is a human factor X concentrate for the treatment of hereditary factor X deficiency.
Hereditary factor X deficiency is a rare bleeding disorder caused by a lack of clotting factor X in the blood. Coagadex is a plasma-derived blood coagulation factor X concentrate used to treat patients 12 years and older with mild hereditary factor X deficiency for the control of bleeding episodes, and perioperative management of bleeding.
Allergic type hypersensitivity reactions, including anaphylaxis, are possible with Coagadex. Patients should stop treatment and contact their healthcare provider right away if they experience any of the following symptoms: difficulty breathing, chest tightness, swelling of the face, rash or hives. Coagadex is contraindicated in patients with known hypersensitivity to any of the components of the product.
Coagadex is made from human plasma and may contain infectious agents such as viruses and, theoretically, the Creutzfeldt-Jakob disease agent. No cases of transmission of viral diseases, vCJD or CJD, have been associated with the use of Coagadex.
In clinical studies, the most common side effects (frequency ≥5% of subjects) of Coagadex were infusion site erythema, infusion site pain, fatigue and back pain.
Indications for COAGADEX
COAGADEX, a plasma-derived blood coagulation factor X concentrate, is indicated in adults and children (aged 12 years and above) with hereditary factor X deficiency for:
•On-demand treatment and control of bleeding episodes
•Perioperative management of bleeding in patients with mild hereditary factor X deficiency
Perioperative management of bleeding in major surgery in patients with moderate and severe hereditary factor X deficiency has not been studied.
Important Safety Information for COAGADEX
COAGADEX is contraindicated in patients with known hypersensitivity to any of the components of the product.
Allergic type hypersensitivity reactions, including anaphylaxis, are possible with COAGADEX. If symptoms occur, patients should discontinue use of the product immediately and contact their physician.
The formation of neutralizing antibodies (inhibitors) to factor X is a possible complication in the management of individuals with factor X deficiency. Carefully monitor patients taking COAGADEX for the development of inhibitors by appropriate clinical observations and laboratory tests.
COAGADEX is made from human plasma and may contain infectious agents, e.g. viruses, the variant Creutzfeldt-Jakob disease (vCJD) agent and, theoretically, the Creutzfeldt-Jakob disease (CJD) agent. No cases of transmission of viral diseases, vCJD or CJD, have been associated with the use of COAGADEX.
In clinical studies, the most common adverse reactions (frequency ≥5% of subjects) with COAGADEX were infusion site erythema, infusion site pain, fatigue and back pain.