更新于：2024-11-01

Factor X Deficiency

凝血因子X缺乏症

更新于：2024-11-01

基本信息

别名

Congenital Stuart factor deficiency、Congenital factor X deficiency、Deficiencies, Factor 10

+ [42]

简介

Blood coagulation disorder usually inherited as an autosomal recessive trait, though it can be acquired. It is characterized by defective activity in both the intrinsic and extrinsic pathways, impaired thromboplastin time, and impaired prothrombin consumption.

关联

人凝血因子X(Bio Products Laboratory)

靶点

F10

作用机制

F10刺激剂

在研机构

Bio Products Laboratory USA, Inc. [+1]

原研机构

Bio Products Laboratory USA, Inc.

在研适应症

凝血因子X缺乏症

非在研适应症-

最高研发阶段批准上市

首次获批国家/地区

美国

首次获批日期2015-10-20

Factor XP Behring(CSL Behring LLC)

靶点

F10 x factor IX

作用机制

F10调节剂 [+1]

在研机构

CSL Behring LLC

原研机构

CSL Behring LLC

在研适应症

血友病B [+1]

非在研适应症-

最高研发阶段批准上市

首次获批国家/地区

西班牙

首次获批日期2015-09-01

Catridecacog

卡德凝血素

靶点

fibrin

作用机制

fibrin调节剂 [+1]

在研机构

Novo Nordisk A/S [+5]

原研机构

Novo Nordisk A/S

在研适应症

凝血因子XIII缺乏症 [+2]

非在研适应症

溃疡性结肠炎 [+3]

最高研发阶段批准上市

首次获批国家/地区

欧盟 [+3]

首次获批日期2012-09-03

项与凝血因子X缺乏症相关的临床试验

DRKS00034191 / SuspendedN/A

Prospective naturalistic study on the effectiveness of alcohol withdrawal treatment taking into account individual impulsivity and aggressiveness

开始日期2024-11-01

申办/合作机构

Uniklinik RWTH Aachen

DRKS00031501 / SuspendedN/AIIT

Investigation of the effects from acoustic stimuli on attentional focus and perception of alcohol-associated stimuli.

开始日期2023-04-15

申办/合作机构

Zentralinstitut für Seelische Gesundheit

DRKS00023940 / RecruitingN/AIIT

Supervised disulfiram treatment in a telemedicine setting - What impact does the loss of personal doctor-patient interaction have on the probability of relapse and patient compliance?

开始日期2023-03-27

申办/合作机构

Zentralinstitut für Seelische Gesundheit

100 项与凝血因子X缺乏症相关的临床结果

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100 项与凝血因子X缺乏症相关的转化医学

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0 项与凝血因子X缺乏症相关的专利（医药）

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512

项与凝血因子X缺乏症相关的文献（医药）

2024-07-11·Blood

Fitusiran reduces bleeding in factor X–deficient mice

Article

作者: Maynadié, Hortense ; Verhenne, Sebastien ; Dubois, Christophe ; Adam, Frédéric ; Casari, Caterina ; Crescence, Lydie ; McCluskey, Geneviève ; Christophe, Olivier D. ; Panicot-Dubois, Laurence ; Lenting, Peter J. ; Denis, Cécile V.

AbstractFactor X (FX) deficiency is a rare bleeding disorder manifesting a bleeding tendency caused by low FX activity levels. We aim to explore the use of fitusiran (an investigational small interfering RNA that silences antithrombin expression) to increase thrombin generation and the in vivo hemostatic potential under conditions of FX deficiency. We therefore developed a novel model of inducible FX deficiency, generating mice expressing <1% FX activity and antigen (f10low mice). Compared with control f10WT mice, f10low mice had sixfold and fourfold prolonged clotting times in prothrombin time and activated partial prothrombin time assays, respectively (P < .001). Thrombin generation was severely reduced, irrespective of whether tissue factor or factor XIa was used as an initiator. In vivo analysis revealed near-absent thrombus formation in a laser-induced vessel injury model. Furthermore, in 2 distinct bleeding models, f10low mice displayed an increased bleeding tendency compared with f10WT mice. In the tail-clip assay, blood loss was increased from 12 ± 16 μL to 590 ± 335 μL (P < .0001). In the saphenous vein puncture (SVP) model, the number of clots generated was reduced from 19 ± 5 clots every 30 minutes for f10WT mice to 2 ± 2 clots every 30 minutes (P < .0001) for f10low mice. In both models, bleeding was corrected upon infusion of purified FX. Treatment of f10low mice with fitusiran (2 × 10 mg/kg at 1 week interval) resulted in 17 ± 6% residual antithrombin activity and increased thrombin generation (fourfold and twofold to threefold increase in endogenous thrombin potential and thrombin peak, respectively). In the SVP model, the number of clots was increased to 8 ± 6 clots every 30 minutes (P = .0029). Altogether, we demonstrate that reduction in antithrombin levels is associated with improved hemostatic activity under conditions of FX deficiency.

2024-07-11·Blood

Paving the way to factor X deficiency therapies

Article

作者: Wood, Jeremy P.

2024-07-01·Cureus

Navigating the Challenges of Factor X Deficiency: A Case Study.

Article

作者: Chaudhary, Gaurav A ; Mahashabde, Madhulika L ; Bhavsar, Harin M

The clotting pathway involves intrinsic and extrinsic pathways converging into a common pathway. These pathways require activated factors that sequentially convert prothrombin to thrombin, which then converts fibrinogen to fibrin, forming a stable clot. Clotting factor deficiency impairs this cascade leading to excessive bleeding or bruising due to insufficient clot formation. Here, we present the case of a 47-year-old female who initially complained of epigastric pain. By the third day of admission, she experienced four to five episodes of bleeding gums, resulting in a blood loss of approximately 300 mL. The patient exhibited abnormal prothrombin time (PT) and international normalized ratio (INR) values, leading to a diagnosis of Factor X (FX) deficiency upon further evaluation. This case report emphasizes the need to diagnose coagulopathies such as FX deficiency and how early diagnosis will help not only in patient care and management but also in screening family members who may be affected.

项与凝血因子X缺乏症相关的新闻（医药）

2024-10-07

·蒲公英Ouryao

CDE：关爱计划项目公示

来源：国家药监局审评中心编辑：wangxinglai2004 2024年09月30日，国家药监局审评中心发布了关爱计划项目信息公示，涉及企业为辉瑞投资有限公司，产品治疗领域为血友病相关疾病，公示期截止10月12日。依据《以患者为中心的罕见疾病药物研发试点工作（“关爱计划”）申报指南》，现将拟纳入“关爱计划”试点项目的基本信息予以公示，公示时间截至2024年10月12日。品种名称：Marstacimab 申报单位：辉瑞投资有限公司适应症：本品适用于患有以下疾病的12岁及以上患者的治疗，以减少出血发作：血友病A（先天性凝血因子VIII缺乏症）不伴凝血因子VIII抑制物，或血友病B（先天性凝血因子IX缺乏症）不伴凝血因子IX抑制物。申报阶段：D阶段-上市申请前/上市申请阶段。工作要点：计划在上市后研究阶段收集与评估安全性、有效性及便利性等相关的患者体验数据。如有异议，请将具体意见发送至邮箱：wangxiaoyan@cde.org.cn。以患者为中心的罕见疾病药物研发试点工作计划（“关爱计划”） “关爱计划”是为了推动以患者为中心的药物研发，落实国家药监局关于加快推动罕见疾病用药的工作部署而在罕见疾病药物研发领域开展的一项试点工作。“关爱计划”旨在坚持“以患者为中心”的药物研发理念，鼓励药物研发单位在罕见疾病药物研发过程中倾听患者声音，关注患者的体验与感受。一、背景目前全球已知的罕见疾病超过7000种。罕见疾病发病率/患病率极低，病情复杂，目前对其认识相对有限，加剧了罕见疾病药物研发难度。当前，“以患者为中心”的药物研发理念逐步被广泛接受。对于罕见疾病药物而言，在研发过程中，落实“以患者为中心”的药物研发理念，关注患者视角，倾听患者声音，将有助于药物研发企业、研究者、监管机构了解罕见疾病，了解患者需求，从而助力于对此类药物研发的科学流程及技术要求的优化。为此，药审中心启动以患者为中心的罕见疾病药物研发试点工作，即“关爱计划”。 “关爱计划”旨在指导申请人在药物研发的全程，引入罕见疾病患者的观点，提升将与药物临床获益-风险评价相关的患者体验整合入罕见疾病药物临床研发中的科学性、规范性及合理性，并在此过程中增强监管机构、药物研发单位、患者等专注于罕见疾病药物研发的各方沟通与协作，最终促进罕见疾病药物的上市，满足临床需求。二、主要内容 “关爱计划”是一项旨在罕见疾病药物研发领域落实以患者为中心的药物研发理念的试点工作。《以患者为中心的罕见疾病药物研发试点工作（“关爱计划”）实施框架》（以下简称《实施框架》）是“关爱计划”的配套文件。《实施框架》将药物研发为5个阶段，覆盖药物研发的全生命周期，具体包括： A阶段：研发立项阶段；B阶段：临床试验开展前（Pre-IND）阶段；C阶段：关键研究前阶段；D阶段：上市申请前/上市申请（Pre-NDA/NDA）阶段；E阶段：上市后阶段。《实施框架》中详细介绍了不同的研发阶段可以开展的、体现“以患者为中心”的研发理念的工作内容和具体实施方法，主要包括在不同阶段如何收集符合该研发阶段需要的患者体验数据、如何将这些数据应用于药物研发过程中、以及如何实施兼顾患者体验的临床试验。三、组织与实施《以患者为中心的罕见疾病药物研发试点工作“关爱计划”的申报指南》（以下简称《申报指南》，见附1）和《实施框架》（见附2），详细介绍了申请加入“关爱计划”的方法和具体要求，供有意向申请加入“关爱计划”的申请人参考。申请人基于自愿原则通过沟通交流途径，根据药物所处的研发阶段，申请加入“关爱计划”中相应的阶段（申报表见附3）。药审中心在A、B、C、D、E各阶段选择1—2个具有代表性的品种纳入“关爱计划”。纳入试点项目的品种需根据《实施框架》中的要求开展和完成所对应阶段相关的工作。申请人在依据现行规定和工作程序开展常规的药物申报及注册工作的同时，还需向药审中心汇报实施框架中所要求的相关工作进展或结果。申请人可以通过沟通交流途径向药审中心递交加入“关爱计划”的申请。药审中心将结合产品特点、所开发/申报的适应症情况、以及申请人所提交的实施计划等，经药审中心罕见疾病工作组和相应适应症团队对是否同意其纳入“关爱计划”进行评价，必要时将召开沟通交流会，就实施计划的细节予以讨论。经评价拟同意纳入“关爱计划”后，药审中心将予以公示，公示期为5个工作日，公示无异议的品种最终将纳入“关爱计划”。

申请上市

2024-10-01

·Pharma CMC

辉瑞「马塔西单抗」拟纳入罕见病研发试点工作（“关爱计划”）

9月30日，CDE发布公告，公示关于将Marstacimab纳入《以患者为中心的罕见疾病药物研发试点工作计划（“关爱计划”）》试点项目。 Marstacimab是一种靶向组织因子通路抑制剂（TFPI）的人源IgG1单克隆抗体。TFPI是一种天然抗凝蛋白，可防止血栓形成。 2023年12月，marstacimab的生物制剂许可申请（BLA）获得了FDA和EMA的受理。其中，FDA将PDUFA日期定在2024年Q4，EMA预计在2025年Q1做出审批决定。今年8月13日，CDE网站显示，辉瑞的马塔西单抗（marstacimab）上市申请正式获得药监局受理，用于A型以及B型血友病。依据《以患者为中心的罕见疾病药物研发试点工作（“关爱计划”）申报指南》，现将拟纳入“关爱计划”试点项目的基本信息予以公示，公示时间截至2024年10月12日。品种名称：Marstacimab 申报单位：辉瑞投资有限公司适应症：本品适用于患有以下疾病的12岁及以上患者的治疗，以减少出血发作：血友病A（先天性凝血因子VIII缺乏症）不伴凝血因子VIII抑制物，或血友病B（先天性凝血因子IX缺乏症）不伴凝血因子IX抑制物。申报阶段：D阶段-上市申请前/上市申请阶段。工作要点：计划在上市后研究阶段收集与评估安全性、有效性及便利性等相关的患者体验数据。如有异议，请将具体意见发送至邮箱：wangxiaoyan@cde.org.cn。国家药品监督管理局药品审评中心 2024年9月30日

申请上市

2024-09-12

·PRNewswire

Kedrion Biopharma's Focus on Advancing Patient Care in Rare Disease to be Showcased at NBDF Conference 2024

ATLANTA, Sept. 11, 2024 /PRNewswire/ -- Kedrion Biopharma Inc. is excited to announce its sponsorship of the National Bleeding Disorders Foundation's (NBDF) 76th Annual Bleeding Disorders Conference (BDC) in Atlanta, Georgia. The event will take place from September 12-14, 2024, and will bring together individuals with bleeding disorders, their families, healthcare professionals, and researchers from across the country. Kedrion's sponsorship of the NBDF underscores the company's commitment to supporting the bleeding disorders community. Kedrion is a leading manufacturer of coagulation and rare disease therapies and is dedicated to providing patients with access to innovative treatments. The NBDF Conference is a unique opportunity for attendees to learn about the latest advancements in bleeding and clotting disorders research and treatment, connect with other individuals and families affected by these conditions, and gain valuable insights from experts in the field. The conference will feature a wide range of educational sessions, workshops, and networking opportunities, covering topics such as: Treatment options and advancements: Attendees will have the chance to learn about the latest developments in treatment options for various bleeding and clotting disorders, including new therapies and clinical trials. Living with a bleeding and or clotting disorder: The conference will address the challenges and opportunities associated with living with a diagnosis, including managing symptoms, navigating healthcare systems, and building support networks. Research and advocacy: Attendees will gain insights into ongoing research efforts and advocacy initiatives aimed at improving the lives of individuals with such disorders. Kedrion will be actively involved in the NBDF Conference, hosting several events and activities designed to engage with attendees and share information about its therapies. Highlights will be a Campfire presentation on the results of the Hereditary Factor X in Americas survey, the 2nd Annual Plasminogen Deficiency Roundtable and The Ultra Rare Networking Hour, a dedicated space for individuals with ultra-rare bleeding and or clotting disorders and their support network to connect and build relationships. This initiative will foster a sense of community and provide a platform for sharing experiences and resources. The NBDF Conference is also a valuable opportunity for Kedrion to connect with individuals living with Plasminogen Deficiency Type 1(PLGD-1), as well as patients with Hereditary Factor X Deficiency (HFXD), two rare and potentially life-threatening disorders. The company will be sharing information about the therapy developments in these areas and its ongoing research efforts. "At Kedrion, every employee is united by a powerful mission: to amplify the voices of patients with rare diseases and to continually improve their journey and care. Together, our global commitment ensures that every step forward is a step towards a brighter, more hopeful future" said Bob Rossilli, CCO, Global Business and US General Manager of Kedrion. Kedrion's participation in the NBDC reflects the company's commitment to improving the lives of individuals with rare diseases. By providing support, education, and access to innovative therapies, Kedrion is working to ensure that patients have the resources they need to live full and healthy lives. To learn more about the NBDF's 76th Annual Bleeding Disorders Conference, please visit National Bleeding Disorders Foundation, formerly NHF | NBDF. Contact: Jennifer Bayliss / [email protected] / 213-703-9604 SOURCE Kedrion Biopharma WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED