In this study, researchers will observe and study the data from participants with hemophilia A who receive damoctocog alfa pegol as prescribed by their doctors. Participants will not receive any advice or changes to their healthcare during the study.
Hemophilia A is a genetic bleeding disorder. It is caused by the lack of a protein called clotting factor 8 (FVIII) that helps blood to clot properly. Lack of FVIII can result in excessive blood loss or bleeding inside the body after being injured or having surgery.
The study drug, damoctocog alfa pegol, can be used to prevent or treat bleeding episodes by replacing missing FVIII in the body of people with hemophilia A. It is already approved for people with hemophilia A who are at least 12 years old and have previously used other hemophilia A treatments.
Through this study, researchers want to learn more about its safety in a real-world setting.
The participants will receive damoctocog alfa pegol as prescribed by their doctors during routine practice according to the approved product information.
The main purpose of this study is to learn more about how safe damoctocog alfa pegol is in Korean participants with hemophilia A who previously used other hemophilia A treatments. To do this, researchers will collect information about any medical problems participants have during their treatment.
Data will be collected from December 2023 to March 2026 and cover a period of about 8 months for each participant. Data will come from participants' health records and information collected during their routine clinic visits.
In this study, only available data from routine care will be collected. No visits or tests are required as part of this study.

开始日期2024-01-24

申办/合作机构

Bayer AG

NCT05643560

/ RecruitingN/A

Long-term Treatment Outcome of Jivi® Prophylaxis on Joint Health in Adult Patients With Hemophilia A

This is an observational study in which data from people with hemophilia A who decide on their own or by recommendation of their doctors to take Jivi are collected and studied. In observational studies, only observations are made without specified advice or interventions.
Hemophilia A is a genetic bleeding disorder that is caused by the lack of a protein in the blood called "clotting factor 8" (FVIII). FVIII is naturally found in the blood where it causes the blood to clump together to help prevent and stop bleeding. People with lower levels of FVIII or with FVIII that does not work properly may bleed for a long time from minor wounds, have painful bleeding into joints, or have internal bleeding.
The study treatment, Jivi (also called damoctocog alfa pegol), is already available for doctors to prescribe to people with hemophilia A to treat and prevent bleeding. It works by replacing the missing FVIII, or the FVIII that does not work properly.
People with hemophilia A need frequent injections of FVIII products into the vein. So called standard half-life (SHL) products need to be given 2 to 4 times a week for the prevention of bleeding. In recent years, new products like Jivi called extended half-life (EHL) products have available. These products last longer in the body so that they require to be given less often with injections up to every 7 days. Thus, these treatments may be easier and more comfortable to stick to in daily life. There is no general plan concerning the best amount of treatment and the frequency of injections for the prevention of bleeding, since the severity may be different and individual risk factors have to be considered. Doctors often decide on a treatment plan based on patient's disease and response.
Clinical studies have already shown that people with hemophilia A benefit from the treatment with Jivi. However, there are no data available coming from the real-world about how well Jivi works to support joint health, measured by ultrasound (US) examination and HEAD-US score.
In this study, researchers want to learn more about how well Jivi works if used for prolonged periods of treatment under real-world settings to prevent problems with joints in people with hemophilia A. How well it works means to find out if participants' joints status can be improved by treatment with Jivi.
To do this, researchers will collect data about participants' joints status by
* making images of participants' joints by using sound waves (ultrasound), and
* using HEAD-US score after 24 months of treatment with Jivi. The researchers will then compare these data to the participants' joints status before treatment start with Jivi.
Besides this data collection, no further tests or examinations are planned in this study.
Some participants in this study will already be receiving treatment with Jivi as part of their regular care no more than 12 months. And some participants will start to take Jivi in this study as prescribed by their doctors during routine practice according to the approved product information.
The researchers will collect data from each patient for a period of 26 months after initiation of the Jivi treatment.
There are no required visits or tests in this study

开始日期2022-12-29

申办/合作机构

Bayer AG

NCT05036278

/ Completed临床4期

A Multicenter, Prospective, Open-label, Clinical Study to Assess the Effect of Using a New Risk Score Approach to Select the Most Appropriate Prophylaxis Regimen for Reaching a Favorable Outcome, When Hemophilia A Patients Switch From Standard Half-life Products to Damoctocog Alfa Pegol (Jivi)

Researchers are looking for a better way to treat people who have hemophilia A. Hemophilia A is a genetic bleeding disorder that is caused by the lack of a protein in the blood called "clotting factor 8" (FVIII). FVIII is naturally found in the blood where it causes the blood to clump together to help prevent and stop bleeding. People with lower levels of FVIII or with FVIII that does not work properly may bleed for a long time from minor wounds, have painful bleeding into joints, or have internal bleeding.
The study treatment, Jivi (also called damoctocog alfa pegol), is already available for doctors to prescribe to people with hemophilia A to treat and prevent bleeding. It works by replacing the missing FVIII, or the FVIII that does not work properly.
People with hemophilia A need frequent injections of FVIII products into the vein. So called standard half-life (SHL) products need to be given 2 to 4 times a week for the prevention of bleeding. In recent years, new products like Jivi called extended half-life (EHL) products have become available. These products last longer in the body so that they require to be given less often with injections every 3-5 days. Thus, these treatments may be easier and more comfortable to stick to in daily life. There is no general plan concerning the best amount of treatment and the frequency of injections for the prevention of bleeding, since the severity may be different and individual risk factors have to be considered. Doctors often decide on a treatment plan based on their experience.
The main purpose of this study is to learn how well a new scoring approach works to select a treatment plan for the prevention of bleeding in people with hemophilia A who switch their treatment from SHL products to Jivi. Different types of information are used to calculate the risk score like bleeding history, certain biological factors, and physical activity of the participant.
All participants will receive Jivi for 6 months.
In the first four weeks, all participants will receive Jivi 2 times a week at a dose level of 40 IU per kilogram body weight (also known as 40 IU/kg/dose, recommended maximum dose is 6,000 IU). Then, based on their risk score, each participant will be assigned to one of three treatment plans:
* participants with a high risk remain on Jivi administration 2 times a week at 40 IU/kg/dose
* participants with a medium risk will switch to Jivi administration every 5 days at 50 IU/kg/dose
* participants with a low risk will switch to Jivi administration every 5 days at 50 IU/kg/dose and after 4 weeks to a less frequent administration (e.g., every 7 days) at 60 IU/kg/dose To check how well the new scoring approach works for choosing the right treatment plan, researchers will look at how many participants have a favourable outcome.
This means that the participant has either fewer bleeding events vs. the pre-study treatment and takes Jivi less often or as often as the previous SHL treatment but with fewer bleeding events, or that the participant has a comparable number of bleeding events but needs to take Jivi less often than the previous treatment. Each participant will be in the study for approximately 7.5 months. During this time, 4 visits to the study site and 3 phone calls are planned. During the study, the doctors and their study team will: • do physical examinations • take blood samples • ask the participants questions about how they are feeling and what adverse events they are having. In addition, participants or their guardians are required to write down the dates of Jivi treatments and bleeding events in an electronic diary and to fill in different questionnaires on their quality of life, health status, work/ school productivity, pain, and treatment satisfaction. In addition, participants are expected to keep appointments for visits and to adhere to the assigned treatment regimen.
An adverse event is any medical problem that a participant has during a study. Doctors keep track of all adverse events that happen in studies, even if they do not think the adverse events might be related to the study treatments.

开始日期2022-07-28

申办/合作机构

Bayer AG

100 项与 Damoctocog Alfa Pegol 相关的临床结果

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100 项与 Damoctocog Alfa Pegol 相关的转化医学

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100 项与 Damoctocog Alfa Pegol 相关的专利（医药）

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项与 Damoctocog Alfa Pegol 相关的文献（医药）

2025-05-01·THROMBOSIS RESEARCH

FVIII half-life products: A real-world experience

Article

作者: Napolitano, Angela ; Simion, Chiara ; Simioni, Paolo ; Zanon, Ezio ; Porreca, Annamaria

BACKGROUND:

Haemophilia A is a genetic coagulation disorder requiring prophylactic Factor VIII (FVIII) treatment to prevent joint damage and enhance quality of life. The introduction of extended half-life (EHL) FVIII products represents a major advancement, addressing patient needs for reduced infusion frequency, improved efficacy, and enhanced safety.

METHODS:

This single-centre observational study examined the real-world use of FVIII products among 124 male patients treated at the Haemophilia Centre of Padua from January 2018 to December 2023. Data on patient characteristics, treatment regimens, and pharmacokinetic profiles were analyzed.

RESULTS:

Among the FVIII products used, Advate®, Elocta®, and Jivi® showed median half-lives of 11.25 [10.75-12.25], 16.50 [13.75-17.75], and 15.38 [13.38-18.63] hours, respectively. Esperoct® exhibited the longest half-life at 19.75 [16.00-24.50] hours, enabling reduced infusion frequency. EHL products showed significantly lower weekly infusion rates compared to standard half-life (SHL) products (1.4 [1.0-2.0] vs. 2.0 [2.0-3.0], p < 0.001), with comparable bleeding control among the different FVIII-EHL.

CONCLUSIONS:

EHL FVIII products offer significant clinical benefits, reducing the burden of frequent infusions and improving adherence while maintaining effective bleeding control. Despite these advancements, comprehensive evaluations of cost, safety, and long-term outcomes are essential to optimize their integration into haemophilia care.

2025-03-01·EUROPEAN JOURNAL OF HAEMATOLOGY

Effectiveness of Damoctocog Alfa Pegol to Treat Patients With Hemophilia A Enrolled in the ATHNdataset

Article

作者: Recht, Michael ; Charlet, Jessica ; Moulton, Thomas ; Charafi, Lena ; Chandler, Martin

ABSTRACT:

Objectives:

Health information for 17 109 people living with hemophilia A (PLwHA) is contained within the ATHNdataset. We aimed to evaluate the real‐world effectiveness of damoctocog alfa pegol (BAY 94‐9027, Jivi®) for hemophilia A.

Methods:

The ATHNdataset was queried for PLwHA receiving damoctocog alfa pegol between January 1, 2010 and April 30, 2022. Data captured via patient charts were analyzed.

Results:

At data cutoff, 205 PLwHA were treated with damoctocog alfa pegol: 150 (73.2%) severe (1 female [0.5%]) and 55 (26.8%) mild/moderate (3 [1.5%] female). In total, 32/205 (25.9%) PLwHA received on‐demand treatment; 172 (83.9%) received prophylaxis—161 (93.6%) continuous prophylaxis. Documented bleed rates were available for 187 (91.2%) PLwHA, including those on prophylaxis and on‐demand regimens, with 150 (80.2%) treated for > 12 months. Overall annualized bleeding rates and proportion of PLwHA with zero bleeds, receiving prophylaxis during the observation period, were mean (SD) 0.26 (1.03) and 138/157 (87.9%), respectively. No new or recurring inhibitors were reported.

Conclusion:

A low number of bleeds were observed with damoctocog alfa pegol in the real world in both male and female PLwHA. Data should be interpreted with caution owing to limitations of real‐world studies and insubstantial data for female PLwHA.

2025-03-01·DRUGS IN R&D

Correction: Damoctocog Alfa Pegol, a PEGylated B-domain Deleted Recombinant Extended Half-life Factor VIII for the Treatment of Hemophilia A: A Product Review

作者: Reding, Mark T ; Ducore, Jonathan ; Mancuso, Maria Elisa ; Meijer, Karina ; Poulsen, Lone H ; Di Minno, Giovanni ; Simpson, Mindy ; Shah, Anita ; Lalezari, Shadan ; Holme, Pål André ; Solms, Alexander ; Kenet, Gili

项与 Damoctocog Alfa Pegol 相关的新闻（医药）

2025-05-20

·药明康德

约60亿美元！辉瑞囊获PD-1/VEGF靶向双抗；首个！只需每季一次，潜在“best-in-class”单抗亮眼结果公布……

拜耳血友病疗法再获FDA批准日前，拜耳（Bayer）公司宣布，美国FDA批准Jivi（重组DNA衍生的长效凝血因子VIII浓缩物）用于7岁及以上血友病A（先天性凝血因子VIII缺乏症）儿科患者。Jivi（BAY94-9027）是一种通过聚乙二醇修饰延长半衰期的重组凝血因子VIII，在临床试验中它的半衰期达到了17.9个小时，是对照组的1.4倍。该疗法在2018年获FDA批准用于常规预防性治疗接受过前期治疗的12岁以上血友病A患者。而此次批准则基于Alfa-PROTECT和PROTECT Kids研究的数据，这些研究证明了Jivi在7至12岁以下重度血友病A儿童中的安全性和有效性。约60亿美元！辉瑞囊获PD-1/VEGF靶向双抗辉瑞公司（Pfizer）日前宣布已与三生制药（3SBio）签署一项全球（不包括中国内地）的独家许可协议，用于开发、生产和商业化SSGJ-707。这是一款双特异性抗体，靶向PD-1和VEGF，当前正在中国开展多个临床试验，适应症包括非小细胞肺癌、转移性结直肠癌和妇科肿瘤。SSGJ-707已在临床试验中展现出初步的疗效和安全性数据。三生制药计划于2025年在中国启动首个3期临床试验。根据协议条款，三生制药及其子公司将向辉瑞授予SSGJ-707在全球（不包括中国内地）范围内的开发、生产和商业化的独家许可权。该协议还赋予辉瑞在中国获得商业化权利的选择权。三生制药将获得12.5亿美元的预付款，并有资格获得与特定开发、监管和商业里程碑相关最高达48亿美元的里程碑付款。首个！只需每季一次，潜在“best-in-class”单抗亮眼结果公布Tourmaline Bio公司今日宣布，其进行中的2期TRANQUILITY临床试验取得积极的顶线结果。该试验评估其在研单抗pacibekitug以每季一次或每月一次皮下注射的方式，用于治疗高敏C反应蛋白（hs-CRP）水平升高的3级或4级慢性肾病（CKD）患者的效果。研究显示，与安慰剂相比，所有pacibekitug治疗组在第90天均实现了快速、显著且持久的hs-CRP水平降低，具有高度统计学意义（所有治疗组p<0.0001）。其中，每季一次50 mg给药组的hs-CRP平均降低幅度超过85%。根据新闻稿，pacibekitug为在临床试验中首个在每季给药频率下可显著降低hs-CRP水平的IL-6抑制剂。TRANQUILITY研究结果支持将pacibekitug推进至潜在针对动脉粥样硬化性心血管疾病（ASCVD）开展的3期心血管结局临床试验，并开展针对腹主动脉瘤（AAA）患者的2期概念验证试验。Pacibekitug是一种作用持久的全人源抗IL-6单克隆抗体，为潜在“best-in-class”疗法，其特点包括天然长半衰期、低免疫原性和对IL-6的高结合亲和力。除正在进行的试验外，pacibekitug此前已在约450名受试者（包括患有自身免疫性疾病的患者）中开展过6项已完成的临床试验。Tourmaline目前正在评估pacibekitug用于ASCVD和甲状腺相关眼病（TED）患者的作用。▲接受pacibekitug治疗患者在第90天实现显著hs-CRP水平降低（图片来源：参考资料[3]）参考资料：[1] U.S. FDA Grants Approval for Jivi® Antihemophilic Factor (Recombinant), PEGylated-aucl in Pediatric Patients 7 to Under 12 Years of Age With Hemophilia A (Congenital Factor VIII Deficiency). Retrieved May 20, 2025 from https://www.businesswire.com/news/home/20250519742201/en/U.S.-FDA-Grants-Approval-for-Jivi-Antihemophilic-Factor-Recombinant-PEGylated-aucl-in-Pediatric-Patients-7-to-Under-12-Years-of-Age-With-Hemophilia-A-Congenital-Factor-VIII-Deficiency[2] Pfizer Enters into Exclusive Licensing Agreement with 3SBio. Retrieved May 20, 2025 from https://investors.pfizer.com/Investors/News/news-details/2025/Pfizer-Enters-into-Exclusive-Licensing-Agreement-with-3SBio/default.aspx[3] Tourmaline Bio Announces Positive Topline Results from the Ongoing Phase 2 TRANQUILITY Trial Evaluating Pacibekitug in Patients with Elevated High-Sensitivity C-reactive Protein and Chronic Kidney Disease. Retrieved May 20, 2025 from https://www.globenewswire.com/news-release/2025/05/20/3084742/0/en/Tourmaline-Bio-Announces-Positive-Topline-Results-from-the-Ongoing-Phase-2-TRANQUILITY-Trial-Evaluating-Pacibekitug-in-Patients-with-Elevated-High-Sensitivity-C-reactive-Protein-an.html免责声明：本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。版权说明：欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转载至其他平台。转载授权请在「药明康德」微信公众号回复“转载”，获取转载须知。👇 分享，点赞，在看，聚焦全球生物医药健康创新

临床3期临床2期引进/卖出临床成功临床结果

2025-05-20

·GBIHealth

逾60亿美元！辉瑞获得三生制药双抗SSGJ-707独家许可

药械追踪No.1 / GSK玛贝兰妥单抗在日本获批用于多发性骨髓瘤2025年5月19日，葛兰素史克（LSE/NYSE：GSK）宣布，其产品Blenrep（玛贝兰妥单抗）联合疗法已获得日本厚生劳动省（MHLW）批准上市，用于治疗复发或难治性多发性骨髓瘤成人患者。该批准基于DREAMM-7和DREAMM-8两项Ⅲ期临床试验的积极结果。这些试验分别评估了Blenrep联合硼替佐米加地塞米松（BVd）以及联合泊马度胺加地塞米松（BPd）的疗效，研究对象为至少接受过一次既往治疗的多发性骨髓瘤患者。Blenrep是一种抗体偶联药物（ADC），由人源化BCMA单克隆抗体与细胞毒性药物甲硫氨酸通过不可切割的连接子结合而成。该药物连接子技术授权自Seagen公司；单克隆抗体则使用了BioWa公司（Kyowa Kirin集团成员）授权的POTELLIGENT技术生产。2025年4月，英国药品和健康产品管理局（MHRA）批准了Blenrep联合疗法，用于治疗至少接受过一次既往治疗的复发或难治性多发性骨髓瘤成人患者。->点击文末阅读原文，解锁完整双语新闻No.2 / 拜耳Jivi获FDA批准拓展适应证至7-12岁A型血友病儿童患者2025年5月20日，拜耳宣布，Jivi已获美国食品药品监督管理局（FDA）批准用于7岁及以上患有A型血友病（先天性凝血因子VIII缺乏症）的儿童患者。Jivi是一种通过重组DNA技术制备的长效凝血因子VIII浓缩剂，此次获批基于Alfa-PROTECT和PROTECT Kids研究的数据。两项研究证实，Jivi在7至12岁严重A型血友病儿童中具有良好的安全性和有效性。此前，Jivi于2018年8月首次获得FDA批准，用于12岁及以上已接受过治疗的A型血友病患者，包括按需治疗和控制出血发作、围手术期出血管理、常规预防以减少出血发作频率。->点击文末阅读原文，解锁完整双语新闻企业动态No.1 / 逾60亿美元！辉瑞获得三生制药双抗SSGJ-707独家许可2025年5月20日，辉瑞（NYSE:PFE）与三生制药集团（1530.HK）达成全球独家许可协议，辉瑞将获得其子公司三生国健（688336.SH）研发的PD-1/VEGF双特异性抗体SSGJ-707在除中国大陆以外的全球独家开发、生产和商业化权利。根据协议条款，三生制药有望获得12.5亿美元首付款，并有资格获得最高48亿美元的开发、监管批准和销售里程碑付款，以及根据授权地区产品销售额计算得到的两位数百分比的梯度销售分成。预计该交易将于第三季度完成，交易完成后，辉瑞将根据双方达成的证券认购协议，向三生制药进行1亿美元的股权投资。SSGJ-707是一款靶向PD-1和VEGF的双特异性抗体，目前正在中国进行多项临床试验，用于治疗非小细胞肺癌、转移性结直肠癌和妇科肿瘤。无论单药还是与化疗联用，SSGJ-707均展示出显著的抗肿瘤活性和良好的安全性，具有同类最优的潜力。目前，SSGJ-707单药一线治疗非小细胞肺癌适应证已进入Ⅲ期临床阶段，联合化疗一线治疗非小细胞肺癌适应证处于临床Ⅱ期阶段、转移性结直肠癌适应证处于临床Ⅱ期阶段，晚期妇科肿瘤适应证处于临床Ⅱ期阶段。->点击文末阅读原文，解锁完整双语新闻No.2 / 8.95亿美元！靖因药业与CRISPR Therapeutics达成siRNA开发合作2025年5月20日，靖因药业与CRISPR Therapeutics（Nasdaq：CRSP）达成战略合作，将共同开发并商业化下一代长效Factor Ⅺ（FⅪ）靶向小干扰RNA（siRNA）疗法SRSD107，用于治疗血栓及血栓栓塞性疾病。根据协议条款，靖因药业将获得CRISPR Therapeutics支付的9500万美元现金及现金等价物作为首付款，并有资格获得超8亿美元的预付款和里程碑付款。双方将对半分摊成本和利润，共同开发SRSD107。此外，协议还授予CRISPR Therapeutics在未来独家引进最多两个额外siRNA项目的优先授权权利。CRISPR将负责该产品在美国的商业化，靖因药业则负责大中华区市场。SRSD107是一款新一代的长效siRNA疗法，旨在选择性抑制凝血因子Ⅺ（FⅪ）。FⅪ靶点在病理性血栓形成中起关键作用，但对正常止血功能的影响较小。通过靶向FⅪ，SRSD107有望在降低血栓事件发生的同时，显著减少出血风险，展现出有别与凝血因子Ⅹ活性（FⅩa）抑制剂的治疗优势。SRSD107的潜在适应证广泛，包括房颤、静脉血栓栓塞症（VTE）、肿瘤相关性血栓、接受血液透析的终末期肾病患者，以及因出血风险限制现有治疗手段的重大骨科手术人群。目前，SRSD107已完成两项Ⅰ期临床试验，结果显示单次给药安全性且耐受性良好。相关研究成果已在2025年美国心脏病学会（ACC）年会和2024年美国血液学会（ASH）年会上发布。SRSD107的Ⅱ期临床试验正在启动，旨在评估其在膝关节置换术患者中预防静脉血栓栓塞症（VTE）的安全性和有效性。->点击文末阅读原文，解锁完整双语新闻No.3 / 2.56亿美元！再生元收购23andMe资产，Lemonaid Health业务除外2025年5月19日，再生元（Nasdaq：REGN）宣布，已成功中标23andMe Holding Co.（Nasdaq：ME）的破产拍卖，将收购该公司几乎所有资产。再生元计划以2.56亿美元的价格收购23andMe的个人基因组服务（PGS）、全面健康和研究服务业务线，以及其生物样本库及相关资产，并确保23andMe的所有消费级基因组服务不间断地继续开展。该交易预计将在2025年第三季度完成，但需获得破产法院和监管机构的批准以及其他惯例的交割条件。23andMe将作为再生元的全资直接或间接子公司运营，并继续作为个人基因组服务提供商开展业务。Regeneron的收购不包括23andMe的Lemonaid Health业务。Lemonaid Health是一个在线按需获取医疗和药房服务的虚拟平台。2021年10月22日，23andMe宣布与Lemonaid Health, Inc.达成最终合并协议，以4亿美元收购Lemonaid Health，为23andMe的消费者业务增加了Lemonaid Health的创新远程医疗和处方药配送服务。2025年1月，据媒体报道，23andMe正在考虑出售Lemonaid Health业务。23andMe成立于2006年，2021年上市，市值巅峰曾达到60亿美元。成立至今，23andMe从未盈利过，截至2024年12月31日，23andMe的现金和现金等价物只剩7940万美元。2025年3月，23andMe申请破产保护，此前曾尝试私有化和寻求收购，但都未能成功。->点击文末阅读原文，解锁完整双语新闻No.4 / 默沙东/第一三共在研ADC I-DXd启动食管鳞癌III期临试2025年5月20日，默沙东（NYSE：MRK）与合作伙伴第一三共（TSE：4568）联合开发的潜在同类首创B7-H3靶向抗体偶联药物（ADC）Ifinatamab Deruxtecan（简称I-DXd）已启动III期IDeate-Esophageal01研究。IDeate-Esophageal01研究旨在评估I-DXd对比研究者选择的化疗方案，在含铂方案和免疫检查点抑制剂经治后疾病进展的不可切除晚期或转移性食管鳞状细胞癌（ESCC）患者中的疗效与安全性。目前，该研究已完成首例患者给药。IDeate-Esophageal01研究的启动基于I/II期IDeate-PanTumor01研究的积极数据，IDeate-PanTumor01显示，I-DXd在多线经治的ESCC患者中具有显著疗效。->点击文末阅读原文，解锁完整双语新闻全球医疗情报领导者解锁隐藏在数据中的商业潜力关于 G B I”自从2002年成立以来，GBI始终以技术为驱动，为药企、器械及行业相关服务商提供贯穿生命周期的全球药品市场竞争数据、全球行业资讯、HCPs洞察、全国医疗器械数据等商业信息与洞察，助力企业在进行战略布局和决策时，脱颖而出。历经20余年的深耕细作GBI已成为95%以上跨国药企、国内头部药企、咨询与投资机构等医疗圈灯塔用户值得信赖的长期合作伙伴。联系我们投稿 | 发稿 | 媒体合作▶ olivia.xu@generalbiologic.com数据库 | 咨询服务 | 资讯追踪▶ 点击左下“阅读原文”完成表单填写点击阅读原文，解锁完整双语新闻

引进/卖出临床3期抗体药物偶联物申请上市临床1期

2025-05-19

·Business Wire

U.S. FDA Grants Approval for Jivi ® Antihemophilic Factor (Recombinant), PEGylated-aucl in Pediatric Patients 7 to Under 12 Years of Age With Hemophilia A (Congenital Factor VIII Deficiency)

WHIPPANY, N.J.--(BUSINESS WIRE)--Bayer announced that the U.S. Food and Drug Administration (FDA) has approved Jivi®, a recombinant DNA-derived, extended half-life factor VIII concentrate, for use in pediatric patients 7 years of age and older with hemophilia A (congenital Factor VIII deficiency). The approval is based on data from the Alfa-PROTECT and PROTECT Kids studies, demonstrating Jivi’s safety and efficacy in children aged 7 to under 12 years with severe hemophilia A. This significant milestone reflects Bayer’s ongoing commitment to providing effective treatment options for the hemophilia community. Jivi was first granted approval by the FDA in August 2018 for use in previously treated adults 12 years of age and older with hemophilia A for: On-demand treatment and control of bleeding episodes Perioperative management of bleeding Routine prophylaxis to reduce the frequency of bleeding episodes. Jivi is not indicated for use in: Children <7 years of age due to a greater risk for hypersensitivity reactions and/or loss of efficacy. Previously untreated patients (PUPs). Treatment of von Willebrand disease. "Bayer remains dedicated to meeting the needs of the hemophilia A community," stated Jessica Charlet, Scientific Director. “The FDA’s approval for pediatric patients 7 to <12 years of age is reflective of Bayer’s commitment to the hemophilia A community and underscores our commitment to supporting families through their treatment journey.” Bayer continues to prioritize support for the hemophilia community, and we are excited to offer Jivi as a treatment option for patients and their families. About Jivi® [antihemophilic factor (recombinant), PEGylated-aucl] Jivi is approved for the routine prophylactic treatment of hemophilia A in previously treated adult and pediatric patients 7 years of age or older. Jivi’s initial recommended dosing for children 7 to under 12 years of age is 60 IU/kg twice weekly. Adjust the dose based on the patient’s clinical response and/or recovery. The recommended initial dosage regimen for adults and adolescents is (30-40 IU/kg) twice weekly with the ability to dose every five days (45-60 IU/kg) and further individually adjust to less or more frequent dosing based on bleeding episodes. Jivi is also approved for on-demand treatment and the perioperative management of bleeding in the same population.1 To learn more about Jivi, visit www.jivi.com. INDICATION JIVI ® is a recombinant DNA-derived, Factor VIII concentrate indicated for use in previously treated adults and pediatric patients 7 years of age and older with hemophilia A (congenital Factor VIII deficiency) for: On-demand treatment and control of bleeding episodes. Perioperative management of bleeding. Routine prophylaxis to reduce the frequency of bleeding episodes. On-demand treatment and control of bleeding episodes. Perioperative management of bleeding. Routine prophylaxis to reduce the frequency of bleeding episodes. Limitations of use JIVI is not indicated for use in: Children <7 years of age due to a greater risk for hypersensitivity reactions and/or loss of efficacy. Previously untreated patients (PUPs). Treatment of von Willebrand disease. Children <7 years of age due to a greater risk for hypersensitivity reactions and/or loss of efficacy. Previously untreated patients (PUPs). Treatment of von Willebrand disease. IMPORTANT SAFETY INFORMATION JIVI is contraindicated in patients who have a history of hypersensitivity reactions to the active substance, polyethylene glycol (PEG), mouse or hamster proteins, or other constituents of the product. Hypersensitivity reactions, including severe allergic reactions, have occurred with JIVI. Monitor patients for hypersensitivity symptoms. Early signs of hypersensitivity reactions, which can progress to anaphylaxis, may include chest or throat tightness, dizziness, mild hypotension and nausea. If hypersensitivity reactions occur, immediately discontinue administration and initiate appropriate treatment. JIVI may contain trace amounts of mouse and hamster proteins. Patients treated with this product may develop hypersensitivity to these non-human mammalian proteins. Hypersensitivity reactions may also be related to antibodies against polyethylene glycol (PEG). Neutralizing antibody (inhibitor) formation has occurred following administration of JIVI. Carefully monitor patients for development of Factor VIII inhibitors, using appropriate clinical observations and laboratory tests. If expected plasma Factor VIII activity levels are not attained or if bleeding is not controlled as expected with administered dose, suspect the presence of an inhibitor (neutralizing antibody). An immune response associated with IgM anti-PEG antibodies, manifested as symptoms of acute hypersensitivity and/or loss of drug effect, has occurred with JIVI administration. In the clinical trials, the IgM anti-PEG antibodies disappeared within 4-6 weeks. No immunoglobulin class switching from IgM to IgG has been observed. A low post-infusion Factor VIII level, in absence of detectable Factor VIII inhibitors, may be due to loss of treatment effect related to high titers of anti-PEG IgM antibodies. In these cases, discontinue JIVI and switch patients to a different anti-hemophilic product. A reduced recovery of Factor VIII after start of JIVI treatment may be due to transient low titers of anti-PEG IgM antibodies. In these cases, increase the dose of JIVI until recovery of Factor VIII returns to expected levels. The most common (incidence ≥5%) adverse reactions in clinical trials in previously treated patients (PTPs) ≥7 years of age were headache, fever, cough, and abdominal pain. For additional important risk and use information, please see the full Prescribing Information. About Alfa-PROTECT Study A multi-center, prospective, single-arm, study to evaluate the safety of Jivi infusions for prophylaxis and treatment of bleeding in previously treated pediatric patients 7 to <12 years of age with severe hemophilia A. The study evaluated the potential risk of hypersensitivity and loss of drug effect associated with an immune response to polyethylene glycol (PEG) during the first 4 exposure days to Jivi. A total of 35 patients with a median age of 8 years (range: 7 to 11) were enrolled and treated prophylactically for a minimum of 50 EDs and 26 weeks. The patients received a treatment regimen of Jivi twice weekly (40-60 IU/kg) at investigator’s discretion. Thirty-two patients completed the treatment phase and were offered continuation in an 18 month extension study. A key secondary endpoint was the annualized bleed rate (ABR) About PROTECT Kids Study A multi-center, prospective, single-arm trial to evaluate the pharmacokinetics, safety, and efficacy of Jivi for prophylaxis and treatment of bleeding in previously treated pediatric patients <12 years of age with severe hemophilia A. The primary efficacy endpoint was the annualized bleed rate (ABR). About Hemophilia A Hemophilia affects approximately 400,000 people around the world and is a largely inherited disorder in which one of the proteins needed to form blood clots is missing or reduced. Hemophilia A is the most common type of hemophilia, in which blood clotting is impaired because there is a lack or defect of coagulation FVIII. Patients repeatedly experience bleeds in muscles, joints or other tissues, which can result in chronic joint damage over time. Injuries can have severe consequences if not treated appropriately, as the blood clots more slowly in hemophilia patients than in healthy individuals. Based on a study that used data collected on patients receiving care in federally funded hemophilia treatment centers during the period 2012–2018, about 33,000 males in the United States are living with the disorder.2 About Bayer Bayer is a global enterprise with core competencies in the life science fields of health care and nutrition. In line with its mission, “Health for all, Hunger for none,” the company’s products and services are designed to help people and the planet thrive by supporting efforts to master the major challenges presented by a growing and aging global population. Bayer is committed to driving sustainable development and generating a positive impact with its businesses. At the same time, the Group aims to increase its earning power and create value through innovation and growth. The Bayer brand stands for trust, reliability and quality throughout the world. In fiscal 2024, the Group employed around 93,000 people and had sales of 46.6 billion euros. R&D expenses amounted to 6.2 billion euros. © 2025 Bayer BAYER, the Bayer Cross and Jivi are registered trademarks of Bayer. Forward-Looking Statements This release may contain forward-looking statements based on current assumptions and forecasts made by Bayer management. Various known and unknown risks, uncertainties and other factors could lead to material differences between the actual future results, financial situation, development or performance of the company and the estimates given here. These factors include those discussed in Bayer’s public reports which are available on the Bayer website at www.bayer.com. The company assumes no liability whatsoever to update these forward-looking statements or to confirm them to future events or developments. References: 1. Jivi® [antihemophilic factor (recombinant), PEGylated-aucl] [Prescribing Information]. Whippany, NJ: Bayer 2025 2. Centers for Disease Control and Prevention. About Hemophilia. Last Reviewed: May 2025. https://www.cdc.gov/hemophilia/about/index.html PP-JIV-US-2365- 1 May 2025

上市批准临床结果

100 项与 Damoctocog Alfa Pegol 相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D10759	Damoctocog Alfa Pegol	B02BD02	DB14700

研发状态

批准上市

10 条最早获批的记录,

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适应症	国家/地区	公司	日期
血友病A	美国	Bayer Healthcare LLC	2018-08-29

未上市

10 条进展最快的记录,

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适应症	最高研发状态	国家/地区	公司	日期
出血	临床3期	美国	Bayer AG	2013-05-29
出血	临床3期	阿根廷	Bayer AG	2013-05-29
出血	临床3期	奥地利	Bayer AG	2013-05-29
出血	临床3期	比利时	Bayer AG	2013-05-29
出血	临床3期	保加利亚	Bayer AG	2013-05-29
出血	临床3期	加拿大	Bayer AG	2013-05-29
出血	临床3期	希腊	Bayer AG	2013-05-29
出血	临床3期	以色列	Bayer AG	2013-05-29
出血	临床3期	意大利	Bayer AG	2013-05-29
出血	临床3期	立陶宛	Bayer AG	2013-05-29

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临床结果

适应症

分期

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT04085458 (CTgov)	临床4期	血友病A	32	BAY94-9027	遞獵艱鑰襯網築襯糧衊 = 鏇選積淵獵鹽鏇鏇壓淵憲憲積製範製鏇壓壓觸 (廠憲願窪網積顧廠襯壓, 淵願窪糧築衊憲鑰範選 ~ 鹽鹹製製襯構鏇淵憲築) 更多	-	2023-06-28
ISTH2023	N/A	-	10	Damoctocog alfa pegol	廠製糧鹽願積憲繭積鏇(鏇鑰糧鬱衊窪範襯憲醖) = 鑰壓鏇鹹淵築餘壓襯構築範簾製壓願淵選衊繭 (鹹鹹糧觸糧觸鑰願廠願 ) 更多	-	2023-06-24
ISTH2023	N/A	血友病A	-	BAY 94-9027	範憲顧襯壓憲範齋鏇壓(鬱願鏇鏇艱窪鏇網構壓) = 醖餘憲餘鏇鹽鏇製壓醖蓋廠夢選膚積製簾夢觸 (選築淵淵膚糧蓋鏇齋範 )	积极	2023-06-24
ISTH2023	N/A	血友病A	-	BAY 81-8973	範憲顧襯壓憲範齋鏇壓(鬱願鏇鏇艱窪鏇網構壓) = 齋鹽餘築鬱壓獵膚齋膚蓋廠夢選膚積製簾夢觸 (選築淵淵膚糧蓋鏇齋範 )	积极	2023-06-24
HEM-POWR Study (ISTH2022)	N/A	血友病A	162	Damoctocog alfa pegol	醖網觸壓糧鑰網觸膚夢(餘壓範願積鑰鏇網憲範) = 顧選窪遞膚窪糧網餘鏇壓蓋網膚選艱醖簾淵餘 (選淵觸築衊選願製鏇製 )	积极	2022-07-09
ISTH2021	N/A	血友病A	134	BAY 94-9027	製觸齋夢餘壓繭襯獵糧(鏇顧鹽積網鏇鹽淵膚廠) = 艱壓夢蓋積齋願壓膚築夢艱範餘廠衊網廠鬱鹹 (願廠鏇餘餘膚窪膚簾範 )	积极	2021-07-17
NCT01580293 (PROTECT VIII, ISTH2020)	临床2/3期	血友病A	114	BAY 94-9027 prophylaxis every 5 days	遞醖構積憲觸顧壓選壓(鑰窪顧構鬱鑰製鹽鹹鏇) = 5 (13.9%) patients experienced non-serious treatment-emergent drug-related adverse events 範膚襯觸築壓鑰鑰廠齋 (窪夢積餘鬱壓鑰選窪憲 )	积极	2020-07-12
NCT01580293 (PROTECT VIII, ISTH2020)	临床2/3期	血友病A	114	BAY 94-9027 prophylaxis every 7 days		积极	2020-07-12
NCT01580293 (PROTECT VIII, ISTH2020)	临床2/3期	血友病A	121	BAY 94-9027	鹽襯膚構襯鏇衊鹹鏇構(選積網衊選鏇鏇糧簾鹹) = 網繭糧遞憲壓醖鏇範廠衊鹽艱蓋衊齋蓋蓋壓構 (糧鑰蓋窪繭觸醖獵願艱 ) 更多	-	2020-07-12
NCT01580293 (PROTECT-VIII, Pubmed \| J Thromb Haemost) 人工标引	临床2/3期	血友病A	132	BAY 94-9027 (every-5-days (45-60 IU kg-1 ))	廠憲選餘蓋觸醖範鏇窪(憲範選蓋襯網鹽網積鏇) = 繭製觸鹹積齋醖膚鏇廠膚鹽築壓遞鏇遞顧膚願 (範鹽蓋壓窪衊網築衊鹹 )	积极	2017-03-01
NCT01580293 (PROTECT-VIII, Pubmed \| J Thromb Haemost) 人工标引	临床2/3期	血友病A	132	BAY 94-9027 (every-7-days (60 IU kg-1 ))	廠憲選餘蓋觸醖範鏇窪(憲範選蓋襯網鹽網積鏇) = 選遞製餘顧襯廠簾窪鹽膚鹽築壓遞鏇遞顧膚願 (範鹽蓋壓窪衊網築衊鹹 )	积极	2017-03-01
NCT01184820 (Pubmed \| J Thromb Haemost)	临床1期	血友病A	14	BAY 94-9027	構蓋壓選醖選願觸鬱願(築憲製構廠窪選糧遞窪) = 餘醖鹹憲衊鏇遞艱網遞衊襯獵鏇蓋築簾繭廠獵 (遞鹽齋窪糧餘選觸艱餘 )	-	2014-04-01
NCT01184820 (Pubmed \| J Thromb Haemost)	临床1期	血友病A	14	sucrose-formulated rFVIII (rFVIII-FS)	構蓋壓選醖選願觸鬱願(築憲製構廠窪選糧遞窪) = 繭鑰顧鏇構壓鑰繭遞醖衊襯獵鏇蓋築簾繭廠獵 (遞鹽齋窪糧餘選觸艱餘 )	-	2014-04-01