卡德凝血素(Catridecacog) - 药物靶点：fibrin_在研适应症：凝血因子XIII缺乏症，因子Xiii亚基缺乏，出血_专利_临床

概要

基本信息

药物类型

重组凝血因子

别名

Blood Coagulation Factor XIII (Synthetic Human A-Chain Precursor)、Catridecacog (Genetical Recombination)、Catridecacog (genetical recombination) (JAN)

+ [18]

靶点

fibrin

作用方式

调节剂

作用机制

fibrin调节剂(纤维蛋白调节剂)、凝血途径激活剂

治疗领域

遗传病与畸形血液及淋巴系统疾病其他疾病

在研适应症

凝血因子XIII缺乏症因子Xiii亚基缺乏出血

非在研适应症

溃疡性结肠炎凝血因子X缺乏症心脏病+ [1]

原研机构

Novo Nordisk A/S

在研机构

Novo Nordisk, Inc.

Novo Nordisk A/SNovo Nordisk Pharma Ltd.

+ [4]

非在研机构-

权益机构

ZymoGenetics, Inc.

Novo Nordisk A/S

最高研发阶段批准上市

首次获批日期

欧盟 (2012-09-03),

因子Xiii亚基缺乏出血

最高研发阶段(中国)-

特殊审评孤儿药 (美国)、孤儿药 (日本)

登录后查看时间轴

结构/序列

Sequence Code 140469735

来源: *****

关联

14

项与卡德凝血素相关的临床试验

NCT02670213

/ CompletedN/A

A Multicentre, Non-interventional Post Marketing Study of Safety and Effectiveness of NovoThirteenR (rFXIII) During Treatment of Congenital FXIII Deficiency in Japan

This study is conducted in Asia. Tha aim of this study is to investigate Safety and Effectiveness of NovoThirteen® (rFXIII) during treatment of congenital FXIII deficiency in Japan.

开始日期2016-03-16

申办/合作机构

Novo Nordisk A/S

NCT01862367

/ CompletedN/A

Use of rFXIII in Treatment of Congenital FXIII Deficiency, a Prospective Multi-centre Observational Study

This study is conducted globally. The aim of this observational study is to investigate the incidence of specific adverse drug reactions associated with the use of recombinant factor XIII (NovoThirteen®) in patients with congenital FXIII A-subunit deficiency (congenital FXIII deficiency), comprising FXIII antibodies, allergic reactions, embolic and thrombotic events and lack of therapeutic effect.
The study will aim at observing all patients exposed to NovoThirteen® in the EU, and additional patients from selected non-EU countries. Recombinant FXIII (rFXIII) is registered in EU and Switzerland as NovoThirteen® and in Canada as Tretten®.

开始日期2013-05-17

申办/合作机构

Novo Nordisk A/S

NCT01706159

/ Terminated临床2期

A Multicenter, Randomised, Double-blind, Placebo-controlled, Multiple-dose Trial With rFXIII Administered to Subjects With Mild to Moderate Active Ulcerative Colitis

This trial is conducted in Europe. The aim of the trial is to investigate the effect of recombinant factor XIII (rFXIII) administered to subjects with mild to moderate active ulcerative colitis (UC).

开始日期2012-10-01

申办/合作机构

Novo Nordisk A/S

100 项与卡德凝血素相关的临床结果

登录后查看更多信息

100 项与卡德凝血素相关的转化医学

登录后查看更多信息

100 项与卡德凝血素相关的专利（医药）

登录后查看更多信息

45

项与卡德凝血素相关的文献（医药）

2024-02-01·The journal of obstetrics and gynaecology research

Successful perinatal management of a woman with congenital factor XIII deficiency using recombinant factor XIII: A case report and literature review

Review

作者: Kasahara, Makiko ; Murakami, Takashi ; Ito, Yuya ; Takayama, Hiroshi ; Tsuji, Shunichiro ; Tokoro, Shinsuke

Abstract:

Factor XIII deficiency is an extremely rare autosomal recessive genetic disorder, occurring in 1 of 3–5 million people, and is associated with perinatal complications, such as habitual abortion and prolonged bleeding. Although plasma‐derived factor XIII (Fibrogamin®) carries a risk of infection and contains very low concentrated forms of factor XIII (FXIII) used for a pregnant woman with congenital coagulation factor XIII deficiency, recombinant factor XIII (rFXIII, Novo Thirteen®; Tretten®, Novo Nordisk, Bagsværd, Denmark), which has no risk of infection and is highly concentrated, has emerged as a novel formulation. Herein, we report the first case of a Japanese pregnant woman with congenital coagulation factor XIII deficiency successfully managed by rFXIII. She had a good perinatal course without pregnancy‐related complications and transfusion through the perinatal period.

2023-07-01·Blood Transfusion

A multicenter, real-world experience with recombinant FXIII for the treatment of patients with FXIII deficiency: from pharmacokinetics to clinical practice. The Italian FXIII Study.

Article

作者: Pollio, Berardino ; Biasoli, Chiara ; Molinari, Angelo C ; Simioni, Paolo ; Pizzuti, Michele ; Notarangelo, Lucia Dora ; Pea, Federico ; Zanon, Ezio ; Pasca, Samantha ; Peyvandi, Flora ; Cojutti, Piergiorgio ; Di Gregorio, Patrizia ; Ferretti, Antonietta ; Sottilotta, Gianluca

BACKGROUND:

Congenital factor XIII (FXIII) deficiency is a rare coagulation disorder characterized by muscular or mucocutaneous bleeding with life-threatening intracranial hemorrhages (ICHs), especially in cases with severe disease. The best treatment is the use of prophylactic plasma-derived or recombinant FXIII (rFXIII). Few data on the use of rFXIII in the real-world scenario are available. The main goal of this study was to assess the efficacy and safety of catridecacog (NovoThirteen^®) in a population of patients with FXIII deficiency. Other objectives were to compare the different pharmacokinetic (PK) profiles of each patient and to use them to create a tailored prophylaxis regimen.

MATERIALS AND METHODS:

We collected and analyzed all pharmacokinetic and clinical data in our registry of the patients with congenital FXIII deficiency treated with rFXIII at eleven Italian hemophilia centers. Data were collected from January 2019 to December 2020.

RESULTS:

Overall, data on 20 patients with FXIII deficiency were collected, 16 of whom presented with severe disease. Pharmacokinetics was assessed in 18 cases before starting prophylaxis. Prophylaxis was subsequently started in these patients using a wide range of dosages (25.0-80.0 IU/kg; mean 33.8 IU/kg) and infusion intervals (3.0-8.0 weeks). During a mean follow up of 47 months, two minor bleeds and one ICH in a severe patient who had remained under on-demand treatment were reported.

DISCUSSION:

Efficacy and safety of rFXIII were proven in all patients. The dosage and infusion timing for the treated patients sometimes differed to those reported in the MENTOR pivotal studies, thus underlying the importance of tailored management in a real-world scenario.

2022-11-01·Journal of thrombosis and thrombolysis

The pharmacokinetics of recombinant FXIII (catridecacog) from the MENTORTM2 trial to a real-world study: a head-to-head comparison

Article

作者: Pasca, Samantha ; Pea, Federico ; PierGiorgio, Cojutti

BACKGROUND:

FXIII deficiency is a very rare coagulation disorder that can affect equally males and females with an estimated incidence of 1 in 2 million persons worldwide. Due to this rarity, there are only few clinical and pharmacokinetic (PK) data deriving from the real-world.

AIM:

The aim of this report is to compare head-to-head the pharmacokinetic data of catridecacog derived from the MENTOR^TM2 trial with our real-world (RW) study.

METHODS:

The PK-profiles of all patients with FXIII deficiency treated with catridecacog at eleven Italian Hemophilia Centers were compared with PK data obtained by Kerlin et al. in the MENTOR^TM2.

RESULTS:

Overall 18 real-world PK were compared with 23 PK derived from the pivotal study. In the RW 55.6% of patients were females, 26.2% in the MENTOR^TM2 (p < 0.05). The mean dosage of drug used for the PK assessment was 35 IU/kg in the MENTOR^TM2, and 33.9 IU/kg in the RW study.

2

项与卡德凝血素相关的新闻（医药）

2022-09-01

·BioSpace

Novo Nordisk Shells Out $1.1B for Forma, Sickle Cell Drug

Novo Nordisk has entered into an agreement to acquire Forma Therapeutics for $1.1 billion, the company announced Thursday. The deal includes Forma’s lead development candidate, etavopivat. The drug is being assessed in the Phase II GLADIOLUS trial in patients with transfusion-dependent sickle cell disease (SCD) and thalassemia and in the Phase II/III HIBISCUS trial in SCD. Etavopivat is an oral, once-daily, selective pyruvate kinase-R (PKR) activator. It is being developed for anemia and red blood cell health in SCD patients. "By adding Forma’s differentiated approach to address unmet needs for patients, we are taking a step forward in enhancing our sickle cell disease pipeline," said Ludovic Helfgott, EVP and head of rare disease at Novo Nordisk, in a statement. Novo Nordisk is better known for its diabetes and obesity products, including Tresiba, Rybelsus, Victoza and Ozempic. It also has marketed products for hemophilia, such as NovoSeven (recombinant factor VIIa), Esperoct (turoctocog alfa pegol, N8-GP) and NovoThirteen (recombinant factor XIII). Frank D. Lee, president and CEO of Forma, called the announcement "an exciting milestone". “Novo Nordisk will partner closely with the sickle cell community to amplify our impact for patients around the world who urgently need new treatment options," he said in a statement. Forma’s board has unanimously approved the deal, which is expected to close in the fourth quarter of this year. In an email to BioSpace, Novo Nordisk said, "Novo Nordisk has a strong heritage and commitment of 40 years in rare blood disorders, initially within hemophilia and expanding to other rare blood disorders. The acquisition of Forma Therapeutics, including its lead candidate etavopivat, enriches and accelerates the development of Novo Nordisk's preclinical and clinical pipeline in haemoglobinopathies such as sickle cell disease and thalassemia." The deal is for $20 per share in cash, a total equity value of $1.1 billion. This amounts to a 92% premium for Forma’s volume-weighted average price per share over the last 30 days ending August 31. Forma also has programs underway in prostate cancer, acute myeloid leukemia and glioma and preclinical programs in hematological diseases and cancer. On August 2, Forma signed an exclusive, worldwide license deal with Rigel Pharmaceuticals for olutasidenib for relapsed or refractory acute myeloid leukemia. The FDA has accepted Forma’s New Drug Application for the drug with a target action date of February 15, 2023. Rigel paid Forma $2 million upfront. Forma is eligible for another $17.5 million based on various milestone payments, as well as future development and commercial milestones of $215.5 million and tiered royalties in the low-teens to mid-thirties.

并购

2022-09-01

·BioSpace

Novo Nordisk Shells Out $1.1B for Forma, Sickle Cell Drug

Courtesy of joreks/Getty Images Novo Nordisk has entered into an agreement to acquire Forma Therapeutics for $1.1 billion. Forma’s lead development candidate, etavopivat, is being developed for sickle cell disease. Novo Nordisk has entered into an agreement to acquire Forma Therapeutics for $1.1 billion, the company announced Thursday. The deal includes Forma’s lead development candidate, etavopivat. The drug is being assessed in the Phase II GLADIOLUS trial in patients with transfusion-dependent sickle cell disease (SCD) and thalassemia and in the Phase II/III HIBISCUS trial in SCD. Etavopivat is an oral, once-daily, selective pyruvate kinase-R (PKR) activator. It is being developed for anemia and red blood cell health in SCD patients. “By adding Forma’s differentiated approach to address unmet needs for patients, we are taking a step forward in enhancing our sickle cell disease pipeline,” said Ludovic Helfgott, EVP and head of rare disease at Novo Nordisk, in a statement. Novo Nordisk is better known for its diabetes and obesity products, including Tresiba, Rybelsus, Victoza and Ozempic. It also has marketed products for hemophilia, such as NovoSeven (recombinant factor VIIa), Esperoct (turoctocog alfa pegol, N8-GP) and NovoThirteen (recombinant factor XIII). Frank D. Lee, president and CEO of Forma, called the announcement “an exciting milestone”. “Novo Nordisk will partner closely with the sickle cell community to amplify our impact for patients around the world who urgently need new treatment options,” he said in a statement. Forma’s board has unanimously approved the deal, which is expected to close in the fourth quarter of this year. In an email to BioSpace, Novo Nordisk said, “Novo Nordisk has a strong heritage and commitment of 40 years in rare blood disorders, initially within hemophilia and expanding to other rare blood disorders. The acquisition of Forma Therapeutics, including its lead candidate etavopivat, enriches and accelerates the development of Novo Nordisk’s preclinical and clinical pipeline in haemoglobinopathies such as sickle cell disease and thalassemia.” The deal is for $20 per share in cash, a total equity value of $1.1 billion. This amounts to a 92% premium for Forma’s volume-weighted average price per share over the last 30 days ending August 31. Forma also has programs underway in prostate cancer, acute myeloid leukemia and glioma and preclinical programs in hematological diseases and cancer. On August 2, Forma signed an exclusive, worldwide license deal with Rigel Pharmaceuticals for olutasidenib for relapsed or refractory acute myeloid leukemia. The FDA has accepted Forma’s New Drug Application for the drug with a target action date of February 15, 2023. Rigel paid Forma $2 million upfront. Forma is eligible for another $17.5 million based on various milestone payments, as well as future development and commercial milestones of $215.5 million and tiered royalties in the low-teens to mid-thirties.

临床2期引进/卖出并购上市批准

100 项与卡德凝血素相关的药物交易

登录后查看更多信息

外链

KEGG	Wiki	ATC	Drug Bank
D10532	卡德凝血素	B02BD11	DB09310

研发状态

批准上市

10 条最早获批的记录,

登录

后查看更多信息

适应症	国家/地区	公司	日期
凝血因子XIII缺乏症	瑞士	Novo Nordisk Pharma AG	2012-10-25
因子Xiii亚基缺乏	欧盟	Novo Nordisk A/S	2012-09-03
因子Xiii亚基缺乏	冰岛	Novo Nordisk A/S	2012-09-03
因子Xiii亚基缺乏	列支敦士登	Novo Nordisk A/S	2012-09-03
因子Xiii亚基缺乏	挪威	Novo Nordisk A/S	2012-09-03
出血	欧盟	Novo Nordisk A/S	2012-09-03
出血	冰岛	Novo Nordisk A/S	2012-09-03
出血	列支敦士登	Novo Nordisk A/S	2012-09-03
出血	挪威	Novo Nordisk A/S	2012-09-03

未上市

10 条进展最快的记录,

登录

后查看更多信息

适应症	最高研发状态	国家/地区	公司	日期
溃疡性结肠炎	临床2期	保加利亚	Novo Nordisk A/S	2012-10-01
溃疡性结肠炎	临床2期	克罗地亚	Novo Nordisk A/S	2012-10-01
溃疡性结肠炎	临床2期	丹麦	Novo Nordisk A/S	2012-10-01
溃疡性结肠炎	临床2期	匈牙利	Novo Nordisk A/S	2012-10-01
溃疡性结肠炎	临床2期	波兰	Novo Nordisk A/S	2012-10-01
溃疡性结肠炎	临床2期	俄罗斯	Novo Nordisk A/S	2012-10-01
溃疡性结肠炎	临床2期	乌克兰	Novo Nordisk A/S	2012-10-01
炎症	临床2期	保加利亚	Novo Nordisk A/S	2012-10-01
炎症	临床2期	克罗地亚	Novo Nordisk A/S	2012-10-01
炎症	临床2期	丹麦	Novo Nordisk A/S	2012-10-01

登录后查看更多信息

临床结果

适应症

分期

评价

查看全部结果

研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT01862367 (Pubmed) 人工标引	N/A	凝血因子XIII缺乏症	30	Catridecacog	繭製鬱顧夢鑰顧襯蓋夢(鏇廠糧襯鑰淵願築鏇壓) = All 10 SAEs were unlikely related to rFXIII-A2 糧獵繭構鑰遞醖簾顧簾 (獵製餘築繭壓壓廠遞襯 ) 更多	积极	2022-02-27
ISTH2021	N/A	凝血因子XIII缺乏症	20	catridecacog	鹹鏇願觸憲繭餘鏇遞範(觸顧衊餘鏇衊壓簾壓鏇) = 遞獵顧積憲艱築窪鬱襯壓遞願廠顧鏇構觸窪鹹 (鹹廠憲衊壓製壓衊壓顧 ) 更多	积极	2021-07-17
ISTH2020	临床4期	因子Xiii亚基缺乏	-	rFXIII-A2	鏇鹹衊鑰窪築網繭鑰壓(繭夢繭範窪願網艱積齋) = 積製鏇齋觸淵壓鹹夢願淵糧窪鹽鹽蓋獵鬱選網 (鏇壓窪淵廠鏇憲襯構願 )	-	2020-07-12
NCT00978380 (Pubmed \| Thromb Haemost) 人工标引	临床3期	因子Xiii亚基缺乏	60	rFXIII-A2	廠鬱醖網糧鏇簾獵鏇觸(鑰製選壓醖鹹選範網構) = without any safety concerns 鬱鑰餘鏇鹹糧網製積繭 (鬱齋鹹壓鏇艱鑰範鏇膚 ) 更多	积极	2018-03-01
NCT01253811 (mentor™5, Pubmed \| J Thromb Haemost) 人工标引	临床3期	营养不良	6	Catridecacog	鑰鹹衊夢壓淵鬱遞獵繭(簾鹹壓壓衊艱鹹選憲鏇) = Two serious adverse events, unrelated to rFXIII, were reported in a single child, each related to head injury, and neither resulting in intracranial hemorrhage. 艱餘窪網窪鬱積觸願衊 (齋鏇選顧廠選獵積繭鏇 ) 更多	积极	2017-08-01
NCT00978380 (mentor™2 \| mentor(™) 2, CTgov)	临床3期	凝血因子XIII缺乏症 \| 因子Xiii亚基缺乏	63	Recombinant Factor XIII (rFXIII) (Recombinant Factor XIII (rFXIII))	簾顧選襯鬱衊廠範艱獵 = 夢獵餘齋觸淵鑰鑰醖觸願顧鑰製願衊簾衊醖鹹 (糧鏇壓糧餘鹽獵鹹築鑰, 醖積製遞鬱艱鬱膚淵鹽 ~ 願夢顧製觸壓夢積鹹窪) 更多	-	2016-12-13
NCT00978380 (mentor™2 \| mentor(™) 2, CTgov)	临床3期	凝血因子XIII缺乏症 \| 因子Xiii亚基缺乏	63	rFXIII Avecia+rFXIII (rFXIII Novo Nordisk)	餘鏇積積願淵襯構鏇遞(鏇顧膚選壓艱顧糧鬱壓) = 蓋餘鑰鑰糧選膚築獵廠淵積簾夢廠觸衊構顧艱 (鏇鏇淵窪淵衊積獵顧鏇, 遞遞顧餘積願膚選鹹遞 ~ 鹹願醖築醖願壓糧襯憲) 更多	-	2016-12-13
NCT01253811 (mentor™5, CTgov)	临床3期	因子Xiii亚基缺乏	6	catridecacog	觸構製繭窪艱蓋鹹構構 = 觸糧鏇膚壓襯鬱壓遞鹹艱鹽製壓艱憲夢鏇廠窪 (鬱築襯鹹積衊獵築襯鏇, 廠夢網襯觸觸遞簾淵築 ~ 憲襯憲構壓鹹蓋願憲蓋) 更多	-	2016-06-24
NCT00978380 (mentor(™) 2, Pubmed \| J Thromb Haemost)	临床3期	因子Xiii亚基缺乏	23	rFXIII	鏇鬱膚蓋觸鏇夢積顧襯(窪壓簾鏇淵積鹽觸壓積) = 衊選淵鹽鬱鬱蓋窪鬱糧齋網廠壓鬱糧憲齋製衊 (醖淵廠淵鏇願窪淵窪窪 ) 更多	-	2014-12-01
NCT00914589 (CTgov)	临床2期	心脏病 \| 凝血障碍	479	placebo (Placebo)	觸膚選衊鹹鬱膚壓製鹹 = 憲鑰壓艱簾蓋壓鏇糧糧鏇壓窪襯膚選壓鑰糧廠 (餘繭餘窪觸構鹽膚鏇憲, 願繭窪廠鏇糧願鏇網廠 ~ 鑰蓋觸顧膚窪齋願壓壓) 更多	-	2014-11-13
NCT00914589 (CTgov)	临床2期	心脏病 \| 凝血障碍	479	catridecacog (FXIII17.5IU/Kg)	觸膚選衊鹹鬱膚壓製鹹 = 顧製構遞築積積簾憲網鏇壓窪襯膚選壓鑰糧廠 (餘繭餘窪觸構鹽膚鏇憲, 積獵衊醖膚淵願鏇糧鏇 ~ 製鹽憲淵遞選簾鹽願製) 更多	-	2014-11-13
NCT01706159 (CTgov)	临床2期	炎症 \| 溃疡性结肠炎	20	catridecacog (rFXIII)	衊糧廠壓壓鑰構膚範積 = 顧鹹獵築選鹹製網顧網簾齋鏇築齋窪醖衊淵鑰 (夢餘窪鹹願蓋蓋醖鏇艱, 顧憲齋築繭簾鹹鏇顧蓋 ~ 鬱襯鏇齋鑰選選淵蓋選) 更多	-	2014-08-05
NCT01706159 (CTgov)	临床2期	炎症 \| 溃疡性结肠炎	20	placebo (Placebo)	衊糧廠壓壓鑰構膚範積 = 遞艱築觸遞網顧憲淵醖簾齋鏇築齋窪醖衊淵鑰 (夢餘窪鹹願蓋蓋醖鏇艱, 獵壓餘蓋簾獵網襯鏇糧 ~ 鏇憲廠遞醖觸顧餘窪簾) 更多	-	2014-08-05