预约演示
更新于：2025-09-01
Rurioctocog alfa pegol
培重组人凝血因子Ⅷ
更新于：2025-09-01
概要
基本信息
原研机构 |
最高研发阶段批准上市 |
首次获批日期 美国 (2015-11-13), |
最高研发阶段(中国)申请上市 |
特殊审评- |
登录后查看时间轴
结构/序列
Sequence Code 26728
来源: *****
关联
19
项与 培重组人凝血因子Ⅷ 相关的临床试验NCT05707351
A Phase 3, Prospective, Multicenter, Open-label Study of Efficacy, Safety, and Pharmacokinetics of PEGylated Recombinant Factor VIII (ADYNOVATE) Administered for Prophylaxis and Treatment of Bleeding in Chinese Previously Treated Patients With Severe Hemophilia A (FVIII <1%)
NCT05042440
A Phase 1, Single-Site, Open-Label Study to Assess Pharmacokinetics of Efanesoctocog Alfa (BIVV001), Standard Half-Life and Extended Half-Life FVIII After Each Single Intravenous Injection in a Fixed Sequence, in Previously Treated Adults With Severe Hemophilia A
NCT04941898
Adynovate Special Drug Use Result Survey (Perioperative Administration)
100 项与 培重组人凝血因子Ⅷ 相关的临床结果
登录后查看更多信息
100 项与 培重组人凝血因子Ⅷ 相关的转化医学
登录后查看更多信息
100 项与 培重组人凝血因子Ⅷ 相关的专利（医药）
登录后查看更多信息
67
项与 培重组人凝血因子Ⅷ 相关的文献（医药）2025-09-01THROMBOSIS RESEARCH
Real-world safety and effectiveness of rurioctocog alfa pegol in 338 patients with hemophilia A in South Korea: A postmarketing surveillance study
Article
作者: You, Chur Woo ; Kim, Eungsun ; Choi, Eun Jin ; Jung, Aeran ; Kim, Soyon ; Kim, Ji Yoon ; Park, Young-Shil ; Hwang, Taiju ; Park, Sang Kyu ; Yoo, Ki-Young
INTRODUCTION:
Rurioctocog alfa pegol is a recombinant coagulation factor VIII (FVIII) with an extended half-life versus the parent product. Limited real-world data exist on the treatment of hemophilia A with rurioctocog alfa pegol in South Korea. This postmarketing surveillance study assessed its safety and effectiveness in a real-world setting, according to Korean regulatory requirements.
METHODS:
In this prospective, multicenter study (NCT03824522), data were collected from medical records of patients, including children, with hemophilia A receiving rurioctocog alfa pegol as standard clinical practice in South Korea. Safety (adverse events [AEs], adverse drug reactions [ADRs], and unexpected AEs/ADRs) and hemostatic effectiveness were analyzed.
RESULTS:
In total, 338 patients were included (mean age [range], 25.0 [1.0-61.0] years; children <12 years, n = 54 [16 %]; all had a history of FVIII treatment). AEs occurred in 20 (5.9 %) patients (unexpected AEs, n = 18 [5.3 %]; ADRs, n = 4 [1.2 %]; unexpected ADRs, n = 2 [0.6 %]; serious AEs, n = 4 [1.2 %]; serious ADRs, n = 0 [0.0 %]). Among children, AEs occurred in 1 (50 %) and 6 (11.5 %) patients aged <2 and ≥2 to <12 years, respectively. No new safety signals were observed. No bleeding events occurred in 239 (76.1 %) patients on prophylaxis. Hemostatic effectiveness was rated by physicians as excellent/good in most patients. Bleeding events were managed with 1-2 infusions. Treatment effectiveness was maintained in children during the study period. No patients developed inhibitor antibodies against rurioctocog alfa pegol.
CONCLUSION:
Rurioctocog alfa pegol was effective in children and adults, with no new safety signals; most patients on prophylaxis experienced no bleeds during the study.
2025-05-01ADVANCES IN THERAPY
Efficacy and Safety of Recombinant Factor VIII in Previously Untreated and Previously Treated Children with Hemophilia A: A Systematic Review
Review
作者: Sun, Jing ; Wang, Zhenguo ; Zhou, Xuan ; Feng, Xiaoqin
INTRODUCTION:
Inhibitor development is a primary concern for pediatric patients with hemophilia A (HA) undergoing recombinant factor VIII (rFVIII) therapy, yet relevant research is lacking. We aimed to compare the efficacy and safety of standard (SHL) and extended half-life (EHL) rFVIII products in previously treated (PTPs) and untreated (PUPs) pediatric patients with HA.
METHODS:
Following PRISMA guidelines, we searched clinical studies from PubMed, Embase, and Cochrane Library. Data were extracted and a single-arm meta-analysis was performed.
RESULTS:
This systematic review included 16 studies involving 1145 patients. Three studies reported changes in annual bleeding rate (ABR); their results displayed no statistically significant difference in ABR changes in pediatric patients with HA after rFVIII treatment. Ten studies reported inhibitor development, nine focused on PUPs. Here, EHL rFVIII showed a proportion of inhibitors at 27.5% (95% confidence interval [CI] 22.6%; 32.6%), and third-generation SHL rFVIII showed a proportion of inhibitors at 36.4% (27.2%; 46.2%), with a high-titer proportion of 20.9% (12.9%; 30.3%) for the latter. Both SHL rFVIII (octocog alfa) and EHL rFVIII (rurioctocog alfa pegol) presented low proportions of inhibitor development. Octocog alfa exhibited the lowest high-titer inhibitor incidence, marked at 12.7% (5.3%; 24.5%). Eleven studies addressed adverse events (AEs), with octocog alfa showing low reported treatment-related AEs at a proportion of 14.5% (6.5%; 26.7%).
CONCLUSION:
Our analysis revealed that both octocog alfa and rurioctocog alfa pegol showed low inhibitor development, with octocog alfa having few treatment-related AEs. Regular monitoring for inhibitors during rFVIII therapy is important.
2025-01-01ADVANCES IN THERAPY
Efanesoctocog Alfa versus Standard and Extended Half-Life Factor VIII Prophylaxis in Adolescent and Adult Patients with Haemophilia A without Inhibitors
Review
作者: Klamroth, Robert ; Kragh, Nana ; Wojciechowski, Piotr ; Margas, Wojciech ; Bystrická, Linda ; Arnaud, Alix ; Wdowiak, Marlena ; Guyot, Patricia ; Wilson, Amanda ; Tosetto, Alberto
INTRODUCTION:
In the Phase 3 XTEND-1 trial, (NCT04161495) efanesoctocog alfa prophylaxis provided superior bleed protection versus pre-study factor VIII (FVIII) replacement therapy in patients with severe haemophilia A. The aim of this study was to indirectly compare bleed outcomes between efanesoctocog alfa and standard/extended half-life (SHL and EHL) FVIII replacement therapies in adolescent and adult patients with severe haemophilia A without inhibitors.
METHODS:
A systematic literature review was conducted to identify Phase 3 trials of EHL and SHL FVIII replacement therapies for comparison with efanesoctocog alfa data from XTEND-1. Matching-adjusted indirect comparisons were used to compare annualised bleeding rates (ABRs) for any, treated, joint, and spontaneous bleeds between efanesoctocog alfa and comparators. The estimates from respective comparisons were pooled using random-effect meta-analyses to evaluate the overall difference between efanesoctocog alfa and comparator therapies.
RESULTS:
Four EHL therapies (rurioctocog alfa pegol, efmoroctocog alfa, turoctocog alfa pegol, damoctocog alfa pegol) and two octocog alfa SHL therapies were included. In meta-analyses, efanesoctocog alfa was associated with significantly lower ABRs for any [mean difference (95% CI) - 2.24 ( - 3.24; - 1.25)], spontaneous [ - 1.52 ( - 2.33; - 0.72)], and joint bleeds [ - 1.60 ( - 2.32; - 0.88)] versus EHL therapies, and with significantly lower ABRs for any [ - 3.61 ( - 4.43; - 2.79)], treated [ - 1.55 ( - 1.89; - 1.20)], spontaneous [ - 2.52 ( - 3.31; - 1.72)], and joint bleeds [ - 3.42 ( - 4.77; - 2.08)] versus SHL therapies.
CONCLUSION:
Efanesoctocog alfa was associated with significantly lower ABRs (any, spontaneous and joint) compared with EHL or SHL prophylaxis therapies. Patients had, on average, 2.2 and 3.6 fewer bleeds per year versus EHL and SHL therapies, respectively.
100 项与 培重组人凝血因子Ⅷ 相关的药物交易
登录后查看更多信息
研发状态
批准上市
10 条最早获批的记录, 后查看更多信息
登录
| 适应症 | 国家/地区 | 公司 | 日期 |
|---|---|---|---|
| 凝血因子缺乏症 | 日本 | 2017-12-05 | |
| 血友病A | 美国 | 2015-11-13 |
未上市
10 条进展最快的记录, 后查看更多信息
登录
| 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 | 2013-10-15 | |
| 出血 | 临床3期 | 美国 | 2013-10-15 | |
| 出血 | 临床3期 | 日本 | 2013-10-15 | |
| 出血 | 临床3期 | 日本 | 2013-10-15 | |
| 出血 | 临床3期 | 澳大利亚 | 2013-10-15 | |
| 出血 | 临床3期 | 澳大利亚 | 2013-10-15 | |
| 出血 | 临床3期 | 奥地利 | 2013-10-15 | |
| 出血 | 临床3期 | 奥地利 | 2013-10-15 | |
| 出血 | 临床3期 | 保加利亚 | 2013-10-15 | |
| 出血 | 临床3期 | 保加利亚 | 2013-10-15 |
登录后查看更多信息
临床结果
临床结果
适应症
分期
评价
查看全部结果
临床3期 | 120 | 製壓選壓夢願製繭遞夢 = 淵製齋餘鏇範夢憲憲鬱 鏇鹹遞膚構憲鬱壓壓網 (簾鑰鏇夢鑰糧構製醖積, 積淵壓憲鬱壓壓獵鑰鬱 ~ 築淵廠鑰獵憲艱鹹積膚) 更多 | - | 2025-07-28 | |||
临床3期 | 37 | 糧襯餘簾繭網選鹽夢顧(鏇淵願蓋壓夢齋餘願憲) = 範構選製遞衊構夢淵蓋 觸顧廠憲醖廠願夢夢壓 (鏇範醖獵窪鹹蓋積範夢, 13.61) 更多 | - | 2025-05-01 | |||
临床1期 | 血友病A FVIII | 13 | 鑰壓糧選鹽遞壓膚鏇窪(鏇襯構願廠糧網夢製製) = 窪鹽範選鏇網築鬱鹽獵 艱積鹹鬱製艱膚觸願鹽 (製獵襯齋鹽簾遞製網積 ) 更多 | 积极 | 2024-05-14 | ||
鑰壓糧選鹽遞壓膚鏇窪(鏇襯構願廠糧網夢製製) = 簾顧選襯膚窪蓋衊構選 艱積鹹鬱製艱膚觸願鹽 (製獵襯齋鹽簾遞製網積 ) 更多 | |||||||
临床3期 | 80 | 襯簾淵顧觸積齋餘鑰糧(構窪繭鹽醖鑰願遞壓鏇) = Thirteen patients experienced 14 treatment-related adverse events, including 10 cases of FVIII inhibitor development 襯膚醖艱餘鏇齋淵鬱淵 (衊蓋範淵鏇鹹鑰網願蓋 ) | - | 2023-10-03 | |||
临床2/3期 | 10 | 餘夢積構鏇獵簾構壓願(願襯壓鏇艱憲艱憲鑰鹹) = 鹹齋艱簾簾夢衊襯範衊 憲壓齋願鹽繭鹽鹹遞築 (齋積簾簾遞膚積積餘顧 ) | 积极 | 2019-09-01 | |||
临床3期 | 135 | (BAX 855-Low Level) | 構窪積遞襯艱襯夢繭積 = 網齋襯積築廠廠製廠網 觸艱網遞餘築鑰選鹹製 (糧廠鑰衊選膚鑰積獵鏇, 鹽憲衊淵選壓鏇齋膚廠 ~ 艱網築繭製製衊簾窪膚) 更多 | - | 2019-08-26 | ||
(BAX 855-High Level) | 構窪積遞襯艱襯夢繭積 = 鹹築鏇鑰醖襯鑰廠襯遞 觸艱網遞餘築鑰選鹹製 (糧廠鑰衊選膚鑰積獵鏇, 糧獵醖蓋餘糧網選膚餘 ~ 壓夢餘淵鏇願製願夢襯) 更多 | ||||||
临床3期 | 216 | 衊製鑰築壓製鹹鑰壓膚(憲醖繭觸鑰遞蓋醖範觸) = a non-serious mild AE that resolved ~3-4 weeks after study end 獵蓋積鬱衊鏇壓襯壓壓 (鏇製壓鏇鹹繭襯構膚窪 ) | - | 2019-06-10 | |||
临床3期 | 216 | SHP660 prophylaxis | 蓋繭願繭範獵築淵鑰齋(壓遞淵觸繭衊蓋鑰壓網) = 築鑰範構蓋壓夢糧醖製 鑰憲構壓衊蓋窪襯獵繭 (襯鏇築製構衊蓋窪衊醖 ) 更多 | - | 2019-06-10 | ||
临床3期 | 218 | (BAX 855: Age < 2 Years) | 憲夢鹽齋蓋願範蓋範廠 = 淵蓋餘蓋鏇觸淵築夢鑰 醖築壓窪鏇蓋觸顧積獵 (艱鬱餘淵願蓋壓膚願淵, 網衊積憲糧獵糧糧齋遞 ~ 獵艱齋淵壓壓獵壓積積) 更多 | - | 2019-05-01 | ||
(BAX 855: Age >= 2 to <12 Years) | 憲夢鹽齋蓋願範蓋範廠 = 艱鑰繭顧鬱膚簾窪範網 醖築壓窪鏇蓋觸顧積獵 (艱鬱餘淵願蓋壓膚願淵, 艱憲淵糧淵廠淵憲繭鑰 ~ 製簾壓鬱憲積糧艱選鏇) 更多 | ||||||
临床3期 | 31 | polyethylene glycol-ylated FVIII | 膚網餘鹹齋鏇鹽餘壓願(構膚簾夢衊齋糧鹽襯鏇) = 壓糧艱艱鹽艱遞鑰構壓 鏇衊遞繭遞夢製壓鑰鹽 (獵蓋構遞鹹夢壓淵範蓋 ) | 积极 | 2017-03-01 |
登录后查看更多信息
转化医学
使用我们的转化医学数据加速您的研究。
登录
或
药物交易
使用我们的药物交易数据加速您的研究。
登录
或
核心专利
使用我们的核心专利数据促进您的研究。
登录
或
临床分析
紧跟全球注册中心的最新临床试验。
登录
或
批准
利用最新的监管批准信息加速您的研究。
登录
或
生物类似药
生物类似药在不同国家/地区的竞争态势。请注意临床1/2期并入临床2期，临床2/3期并入临床3期
登录
或
特殊审评
只需点击几下即可了解关键药物信息。
登录
或
Eureka LS:
全新生物医药AI Agent 覆盖科研全链路，让突破性发现快人一步
立即开始免费试用!
智慧芽新药情报库是智慧芽专为生命科学人士构建的基于AI的创新药情报平台，助您全方位提升您的研发与决策效率。
立即开始数据试用!
智慧芽新药库数据也通过智慧芽数据服务平台，以API或者数据包形式对外开放，助您更加充分利用智慧芽新药情报信息。
生物序列数据库
生物药研发创新
免费使用
化学结构数据库
小分子化药研发创新
免费使用