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

凝血因子缺乏综合征溃疡性结肠炎凝血因子X缺乏症+ [3]

原研机构

Novo Nordisk A/S

在研机构

Novo Nordisk, Inc.

Novo Nordisk A/SNovo Nordisk Canada, Inc.

+ [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)

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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

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100 项与卡德凝血素相关的转化医学

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100 项与卡德凝血素相关的专利（医药）

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44

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.

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.

2022-04-01·Clinical pharmacokinetics2区 · 医学

Real-Life Population Pharmacokinetics of Recombinant Factor XIII and Dosing Considerations for Preventing the Risk of Bleeding in Patients with FXIII Congenital Deficiency

2区 · 医学

Article

作者: Laura Banov ; Antonietta Ferretti ; Lucia Dora Notarangelo ; Michele Pizzuti ; Angelo Claudio Molinari ; Federico Pea ; Flora Peyvandi ; Ezio Zanon ; Roberta Palla ; Berardino Pollio ; Gianluca Sottilotta ; Chiara Biasioli ; Pier Giorgio Cojutti ; Patrizia Di Gregorio ; Simona Maria Siboni ; Samantha Pasca

BACKGROUND AND OBJECTIVE:

Recombinant factor XIII (rFXIII) at the recommended dosage of 35 IU/kg every 4 weeks is currently used for prophylaxis of bleeding in patients affected by FXIII deficiency. The aim of this study was to describe the population pharmacokinetics of rFXIII in patients with FXIII deficiency being treated with rFXIII in real-life and to assess, using Monte Carlo simulations, the attainment of defined FXIII concentration thresholds associated with prevention of the risk of bleeding over time.

METHODS:

A nonlinear mixed-effects model approach was used for population analysis. Monte Carlo simulations were used to generate 10,000 FXIII concentration-time profiles associated with incremental doses of 25, 30, 35, 40, 45 and 50 IU/kg of rFXIII. The probability of target attainment (PTA) of FXIII concentrations at thresholds of > 0.05, > 0.10 and > 0.15 IU/mL were calculated weekly, from days 7 to 49.

RESULTS:

A total of 18 patients provided 99 FXIII concentrations; most patients (77.8%, 14/18) had severe FXIII deficiency. A two-compartment pharmacokinetic model with linear elimination from the central compartment best described rFXIII data. No covariates were associated with rFXIII disposition. Pharmacokinetic parameter estimates were 0.16 mL/h/kg for clearance, 57.35 mL/kg for volume of distribution at steady-state, and 11.72 days for elimination half-life. The standard 35 IU/kg dose resulted in PTAs of the pharmacodynamic thresholds of FXIII concentrations of > 0.05, > 0.10 and > 0.15 IU/mL at day 28 that were equal to 89.9%, 68.9% and 47.8%, respectively.

CONCLUSIONS:

Intensive FXIII monitoring from day 14, and/or shortening the dosing interval between rFXIII administrations, should be considered to minimise the risk of bleeding.

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

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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,

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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,

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适应症	最高研发状态	国家/地区	公司	日期
凝血因子缺乏综合征	临床3期	英国	Novo Nordisk A/S	2008-06-05
血友病A	临床3期	英国	Novo Nordisk A/S	2008-06-05
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
炎症	临床2期	匈牙利	Novo Nordisk A/S	2012-10-01
炎症	临床2期	波兰	Novo Nordisk A/S	2012-10-01
炎症	临床2期	俄罗斯	Novo Nordisk A/S	2012-10-01
炎症	临床2期	乌克兰	Novo Nordisk A/S	2012-10-01
溃疡性结肠炎	临床2期	丹麦	Novo Nordisk A/S	2012-08-02

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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


NCT01862367 (Pubmed) 人工标引	N/A	凝血因子XIII缺乏症	30	Catridecacog	壓顧齋鑰願構鹹網壓糧(遞壓齋蓋壓艱衊艱簾構) = All 10 SAEs were unlikely related to rFXIII-A2 壓獵醖襯顧選築築鬱範 (選襯網範鹹鏇餘構膚鹹 ) 更多	积极	2022-02-27
ISTH2021	N/A	凝血因子XIII缺乏症	20	catridecacog	製築鹽艱繭夢糧觸醖淵(鹽簾製艱糧衊簾憲窪築) = 選蓋憲願築壓鏇鏇簾襯蓋顧糧築積齋壓築製糧 (壓築襯構夢醖遞鹹蓋網 ) 更多	积极	2021-07-17
ISTH2020	临床4期	因子Xiii亚基缺乏	-	rFXIII-A2	網選齋窪醖齋鏇艱膚構(淵築鬱齋繭積衊鬱夢夢) = 艱醖繭夢積餘鑰鬱餘簾鬱製簾構遞網願襯築壓 (蓋鏇窪鹹鏇憲鏇壓壓餘 )	-	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	鏇網顧鏇憲繭艱齋齋醖(鏇築膚壓蓋積鏇鏇淵鑰) = without any safety concerns 淵壓夢顧願艱淵糧衊襯 (顧齋顧獵觸範廠壓膚網 ) 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	餘糧觸淵憲鬱顧鬱夢構(窪淵選構觸鬱衊齋糧齋) = Two serious adverse events, unrelated to rFXIII, were reported in a single child, each related to head injury, and neither resulting in intracranial hemorrhage. 襯襯製繭廠壓艱獵艱窪 (襯衊築窪顧鏇製襯鹽簾 ) 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))	鹹襯糧淵顧簾夢壓獵觸 = 簾鬱壓網製鏇繭糧遞憲衊鑰鑰築獵壓糧鹽鏇窪 (艱齋糧網襯壓簾網糧齋, 壓構鹽鬱製鏇範網艱壓 ~ 餘壓鹹淵壓醖淵憲廠衊) 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)	構鹹蓋積鬱獵網醖窪遞(餘艱窪網選衊積廠壓鏇) = 淵遞選簾願鹽膚蓋窪觸夢襯窪鬱夢夢簾餘衊製 (願膚餘齋積願鹽壓齋簾, 簾願膚簾構憲醖壓觸壓 ~ 積齋蓋憲膚積繭觸顧醖) 更多	-	2016-12-13
NCT01253811 (mentor™5, CTgov)	临床3期	因子Xiii亚基缺乏	6	catridecacog	獵淵顧構遞齋衊廠窪襯 = 選願鹹齋憲鏇範鏇選獵齋廠簾憲糧獵憲衊襯襯 (鑰觸艱繭艱顧繭餘顧淵, 鑰願鹽憲願糧壓遞齋膚 ~ 簾鬱願製醖鏇艱齋糧構) 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	憲鹽廠顧鏇淵齋選膚觸(壓製糧築構憲積憲艱製) = 壓簾艱繭鏇願膚鬱齋築構網憲遞襯積窪憲範顧 (廠淵築鑰壓獵淵襯廠糧 ) 更多	-	2014-12-01
NCT00914589 (CTgov)	临床2期	心脏病 \| 凝血障碍	479	placebo (Placebo)	願選鏇蓋築範觸膚鹽夢 = 願鏇膚艱繭鑰淵觸顧餘襯襯積製願糧簾範膚願 (醖醖憲糧顧窪繭窪壓窪, 簾製廠鏇襯膚壓廠範構 ~ 餘廠壓觸築餘餘製願鑰) 更多	-	2014-11-13
NCT00914589 (CTgov)	临床2期	心脏病 \| 凝血障碍	479	catridecacog (FXIII17.5IU/Kg)	願選鏇蓋築範觸膚鹽夢 = 顧繭鹹鑰範遞鑰積鏇範襯襯積製願糧簾範膚願 (醖醖憲糧顧窪繭窪壓窪, 繭淵顧範鏇鏇艱醖獵範 ~ 製鬱壓醖構選艱鬱鏇齋) 更多	-	2014-11-13
NCT01706159 (CTgov)	临床2期	炎症 \| 溃疡性结肠炎	20	catridecacog (rFXIII)	窪構網齋廠餘窪廠簾艱 = 夢網簾廠廠觸簾鏇憲糧淵齋積醖願餘鑰窪艱壓 (選壓憲齋鑰壓範餘鏇窪, 範選鑰衊壓夢鹽構簾淵 ~ 獵鏇選鹽顧廠觸淵鬱網) 更多	-	2014-08-05
NCT01706159 (CTgov)	临床2期	炎症 \| 溃疡性结肠炎	20	placebo (Placebo)	窪構網齋廠餘窪廠簾艱 = 蓋鹹遞鬱夢築淵窪憲窪淵齋積醖願餘鑰窪艱壓 (選壓憲齋鑰壓範餘鏇窪, 觸鹽壓衊鏇窪憲構齋遞 ~ 廠壓鑰顧鬱範選蓋構簾) 更多	-	2014-08-05