糖PEG化-exei_Esperoct_糖PEG化-exei粉末预填充注射溶液antihemophilic说明书

  [caption id="attachment_35888" align="alignleft" width="300"] Esperoct Kit 500IU/4mL（抗血友病因子[重组]，糖PEG化-exei粉末预填充注射溶液）[/caption]药店国别： 产地国家：美国 处 方 药：是 所属类别：500单位/4毫升/套件 包装规格：500单位/4毫升/套件 计价单位：套件 生产厂家中文参考译名:诺和诺德 生产厂家英文名:Novo Nordisk Inc 原产地英文商品名:Esperoct powder Prefilled solution 500IU/4mL/Kit 原产地英文药品名:antihemophilic factor[recombinant], glycopegylated-exei 中文参考商品译名:Esperoct粉末预填充注射溶液 500单位/4毫升/套件 中文参考药品译名:抗血友病因子[重组]，糖PEG化-exei 曾用名:N8-GP 简介： 近日，美国食品和药物管理局(FDA)已批准Esperoct(turoctocog alfa pegol，N8-GP)用于A型血友病成人及儿童患者，用于常规预防以减少出血发作频率、按需治疗控制出血发作、围手术期出血管理。 诺和诺德执行副总裁兼首席科学官Mads Krogsgaard Thomsen表示，“我们对Esperoct获得美国FDA批准感到非常高兴，该药将为A型血友病患者的治疗方案提供一个重要扩展。我们相信，Esperoct将为患者提供一种减轻负担、简单、固定剂量的血友病治疗方案，以预防和治疗出血事件，从而提高生活质量。” NovoEight是诺德诺德第三代重组凝血因子VIII产品turoctocog alpha的商品名，于2013年获FDA和欧盟批准，用于A型血友病患者的预防性治疗和按需治疗，该产品利用了最新的基因重组及蛋白纯化技术 批准日期：2019年2月21日 公司：诺和诺德 ESPEROCT(抗血友病因子[重组]，antihemophilic factor (recombinant), glycopegylated-exe)冻干粉末溶液，用于静脉注射 美国最初批准：2019年 作用机制: ESPEROCT是一种糖基化形式的重组抗血友病因子，暂时取代先天性甲型血友病患者有效止血所需的缺失凝血因子VIII。ESPEROCT中的因子VIII与40-kDa聚乙二醇分子共轭，与非聚乙二醇化分子相比，其增加半衰期并降低清除率。 适应症和用法: ESPEROCT(抗血友病因子[重组]，glycopegylated-exei)是凝固因子VIII浓缩物，适用于成人和儿童 患有血友病A： •按需治疗和控制出血事件 •围手术期出血管理 •常规预防，以减少出血发作的频率。 ESPEROCT不适用于治疗冯维勒布兰德病。 剂量和给药: 仅用于重建后的静脉输液 •ESPEROCT的每个样品瓶标签都以国际单位(IU)表示实际的因子VIII活动。 •按需治疗/控制出血事件：青少年/成人，轻度/中度出血40IU/kg体重，主要出血体重50 IU/kg体重;儿童(<12岁)，65IU/kg体重轻微/中度/大出血。 •围手术期处理：对于轻微/大手术：青少年/成人：术前剂量为50IU/kg体重;儿童(<12岁)，术前剂量为65 IU/kg体重。给药频率由主治医师确定。 •常规预防：青少年/成人，每4天50 IU /kg;英寸(<12岁)，每周两次65IU/kg。可以基于出血样本将治疗方案单独调整至更少或更频繁的剂量。 •根据血友病的严重程度，ESPEROCT也可以根据血友病的严重程度，按需治疗/控制出血事件或围手术期处理来达到特定目标因子VIII活性水平。要达到特定目标因子VIII活性水平，请使用以下公式： 剂量(IU)=体重(kg)×期望的因子VIII增加(IU/dL或%正常)×0.5(IU/kg/IU/dL)。 剂量形式和强度: ESPEROCT可作为单剂量小瓶中的冻干粉末，剂量为500,1000,1500,2000和3000IU/瓶。 禁忌症: 不要用于已知对ESPEROCT或其组分过敏的患者，包括仓鼠蛋白。 警告和注意事项: •可能发生过敏反应，包括过敏反应。如果发生过敏反应，则停止ESPEROCT并给予适当的治疗。 •已发生中和抗体(抑制剂)的开发。如果用ESPEROCT的推荐剂量不控制出血，或者如果未达到预期的血浆因子VIII活性水平，则进行测量因子VIII抑制剂浓度的测定。 不良反应: 最常报告的不良反应(≥1%)是皮疹，发红，瘙痒和注射部位反应。 儿科用药：儿童可以获得更高的清除率(基于公斤体重)，更短的半衰期和更低的增量恢复。可能需要更高和更频繁的剂量。 包装提供/存储和处理: 如何提供: •ESPEROCT以包含名义上500,1000,1500,2000或3000IU因子VIII活性的单剂量小瓶组成的包装提供; 含有0.9%盐水溶液的MixPro®预填充稀释剂注射器; 以及具有25微米过滤器的无菌适配器，其用作无针重构装置。 •每个ESPEROCT纸箱和样品瓶标签上都标明了IU中的实际因子VIII活性。 剂量强度 帽颜色指示符 NDC： 500IU 红色 NDC：0169850001 •ESPEROCT单剂量小瓶[NDC 0169 8501 11] •预填充注射器，含4mL无菌盐水稀释剂[NDC 0169 8008 98] •样品瓶适配器 1000IU 绿色 NDC：0169810001 •ESPEROCT单剂量小瓶[NDC 0169 8101 11] •预填充注射器，含4mL无菌盐水稀释剂[NDC 0169 8008 98] •样品瓶适配器 1500IU 灰色 NDC：0169 8150 01 •ESPEROCT单剂量小瓶[NDC 0169 8151 11] •预填充注射器，含4mL无菌盐水稀释剂[NDC 0169 8008 98] •样品瓶适配器 2000IU 黄色 NDC：0169 8200 01 •ESPEROCT单剂量小瓶[NDC 0169 8201 11] •预填充注射器，含4 mL无菌盐水稀释剂[NDC 0169 8008 98] •样品瓶适配器 3000IU 黑色 NDC：0169 8300 01 •ESPEROCT单剂量小瓶[NDC 0169 8301 11] •预填充注射器，含4 mL无菌盐水稀释剂[NDC 0169 8008 98] •样品瓶适配器 IU =国际单位 •ESPEROCT样品瓶由玻璃制成，用氯丁橡胶塞(不是用天然橡胶乳胶制成)封闭，并用铝盖密封。 •预填充稀释剂注射器由玻璃制成，带有硅化溴化丁基橡胶柱塞(不是用橡胶板制成)。 •封闭式样品瓶和预装稀释剂注射器配有防篡改的折叠帽，由聚丙烯制成。 存储和处理: •将ESPEROCT存放在原始包装中，以保护ESPEROCT样品瓶免受光照。 •在制造日期至标签上注明的失效日期之前，将制备的ESPEROCT粉末状态保存在36°F至46°F(2°C至8°C)，最长30个月。 •ESPEROCT可在30个月的时间内在室温[不超过86°F(30°C)]下储存长达12个月。将产品从冰箱中取出时，将日期记录在纸箱上。在室温下储存的总时间不应超过12个月。室温保存后，请勿将产品放回冰箱。 •请勿在小瓶上印刷的失效日期之后或在从冷藏中取出之日起12个月后使用ESPEROCT(以较早者为准)。 •不要冻结ESPEROCT。 •在室温下储存后，在重建后4小时内使用ESPEROCT，或者在冰箱中储存后24小时内使用ESPEROCT。将重构的产品储存在小瓶中。 •重新产品必须在室温下放置4小时后丢弃，或在冰箱中放置24小时后丢弃。 Proper Name: Antihemophilic Factor (Recombinant), GlycoPEGylated-exei Tradename: ESPEROCT Manufacturer: Novo Nordisk, Inc. Indication: For use in adults and children with hemophilia A for: (1) on-demand treatment and control of bleeding episodes, (2) perioperative management of bleeding, and (3) routine prophylaxis to reduce the frequency of bleeding episodes. US Food and Drug Administration (FDA) and a Marketing Authorisation Application (MAA) to the European Medicines Agency (EMA) for N8-GP, an extended half-life factor VIII for treatment of people with haemophilia A. The submission is based on results from the pathfinder clinical trial programme. The pathfinder programme included more than 250 people with haemophilia A and investigated efficacy and safety of N8-GP in adults and children as well as people undergoing surgery. In the trial, adults treated prophylactically with N8-GP every fourth day experienced a median annualised bleeding rate of 1.3 episodes compared to 30.9 episodes for people treated on-demand. Paediatric participants experienced a median annualised bleeding rate of 1.95 episodes when administered twice weekly. In the surgery trial, all surgeries were effectively performed with N8-GP, and clinical efficacy eva luated by haemostatic response was reported as 'excellent' or 'good' in 43 out of the 45 performed surgeries. Across the pathfinder clinical trial programme, N8-GP demonstrated a safe and well-tolerated profile. "We are excited to file the regulatory application for long-acting N8-GP in the US and EU," said Mads Krogsgaard Thomsen, executive vice president and chief science officer of Novo Nordisk. "Based on the results from the global pathfinder clinical trial programme, we believe N8-GP can reduce the burden of treatment by decreasing the number of intravenous infusions while achieving the benefits in terms of efficacy and safety for people with haemophilia A." About N8-GP N8-GP (turoctocog alfa pegol) is a glycopegylated form of turoctocog alfa designed for prolonged half-life. The site specific glycopegylation is within the truncated B-domain. N8-GP is a B-domain modified form of turoctocog alfa and hence the active factor VIII generated by thrombin activation is identical to both activated endogenous FVIII and turoctocog alfa. About the pathfinder phase 3a clinical trial programme The pathfinder programme included more than 250people with haemophilia A and investigated safety and efficacy of N8-GP, treatment of bleeds, prophylaxis and surgery, in adults as well as children. Pathfinder was a global trial eva luating safety and efficacy of N8-GP, when administered for prophylaxis and on-demand treatment in people with haemophilia A, who are 12 years or older. 175 people were treated with a prophylactic regimen of 50 IU/kg every fourth day, and 12 people received on-demand treatment. People were treated for up to 21 months, resulting in median annualised bleeding rates of 1.3 and 30.9 episodes for people treated prophylactically and on-demand, respectively. The pharmacokinetic data documented a single dose half-life of 18.4 hours and a mean trough level of 4% measured immediately before next dose for people on prophylaxis treatment. Pathfinder was a surgery trial which was performed as an open-label, multi-national trial eva luating the efficacy and safety of N8-GP when administered for perioperative management in previously treated patients with severe haemophilia A who were 12 years or older. The trial included 33 people who underwent 45 major surgeries. People received a single preoperative dose of N8-GP, at a median of 51 IU/kg. All surgeries were effectively performed with N8-GP, and clinical efficacy eva luated by haemostatic response was reported as 'excellent' or 'good' in 43 out of the 45 performed surgeries. N8-GP appeared to have a safe and well-tolerated profile. Pathfinder was a paediatric trial which was performed as a global trial eva luating safety and efficacy for N8-GP, when administered for prophylaxis in previously treated children with haemophilia A, who were between 0 to 11 years old. In the trial, 34 children 0-5 years of age and 34 children 6-11 years of age received prophylactic treatment and treatment of bleeding episodes. All children were treated with a regimen of 50-75 IU/kg twice weekly for 26 weeks. Median annualised bleeding rates of 1.95 episodes were obtained in the trial. N8-GP appeared to have a safe and well-tolerated profile.