This study is conducted in Japan of vonicog alfa (rVWF) used to treat pediatric participants with Von Willebrand Disease (vWD).
The main aim of the study is to evaluate adverse drug reaction and effectiveness of vonicog alfa (rVWF).
During the study, pediatric participants with vWD will be administered with rVWF under routine normal practice. The investigators will evaluate adverse events due to rVWF for 1 year from the start of drug administration.
The study sponsor will not be involved in how the participants are administered but will be recorded what happens during the study.

开始日期2026-02-19

申办/合作机构

Takeda Pharmaceutical Co., Ltd.

NCT07129343

/ Recruiting临床3期

A Clinical Study to Evaluate the Safety, Efficacy and Pharmacokinetics of Recombinant Von Willebrand Factor (rVWF) With or Without ADVATE in the Treatment of Bleeding Episodes in Chinese Subjects Diagnosed With Von Willebrand Disease

The main aim of this study is to find out if VONVENDI is safe for adult Chinese participants with VWD. The study will also check how well VONVENDI helps control bleeding with or without product ADVATE in the participants who may need elective surgery or dental procedures. In addition, the study will also examine how VONVENDI is processed by the body (known as pharmacokinetic [PK]) and how the drug helps the body respond or improve a condition (pharmacodynamic [PD]).
Participants will receive an initial dose of VONVENDI of 40 to 80 international units per kilogram (IU/kg) of body weight. If a participant's baseline factor VIII (FVIII) level is not high enough to help stop bleeding, VONVENDI will be given along with 30 to 45 IU/kg of ADVATE rFVIII.
Participants will be in the study for approximately 14 months. During the study, participants will be followed up at clinics or over telephone calls.

开始日期2025-10-13

申办/合作机构

Takeda Pharmaceutical Co., Ltd.

ChiCTR2500108310

/ Not yet recruiting临床3期IIT

A Clinical Study to Evaluate the Safety, Efficacy and Pharmacokinetics of Recombinant von Willebrand Factor (rVWF) With or Without ADVATE in the Treatment of Bleeding Episodes in Chinese Subjects Diagnosed With von Willebrand Disease

开始日期2025-03-21

申办/合作机构

苏州大学附属第一医院

100 项与伏尼凝血素α 相关的临床结果

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100 项与伏尼凝血素α 相关的转化医学

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100 项与伏尼凝血素α 相关的专利（医药）

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项与伏尼凝血素α 相关的文献（医药）

2026-04-01·Journal of Comparative Effectiveness Research

Real-word evidence on healthcare resource use and associated costs in on-demand users of replacement therapies in von Willebrand disease in France: the FORvWARD study.

Article

作者: Belhassen, Manon ; Favre-Besse, Franck ; Polack, Benoit ; Trossaërt, Marc ; Biron-Andreani, Christine ; Nerich, Virginie ; Lefèvre, Cinira ; Pouvourville, Gérard de ; Née, Mélanie ; Bornier, Nadège ; Micallef, Claire Marant ; Chatelanaz, Catherine ; Beoletto, Julien

Background: Real-world data about use of Von Willebrand factor (VWF) concentrates to manage on-demand patients with Von Willebrand disease (VWD) are scarce. Aim: To describe and compare patients' characteristics, treatment patterns, healthcare resource use and associated costs of patients with VWD using VWF concentrates. Materials & methods: Using the French healthcare claims database, we included adult patients with ≥1 reimbursement for a replacement therapy (RT) containing VWF concentrate between 1 January 2017 and 30 September 2021 and followed them from first RT dispensation to 31 December 2021. Treatment patterns, healthcare resource use and associated costs of RT on-demand users were evaluated over each 30-days exposure period (EP) starting the first day of each hospital stay with ≥1 RT administration. In- and out-hospital RT doses and FVIII, number of general practitioner and nurse visits, in- and out-hospital RT dispensings and length of hospitalizations and their costs were described and compared across RTs using adjusted Generalized Estimating Equation models accounting for confounding factors. Results: Among 2540 on-demand RT users, WILFACTIN^® was the main RT used, followed by VONCENTO^®, VEYVONDI^®, EQWILATE^® and WILSTART^®. Overall, the mean total RT dose was 12,962 IU and the mean cost was €21,034/EP. Compared with VEYVONDI^®-treated EP, WILFACTIN^®-treated EP had significantly longer stay duration, had more out-hospital RT dose and had higher overall and in-hospital costs; VONCENTO^®-treated EP had more overall and in-hospital RT dose, and had higher in-hospital and RT-related costs. Conclusion: This first real-world study suggests that VEYVONDI^® seems to be a cost-saving RT compared with other RT. Future studies including clinical data should provide further evidence.

2026-02-10·Blood Advances

Modeling response to high-purity von Willebrand factor: toward tailored dosing during surgery in von Willebrand disease

Article

作者: Daniel, Mélanie Y. ; Dupont, Annabelle ; Zawadzki, Christophe ; Jeanpierre, Emmanuelle ; Marichez, Catherine ; Rauch, Antoine ; Denis, Cécile V. ; Goudemand, Jenny ; Lenting, Peter J. ; Paris, Camille ; Susen, Sophie ; Delavenne, Xavier ; Pradines, Bénédicte

Abstract:

In surgical settings, managing von Willebrand disease (VWD) often requires von Willebrand factor (VWF) concentrate administration, and close monitoring of factor VIII (FVIII) and VWF to balance bleeding control with the risk of excessive factor elevation. This management is further complicated by VWD-related variability and the pharmacokinetic (PK) impact of VWF products, containing or not exogenous FVIII. We hypothesized that building a PK model based on high-purity VWF concentrates may clarify the VWF-FVIII relationship by eliminating exogenous FVIII interference. Our aim was to characterize the PK/pharmacodynamic (PD) relationship and variability of a high-purity VWF concentrate in patients with VWD undergoing surgery. A retrospective analysis was conducted on patients treated perioperatively with a high-purity plasma-derived VWF concentrate (Wilfactin, Laboratoire Français du Fractionnement et des Biotechnologies, France), nearly devoid of FVIII, from January 2016 to December 2018. Data were analyzed using a population PK/PD modeling approach. VWF antigen (VWF:Ag) was prioritized as it better reflects VWF-FVIII binding capacity rather than VWF activity, which measures platelet interaction. Covariates influencing VWF:Ag and FVIII:C PK/PD were analyzed. Seventy-four patients undergoing 99 surgeries were included. The model revealed substantial variability in VWF:Ag and FVIII:C volume of distribution, clearance, and baseline levels. Covariates such as age, bodyweight, VWD type, blood group, and VWF propeptide significantly affected PK/PD parameters. Interindividual variability in PK parameters (eg, clearance, volume), ranged from 25.3% to 73.8%. High interindividual PK/PD variability was observed with high-purity VWF concentrate. This highlights the need to develop a robust dose-adjustment model to optimize factor concentrate utilization, avoid unnecessary waste, and reduce the frequency of laboratory monitoring.

2026-02-01·JOURNAL OF THROMBOSIS AND HAEMOSTASIS

Prevention of hemorrhage after implantation of mechanical circulatory support with a purified von Willebrand factor concentrate: results of the early terminated randomized controlled trial

Article

作者: Dambrin, Camille ; Fiore, Mathieu ; Smadja, David M ; Lenting, Peter J ; Flecher, Erwan ; Jeanpierre, Emmanuelle ; Rauch, Antoine ; Barandon, Laurent ; Bouchot, Olivier ; Nedelec, Fabienne ; Repessé, Yohann ; Vincentelli, André ; Moussa, Mouhamed D ; Rozec, Bertrand ; Voisin, Sophie ; Laporte, Silvy ; Boissier, Elodie ; De Maistre, Emmanuel ; Labreuche, Julien ; Ajzenberg, Nadine ; Lebreton, Guillaume ; Susen, Sophie

BACKGROUND:

Bleeding complications are frequent under left ventricular assist device (LVAD) support. LVAD-associated high shear stress induces increased proteolysis of circulating von Willebrand factor (VWF), which may contribute to this high bleeding rate.

OBJECTIVES:

To assess if a prophylactic administration of a VWF concentrate could reduce bleeding rate in LVAD patients METHODS: In this multicenter open-label randomized controlled study, we investigated the efficacy, safety, and pharmacokinetic of a plasma-derived VWF concentrate (WILFACTIN, LFB), administered prophylactically twice-weekly at 50 IU.kg^-1 for 3 months after LVAD implantation, compared with standard of care. The efficacy end points were the difference in the incidence rate of any, clinically significant or major bleeding postrandomization. Bleeding and thrombotic events were reviewed at weeks 1 and 2 and then every 2 weeks. The study ended prematurely due to deficient recruitment and drug supply constraints, leading to prioritization of prescriptions for von Willebrand disease patients. We present the available data.

RESULTS:

Twenty-nine adult patients (166 planned) were randomized and analyzed. VWF prophylaxis resulted in a nonsignificant 55% reduction in bleeding incidence rate compared with standard of care (risk ratio, 0.45; 95% CI, 0.18-1.04; P = .06). Accordingly, there was a similar reduction of clinically relevant and major bleeding in VWF arm. Severe adverse events were similar postrandomization between VWF arm and control arm including deaths (2 and 4, respectively) and thrombotic events (2 in each arm). In pharmacokinetic analysis, a rapid degradation of WILFACTIN occurred within 24 hours.

CONCLUSION:

The lack of observed therapeutic efficacy of VWF prophylaxis in LVAD setting is likely due to a short-lived prohemostatic effect due to rapid degradation of VWF concentrate.

项与伏尼凝血素α 相关的新闻（医药）

2026-03-06

·allsci.com

Hemab Therapeutics takes FDA Breakthrough status for sutacimig in Glanzmann thrombasthenia

Hemab Therapeutics, a biotech headquartered in Cambridge, Massachusetts, and Copenhagen, Denmark, announced receipt of US FDA Breakthrough Therapy Designation (BTD) for sutacimig, a subcutaneously administered bispecific antibody, for the prevention of bleeding episodes in patients with Glanzmann thrombasthenia (GT). Sutacimig, formerly designated HMB-001, binds and stabilizes endogenous Factor VIIa with one antibody arm while binding TLT-1 on activated platelets with the other, facilitating hemostatic plug formation. Hemab Therapeutics is a privately held company with no public stock ticker. BTD provides Hemab Therapeutics with intensive FDA guidance on drug development, organizational commitment from senior FDA managers, and eligibility for rolling review, which permits submission of completed sections of a marketing application before the entire filing is ready. Sutacimig’s clinical progress Sutacimig was originated and developed in-house by Hemab, which was founded in 2019 from academic research in Scandinavian coagulation biology. The BTD was granted on the basis of data from the completed Phase II multiple ascending dose portion of Hemab’s Phase I/II clinical trial (NCT06211634). In that study, the weekly dosing group achieved an estimated 87% reduction in annualized treated bleeding rate, with consistent reductions observed across bleed types, locations, and dosing cohorts. All tested doses produced over a 50% reduction in treated bleeding events. No major safety signals were reported in public disclosures from the Phase II portion. Hemab has stated plans to advance sutacimig into a pivotal Phase III study. Prior to this BTD, sutacimig had already received Fast Track Designation and Orphan Drug Designation from the FDA, orphan medicinal product designation in the European Union, and Innovative Licensing and Access Pathway (ILAP) designation from the UK Medicines and Healthcare products Regulatory Agency. Research context GT is a rare, inherited platelet disorder caused by deficiency or dysfunction of the glycoprotein IIb/IIIa complex, resulting in impaired platelet aggregation and recurrent, sometimes life-threatening bleeding. Data from the international GT360 natural history study reported that 88% of 117 participants experienced at least one bleed in the prior week, and 65% required a bleed-related hospital visit within six months. Over 80% had missed work or school, and more than 50% faced restrictions on social activities and travel. No prophylactic treatment for GT has received regulatory approval. The current standard of care relies on episodic, on-demand interventions, principally recombinant activated Factor VIIa (NovoSeven, Novo Nordisk) and platelet transfusions, neither of which was developed or approved as a prophylactic bleeding disorder treatment for GT. No novel drugs for GT have been approved in the past three years. The competitive landscape for therapies targeting von Willebrand factor or platelet glycoprotein receptors includes caplacizumab (an anti-vWF nanobody for thrombotic thrombocytopenic purpura), vonicog alfa (recombinant vWF for von Willebrand disease), and BT200 (a pegylated RNA aptamer targeting vWF). However, none of these molecules are in development for GT, and none share sutacimig’s bispecific mechanism simultaneously engaging Factor VIIa and activated platelet TLT-1. Unlike agents such as caplacizumab, which block vWF-platelet interactions to prevent pathological clotting, sutacimig is designed to promote hemostasis by concentrating endogenous Factor VIIa at the site of platelet activation — a mechanistically distinct, pro-hemostatic approach. No other bispecific antibody targeting both Factor VIIa and a platelet surface receptor for a pro-hemostatic indication was identified in active clinical development. Hemab’s pipeline also includes HMB-002 for von Willebrand disease and a Phase II trial of sutacimig in congenital Factor VII deficiency (NCT07347249), indicating broader ambitions for the bispecific platform across coagulation disorders. Your email address will not be published. Required fields are marked * Comment * Name * Email * Website

孤儿药快速通道临床2期临床结果突破性疗法

2026-01-22

·新浪医药

辉瑞 1 类新药新适应症申报上市

1 月 22 日，CDE 官网显示，辉瑞 1 类新药「马塔西单抗注射液」新适应症申报上市，注册分类为 2.2 类。根据公开信息和临床试验进展，Insight 数据库推测本次适应症为抑制先天性血友病患者（存在凝血因子 VIII 或因子 IX 抑制剂）的出血倾向。截图来源：CDE 官网马塔西单抗（Marstacimab、PF-6741086）是辉瑞开发的一种靶向组织因子途径抑制物（TFPI）K2 结构域的单克隆抗体，属于血友病再平衡疗法。TFPI 是一种天然抗凝蛋白，具有防止血栓形成和恢复止血的功能。辉瑞曾针对马塔西单抗开展一项关键 III 期试验 BASIS（登记号：NCT03938792）。这是一项全球性的 III 期、开放标签、多中心研究，旨在评估马塔西单抗对 12 岁至 75 岁患有重型血友病 A（定义为 FVIII <1%）或中重型至重型血友病 B（定义为 FIX 活性 ≤2%）且伴有或不伴有抑制物的青少年和成人患者的疗效和安全性。该研究表明，在年出血率这一主要终点方面，预防性使用马塔西单抗优于既往按需使用旁路制剂的替代疗法。此外，其安全性也良好。 Insight 数据库显示，该药已陆续在美国、欧盟、日本和中国获批上市。获批适应症为：用于规律性治疗患有不伴凝血因子抑制物的重型血友病 A（先天性凝血 VIII 因子缺乏症，FVIII<1%）或者重型血友病 B（先天性凝血 IX 因子缺乏症，FIX<1%）的 12 岁及以上且体重不低于 35 公斤的患者，以减少出血的发作。该药获批后，成为全球首个 B 型血友病每周一次皮下注射药物，也是首个适用于 A/B 型血友病的固定剂量方案。 2025 年 12 月，辉瑞宣布在日本提交马塔西单抗新适应症的上市申请，拟增加「抑制先天性血友病患者（存在凝血因子 VIII 或因子 IX 抑制剂）的出血倾向」的适应症。本次是同适应症在国内获得 CDE 正式受理。目前全球在研的血友病疗法数量众多，涵盖了多种不同的技术路径和治疗策略。赛诺菲的芬妥司兰钠是全球首个靶向抗凝血酶的 siRNA 疗法，在给药频率上每年仅需 6 针。此前获批的血友病基因疗法，有武田的注射用伏尼凝血素α、BioMarin Pharmaceutical公司的基因疗法Roctavian（valoctocogene roxaparvovec）等。血友病市场规模正在增长，预计到2028年将达到数十亿元的市场规模。辉瑞、赛诺菲、拜耳、武田等跨国企业均有相关药物进入临床；国内公司中国生物、国药集团、华兰生物新等也在进行各个阶段的临床研究。加入读者交流群： —精彩回顾— 我国启动全球最大规模尿液HPV筛查研究针对红斑狼疮！阿斯利康首创生物制剂 III 期成功恒瑞乙肝「突破性疗法」来袭 ADC之后，ABC来了全球首例！中国医生体内CAR-T成功治疗狼疮商务合作：杨小雨 15210041717

临床3期基因疗法免疫疗法抗体药物偶联物细胞疗法

2025-12-12

·医药专利

见证奇迹，2025年中国上市的第102款新药——赛诺菲血友病 siRNA 芬妥司兰钠

中国NMPA2025年批准新药102个（新药定义为含中国首次上市的活性成分）。 2025.12.11药监局公告了12月9日批准的4个新药，中国2025年上市新药99/100/101/102的序号怎么排呢？库莫西利胶囊、玛帕西沙韦胶囊、佐来曲替尼片、芬妥司兰钠注射液。 1类新药中药生物药化药合计 5 36 60 101 国产 5 23 35 63 进口 0 13 25 38 读者意见征询见证中国创新药当代奇迹——新药审批突破100 对于中国创新药产业链相关从业者及关注者而言，2025年注定是载入史册的里程碑之年。截至12月3日，国家药品监督管理局已批准98个创新药上市，全年突破百款新药已成定局，这一成就将刷新全球单一国家年度新药获批数量的历史纪录。为铭记这一医药创新黄金时代的辉煌时刻，医药专利威公众号自年初起持续跟踪报道，现计划于年末推出纪念特辑《2025 年中国上市的 100 个新药》，现面向行业同仁及社会各界开展三项意见征集：一、图书预售意向调研本书拟收录100个获批新药的研发历程、技术突破、临床价值及产业影响等深度内容，200-400页，定价区间100-200元。现征集首批预售意向，您的反馈将直接影响首印数量及发行策略。如果意向低于500人，将取消本次编辑出版。二、联合编辑团队招募计划组建10人专家编辑委员会，成员需具备医药研发、临床研究、注册申报或医药传媒等领域专业背景。入选者将深度参与内容架构设计、案例筛选及专业审校工作，获颁纪念证书并载入图书编委会名录。三、战略合作伙伴征集本书的编辑、排版、印刷与发行需投入一定成本，面向医药企业开放10个广告合作席位，合作形式包括但不限于内页广告、专题冠名、数据支持等。合作企业将获得品牌露出、新品宣传及行业峰会优先参与权等增值服务，共同见证中国医药创新生态的蓬勃发展。（征集截止日期：2025年12月15日，详情请联系编辑部邮箱：pharmpatent@163.com，主题注明简要注明定书、编辑、合作；或加编辑微信读者意见调查 102 芬妥司兰钠注射液（2025.12.09）血友病 12 月 10 日晚间，赛诺菲宣布其全球首个且唯一的血友病 siRNA 疗法芬妥司兰钠（Fitusiran，赛菲因®，Qfitlia®）获 NMPA 批准上市, 适用于患有以下疾病的 12 岁及以上儿童和成人患者的常规预防治疗，以防止出血或降低出血发作的频率: 存在或不存在凝血因子 V 川抑制物的重型 A 型血友病 (先天性凝血因子 VII 缺乏，FVI<1%) 或存在或不存在凝血因子 IX 抑制物的重型 B 型血友病 (先天性凝血因子|X 缺乏,FIX<1%)。血友病是我国首批被纳入目录的罕见病之一，目前国内登记患者数约4万余例。据《中国血友病患者治疗模式与疾病负担研究报告》显示[4]，重型血友病患者年均出血次数高达32次，每十天左右一次的"出血负担"是患者面临的巨大难关，其中90.42%的患者发生关节出血。 2025年版《血友病治疗中国指南》将非因子治疗定义为规律替代治疗（预防治疗）之一，作为重型以及反复发生出血的中间型血友病患者的管理基石。规范的规律替代治疗是控制出血的关键，但规范意味着患者每周需要进行2-3次预防性的足剂量凝血因子注射，每年100多次静脉注射，沉重的"针头负担"让不少患者望而却步。该疗法通过每年最少 6 次皮下注射的创新机制, 有望破解中国血友病人群长期面临的“出血”与“针头”双重负担, 推动中国血友病治疗模式向规范化、可持续转型，拓展了中国规律替代治疗新选择。 Mechanism of Action QFITLIA is a double-stranded siRNA that causes degradation of AT messenger RNA (mRNA)through RNA interference, reducing plasma AT levels. 作用机制 QFITLIA是一种双链小干扰RNA（siRNA），通过RNA干扰机制降解血管紧张素转换酶（AT）信使RNA（mRNA），从而降低血浆AT水平。 2025年3月，赛菲因®（芬妥司兰钠注射液）已成功获得美国食品药品监督管理局（FDA）的批准。此次在中国成功获批，为中国血友病规律替代治疗提供了全新选择，使国内患者能够及时共享全球医学进展。值得一提的是，此前赛菲因®已被纳入国家药品监督管理局药品审评中心（CDE)《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。芬妥司兰钠布局有 6 项 III 期临床试验，对不同群体展开了细致的研究，其中 5 项试验地区包括中国： ATLAS-INH（NCT03417102/CTR20182299）：针对有抑制剂患者，对比芬妥司兰钠相较于旁路制剂的有效性 ATLAS-A/B（NCT03417245/CTR20182301）：针对无抑制剂患者，对比芬妥司兰钠相较于凝血因子按需治疗的有效性 ATLAS-PPX（NCT03549871/CTR20182302）：一项转换治疗研究，研究既往接受凝血因子或旁路制剂预防性治疗的 A 型或 B 型血友病患者从传统疗法转向芬妥司兰钠的获益 ATLAS-OLE（NCT03754790/CTR20200294）：探索芬妥司兰钠的长期疗效和安全性 ATLAS-NEO（NCT05662319/CTR20230417）：在有或无抑制剂的 ≥ 12 岁重度血友病男性青少年或成人受试者中探索芬妥司兰钠的预防治疗疗效和安全性 ATLAS 研究数据主要结果包括：与按需使用凝血因子浓缩物相比，使用 Qfitlia 预防治疗的无抑制剂患者的 ABR 出血显著减少 71%（估计平均值：ABR 9.0 vs. 31.4；p<0.0001），与按需使用旁路制剂相比，有抑制剂患者的 ABR 出血减少 73%（估计平均值：ABR 5.1 vs. 19.1；p=0.0006）。在开放标签扩展研究期间观察到的 ABR 中位数，在无抑制剂患者中为 3.8（IQR：0.0–11.2），在有抑制剂患者中为 1.9（IQR：0.0–5.6）。在开放标签扩展研究期间观察到的年自发性出血率中位数，在无抑制剂患者中为 1.9（四分位距 (IQR)：0.0-7.5），在有抑制剂患者中为 1.9（IQR： 0.0-3.7)。开放标签扩展研究中，近一半的患者经历了一次或更少的出血（31% 0 次出血和 47% 0-1 次出血）。安全性方面，可能出现严重的不良反应，包括血栓事件、急性和复发性胆囊疾病以及肝毒性。最常见的不良反应（发生率 >10%）是病毒感染、鼻咽炎和细菌感染。此前获批的血友病基因疗法，有辉瑞的马塔西单抗【2025-11-23】规律长效血友病药物马塔西单抗——2025年中国上市的第92款新药武田的注射用伏尼凝血素α、BioMarin Pharmaceutical公司的基因疗法Roctavian（valoctocogene roxaparvovec）等。还有中国上海信致医药科技有限公司申报的波哌达可基注射液（Dalnacogene Ponparvovec Injection，【2025-04-10】2025年中国上市的第30款创新药——波哌达可基注射液，中国首个基因治疗药物血友病市场规模正在增长，预计到2028年将达到数十亿元的市场规模。 101 佐来曲替尼片（2025.12.09）NTRK融合基因实体瘤见证奇迹，2025年中国上市的第101款新药——诺诚健华TRK抑制剂佐来曲替尼 100 玛帕西沙韦胶囊（2025.12.09）流感见证奇迹，2025年中国上市的第100款新药——健康元流感新药TG-1000玛帕西沙韦 99 库莫西利胶囊（2025.12.09）乳腺癌正大天晴全球首款CDK2/4/6抑制剂库莫西利——2025年中国上市的第99款新药 98西诺氨酯片（2025.12.03）癫痫抗癫痫西诺氨酯——2025年中国上市的第98款新药 97盐酸索安非托片（2025.12.03）日间过度嗜睡打工人瞌睡提神药索安非托——2025年中国上市的第97款新药 96 苹果酸司妥吉仑片（2025.12.03）高血压上海医药高血压新药司妥吉仑——2025年中国上市的第96款新药 95 瑞米布替尼片（2025.11.25）荨麻疹慢性荨麻疹瑞米布替尼——2025年中国上市的第95款新药 94 匹康奇拜单抗注射液（2025.11.25）银屑病银屑病3个月一针匹康奇拜单抗——2025年中国上市的第94款新药 93 注射用镓[68Ga]戈泽肽（2025.11.25）前列腺癌诊断放射诊断镓[68Ga]戈泽肽——2025年中国上市的第93款新药 92 马塔西单抗注射液（2025.11.21）血友病规律长效血友病药物马塔西单抗——2025年中国上市的第92款新药 91维培那肽注射液（2025.11.14）T2DM GLP-1RA周制剂维培那肽——2025年中国上市的第91款新药 90 镥[177Lu]特昔维匹肽注射液（2025.11.10）前列腺癌诺华中国首款放射配体核药镥[177Lu]——2025年中国上市的第90款新药 89 盐酸沙瑞环素片（2025.11.10）痤疮痤疮口服抗生素沙瑞环素——2025年中国上市的第89款新药 88 普基奥仑赛注射液（2025.11.7）r/r B-ALL 百万CAR-T第8款普基奥仑赛儿童白血病首款——2025年中国上市的第88款新药 87 注射用卡拉西珠单抗（2025.11.5）iTTP 赛诺菲罕见病药卡拉西珠单抗疗效仅缩短病程0.19天——2025年中国上市的第87款新药 86 奥格特韦钠胶囊（2025.11.5）新冠病毒迟来的新冠药奥格特韦——2025年中国上市的第86款新药 85 注射用维贝柯妥塔单抗（2025.10.30）鼻咽癌乐普生物全球首创EGFR ADC维贝柯妥塔单抗——2025年中国上市的第85款新药 84 马来酸美凡厄替尼片（2025.10.24）肺癌华东医药第二个新药美凡厄替尼——2025年中国上市的第84款新药 83 那米司特片（2025.10.24）肺纤维化肺纤维化新药那米司特——2025年中国上市的第83款新药 82 注射用博度曲妥珠单抗（2025.10.17）乳腺癌科伦第二个ADC博度曲妥珠单抗——2025年中国上市的第81款新药 81 瑞玛比嗪注射液（2025.10.17）GFR诊断华东GFR诊断瑞玛比嗪——2025年中国上市的第81款新药 80 泽卢克布仑钠注射液（2025.10.11）重症肌无力（MG）重症肌无力新药泽卢克布仑钠——2025年中国上市的第80款新药 80 泽卢克布仑钠注射液（2025.10.11）重症肌无力（MG）重症肌无力新药泽卢克布仑钠——2025年中国上市的第80款新药 79 莫米司特片（2025.10.11）银屑病银屑病新药莫米司特——2025年中国上市的第79款新药 78 宗艾替尼片（2025.8.29）非小细胞肺癌（NSCLC）勃林格殷格翰肺癌新药宗艾替尼——2025年中国上市的第78款新药 77 泽美妥司他片（2025.8.29）淋巴瘤恒瑞首个国产EZH2抑制剂泽美妥司他——2025年中国上市的第77款新药 76 口服六价重配轮状病毒减毒活疫苗（Vero细胞）（2025.8.22）六价重配轮状病毒减毒活疫苗——2025年中国上市的第76款新药 75 注射用德达博妥单抗（2025.8.22）乳腺癌 ADC德达博妥单抗——2025年中国上市的第75款新药 74 地罗阿克片（2025.8.22）非小细胞肺癌（NSCLC）地罗阿克——2025年中国上市的第74款新药，13 款 ALK 抑制剂 73 盐酸阿曲生坦片（2025.8.22） IgA肾病 IgA肾病新药阿曲生坦——2025年中国上市的第73款新药 72 参蒲颗粒（2025.8.1）慢性盆腔痛康缘1.1类中药新药参蒲颗粒——2025年中国上市的第72款新药 71 甲磺酸兰泽替尼片（2025.7.30） NSCLC 三代非小细胞肺癌兰泽替尼——2025年中国上市的第71款新药 70 雷尼基奥仑赛注射液（2025.7.30） R/R LBCL国产第7款CAR-T雷尼基奥仑赛——2025年中国上市的第70款新药 69 盐酸妥诺达非片（2025.7.25） EDED新药妥诺达非，扬子江22年22项临床坚持不懈——2025年中国上市的第69款新药 68 重组人白蛋白注射液（2025.7.18）低白蛋白血症禾元生物双喜临门，成功IPO+“稻米造血”重组人白蛋白注射液上市——2025年中国上市的第68款新药 67 玛硒洛沙韦片（2025.7.18）流感流感新药玛硒洛沙韦17个月完成审批——2025年中国上市的第67款新药 66 利沙托克拉片（2025.7.8） CLL/SLL 利沙托克拉、褪黑素——2025年中国上市的第65-66款新药 65 褪黑素颗粒（2025.7.8）失眠 64 盐酸司美那非片（2025.7.8） ED ED新药司美那非——出于淫羊藿更胜于淫羊藿，为淫羊藿正名——2025年中国上市的第64款新药为阅读方便，上半年新药不再赘述，请看链接 2025上半年中国创新药收官分析：63款新药获批，国产40款！中国获批创新药清单（2025.12.11）102个 1类新药中药生物药化药合计 5 37 60 102 国产 5 23 35 63 进口 0 14 25 39 长按下方二维码立即咨询、领取资料

siRNA上市批准临床申请

100 项与伏尼凝血素α 相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D08681	伏尼凝血素α	B02BD10	DB12872

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
1型血管性血友病	美国	Takeda Pharmaceuticals U.S.A., Inc.	2025-09-05
2型血管性血友病	美国	Takeda Pharmaceuticals U.S.A., Inc.	2025-09-05
冯·维勒布兰德病	欧盟	Baxalta Innovations GmbH	2018-08-31
冯·维勒布兰德病	冰岛	Baxalta Innovations GmbH	2018-08-31
冯·维勒布兰德病	列支敦士登	Baxalta Innovations GmbH	2018-08-31
冯·维勒布兰德病	挪威	Baxalta Innovations GmbH	2018-08-31
血管性血友病3型	美国	Takeda Pharmaceuticals U.S.A., Inc.	2015-12-08

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	Baxalta, Inc.	2011-11-01
出血	临床3期	日本	Baxalta, Inc.	2011-11-01
出血	临床3期	澳大利亚	Baxalta, Inc.	2011-11-01
出血	临床3期	奥地利	Baxalta, Inc.	2011-11-01
出血	临床3期	比利时	Baxalta, Inc.	2011-11-01
出血	临床3期	保加利亚	Baxalta, Inc.	2011-11-01
出血	临床3期	加拿大	Baxalta, Inc.	2011-11-01
出血	临床3期	德国	Baxalta, Inc.	2011-11-01
出血	临床3期	印度	Baxalta, Inc.	2011-11-01
出血	临床3期	意大利	Baxalta, Inc.	2011-11-01

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临床结果

适应症

分期

评价

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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


NCT04344860 (VWD-Woman, Pubmed \| Blood Adv)	临床3期	1型血管性血友病	20	Tranexamic acid + recombinant VWF	鹹淵鏇觸鹹夢製憲齋餘(繭鏇獵製窪蓋壓製衊糧): P-Value = 0.49 更多	不佳	2025-12-09
				Recombinant VWF
ASH2025	N/A	冯·维勒布兰德病	9	Recombinant von willebrand factor (severe VWD)	窪鑰餘餘鑰鏇憲鹹鏇壓(艱餘艱衊製築顧壓壓窪) = There were no reported adverse events defined by the European Haemophilia Safety Surveillance program, including treatment-emergent side effects, hypersensitivity/allergic reactions, thrombotic events, or events of VWF inhibitor development. 壓網觸糧積簾膚齋壓鹹 (顧繭範築壓築範製繭艱 )	积极	2025-12-06
NCT04344860 (VWD Woman Trial \| VWD-Woman, CTgov)	临床3期	冯·维勒布兰德病	20	Recombinant Von Willebrand factor+Tranexamic Acid Injection [Cyklokapron] (rVWF plus TA)	鬱獵鏇淵積餘構簾淵顧(鏇鹽鏇鬱顧餘構築糧簾) = 顧簾獵簾積鹽夢鏇範鬱襯觸壓願衊艱餘顧願艱 (鹹鹽鑰廠襯選構壓膚繭, 802.5) 更多	-	2025-10-31
				Recombinant Von Willebrand factor (rVWF alone)	鬱獵鏇淵積餘構簾淵顧(鏇鹽鏇鬱顧餘構築糧簾) = 製壓淵艱憲鏇艱餘鏇鹽襯觸壓願衊艱餘顧願艱 (鹹鹽鑰廠襯選構壓膚繭, 355.08) 更多
ASH2024	N/A	冯·维勒布兰德病	-	Continuous prophylaxis with recombinant VWF	積築範齋積淵獵選鬱衊(廠蓋廠醖鑰製鹽憲鏇壓) = One participant reported 9 adverse events (AEs); all AEs were resolved and determined to be unrelated to rVWF treatment 鑰鏇選蓋醖壓願齋願積 (蓋製遞衊遞憲艱糧窪廠 ) 更多	-	2024-12-08
NCT02973087 (Pubmed \| Circulation \| Blood) 人工标引	临床3期	冯·维勒布兰德病	23	Recombinant von Willebrand factor (Prior On-Demand group)	壓淵顧壓鏇顧網夢選夢(餘獵衊廠鹽壓繭顧糧遞) = 遞膚窪鏇襯顧鑰鏇範齋製顧獵網糧膚廠鹽鏇壓 (鹽觸醖鹹糧窪鏇鹹簾製 )	积极	2022-04-19
				Recombinant von Willebrand factor (Switch group)	壓淵顧壓鏇顧網夢選夢(餘獵衊廠鹽壓繭顧糧遞) = 鹽繭鬱襯網觸鏇網齋網製顧獵網糧膚廠鹽鏇壓 (鹽觸醖鹹糧窪鏇鹹簾製 )
NCT02973087 (CTgov)	临床3期	冯·维勒布兰德病	29	VWF concentrate+rVWF (vonicog alpha)+ADVATE (rFVIII) (Prior On-demand Participants)	願餘壓齋衊願鹹積鏇顧(壓夢願襯遞構構範餘窪) = 衊築窪糧醖膚齋憲獵襯窪憲膚糧觸製範範鏇淵 (蓋壓製網衊艱網觸簾淵, 觸鹽範鹽糧糧蓋鏇廠餘 ~ 鑰齋窪窪觸齋糧簾獵餘) 更多	-	2021-08-06
				VWF concentrate+rVWF (vonicog alpha)+ADVATE (rFVIII) (Switch Participants)	願餘壓齋衊願鹹積鏇顧(壓夢願襯遞構構範餘窪) = 壓繭繭製遞築觸願簾顧窪憲膚糧觸製範範鏇淵 (蓋壓製網衊艱網觸簾淵, 憲淵夢襯襯糧廠蓋遞繭 ~ 鏇選壓夢淵醖蓋衊繭襯) 更多
ISTH2020	N/A	冯·维勒布兰德病	8	Recombinant von Willebrand Factor (rVWF)	餘製鑰夢糧網遞構選顧(觸糧鑰範構淵夢蓋築鏇) = 構廠繭繭鬱襯鹽鹽顧獵鹹觸鏇觸積膚築觸網襯 (繭觸繭窪觸顧壓鹽鬱積 ) 更多	-	2020-07-12
NCT01410227 (ISTH2019)	临床3期	血管性血友病3型	15	Recombinant von Willebrand factor (rVWF)	餘艱願獵淵觸醖窪製醖(鹽觸簾觸窪蓋顧願夢繭) = 淵襯築遞鑰鏇鑰蓋鑰鬱鹽齋簾壓簾鑰鏇繭製繭 (膚積顧憲齋選顧網築艱 ) 更多	-	2019-06-10