Antihemophilic factor/von Willebrand factor complex(Grifols SA)(Antihemophilic factor/von Willebrand factor complex(Grifols SA)) - 药物靶点：F10_在研适应症：获得性因子 VIII缺乏症，血友病A，冯·维勒布兰德病_专利_临床

概要

基本信息

药物类型

非重组凝血因子

别名

Alphanate、Factor VIII/von Willebrand factor、Fanhdi

+ [1]

靶点

F10

作用方式

刺激剂

作用机制

F10刺激剂(凝血因子X刺激剂)、凝血途径激活剂、凝血因子VIII替代物

+ [1]

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

获得性因子 VIII缺乏症血友病A冯·维勒布兰德病

非在研适应症-

原研机构

Grifols SA

在研机构

Grifols Biologicals, Inc.

Grifols SAAlpha Therapeutic Corp.

非在研机构

Grifols Therapeutics, Inc.

最高研发阶段批准上市

首次获批日期

美国 (1978-08-15),

获得性因子 VIII缺乏症血友病A冯·维勒布兰德病

最高研发阶段(中国)-

特殊审评孤儿药 (美国)

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关联

6

项与 Antihemophilic factor/von Willebrand factor complex(Grifols SA) 相关的临床试验

NCT03095287

/ Terminated临床2期

A Multicenter Phase 2 Open-Label, Single-Arm, Prospective, Interventional Study of Plasma-Derived Factor VIII/VWF (Alphanate®) in Immune Tolerance Induction Therapy in Subjects With Congenital Hemophilia A

This is a multicenter, multinational, prospective, single-arm, nonrandomized, open-label study, planned in of approximately 25 male participants with congenital hemophilia A who will receive their first (primary) immune tolerance induction (ITI) treatment with alphanate.
The study consists of 2 phases:
An ITI Treatment Phase in which all eligible participants will receive ITI treatment with alphanate for a period of up to 33 months. Upon confirmation of complete immune tolerization, participants will then enter a 12-month Prophylactic Phase. If, after 33 months of ITI, a participants has achieved partial immune tolerance, the participants will enter a 12-month Prophylactic Phase.
A 12-month Prophylactic Phase for all participants who meet the criteria for complete or partial success to continue on a prophylactic dosing regimen of alphanate. Due to limited enrollment, this study was early terminated.

开始日期2018-01-03

申办/合作机构

Grifols Therapeutics, Inc.

[+1]

CTRI/2017/04/008336

/ Active, not recruiting临床2期

A Multicenter, Phase 2 Open-Label, Single-Arm, Prospective, InterventionalStudy of Plasma-Derived Factor VIII/VWF (AlphanateÃ?Â®) in Immune Tolerance InductionTherapy in Subjects with Congenital Hemophilia A and Inhibitors - Nil

开始日期2017-05-02

申办/合作机构-

EUCTR2012-003450-92-ES

/ Unknown statusN/A

EVALUATION OF THE PHARMACOKINETIC PROFILE, CLINICAL EFFICACY AND SAFETY OF THE VON WILLEBRAND FACTOR CONTAINED IN FANHDI® (DOUBLE-INACTIVATED HUMAN ANTI-HEMOPHILIC FACTOR) IN PEDIATRIC PATIENTS WITH VON WILLEBRAND DISEASE - Study of Fanhdi® in pediatric patients with VWD

开始日期2013-11-23

申办/合作机构

Instituto Grifols SA

100 项与 Antihemophilic factor/von Willebrand factor complex(Grifols SA) 相关的临床结果

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100 项与 Antihemophilic factor/von Willebrand factor complex(Grifols SA) 相关的转化医学

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100 项与 Antihemophilic factor/von Willebrand factor complex(Grifols SA) 相关的专利（医药）

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55

项与 Antihemophilic factor/von Willebrand factor complex(Grifols SA) 相关的文献（医药）

2024-01-01·Clinical and Applied Thrombosis/Hemostasis

Real-World Efficacy and Safety of Plasma-Derived Von Willebrand Factor-Containing Factor VIII Concentrates in Patients With Von Willebrand Disease in Italy

Article

作者: Pieri, Lisa ; Santoro, Cristina ; Borchiellini, Alessandra ; Barillari, Giovanni ; Santoro, Rita Carlotta ; Mir, Roser ; Federici, Augusto B. ; Tosetto, Alberto ; Zanon, Ezio ; Santi, Roberto Mario ; De Cristofaro, Raimondo ; Mairal, Esther

Plasma-derived von Willebrand factor-containing factor VIII concentrates (pd-VWF/FVIII-C) are the mainstay of treatment in von Willebrand disease (VWD). Real-world data on efficacy and safety of these pd-VWF/FVIII-C are required. To retrospectively evaluate the efficacy and safety of pd-VWF/FVIII-C (Fanhdi® and Alphanate®, Grifols) in clinical practice in Italy. A multicentric, observational, retrospective study at 10 Italian centers was conducted. Eligible patients diagnosed with inherited VWD (ISTH criteria) were treated with either Fanhdi® or Alphanate® for bleeding episodes, prevention of surgical bleeding and secondary long-term prophylaxis (SLTP) according to clinical practice with medical records collected from January 2007 to December 2019. Efficacy/safety of pd-VWF/FVIII-C was assessed according to FDA-agreed objective criteria following regulatory procedures. Fifty-seven patients (M/F: 21/36) were enrolled in the study with the following VWD types: VWD1 (n = 29, 52%), VWD2A (n = 10, 18%), VWD2B (n = 7, 12%), VWD2M (n = 2, 4%), VWD2N (n = 1, 2%), VWD2 unclassified (n = 1, 2%), and VWD3 (n = 7, 12%). These pd-VWF/FVIII-C were used to manage 58 bleeding episodes (n = 24 patients), 100 surgeries (n = 47 patients), and 7 SLTP (n = 6 patients). Global clinical efficacy with these pd-VWF/FVIII-C was reported to be excellent/good in 85% of bleeding episodes, 98% of surgeries, and 100% of SLTP. As far as safety, no adverse-drug-related episodes, immunogenic or thrombotic events were reported. This study confirmed that Fanhdi® and Alphanate® were effective and safe in the management of bleeding episodes, the prevention of bleeding during surgeries and for SLTP in Italian patients with inherited VWD.

2023-06-13·Cureus

Anesthetic Management Including Postoperative Regional Anesthesia in a Young Adult Patient With Von Willebrand Disease and Osteogenesis Imperfecta

作者: Crespo, Maria J ; Torres-Pérez, Hector ; Rosa-Carrasquillo, Cristian ; Rivera-Pérez, Dennys ; Reyes-Sullivan, Allan J

There are no established guidelines regarding anesthesia with a peripheral nerve block (PNB) in the young adult population with von Willebrand disease (vWD) type I. We present a case of a successful PNB outcome in a 20-year-old male patient with vWD type I, osteogenesis imperfecta (OI), and rheumatoid arthritis (RA) who underwent an intramedullary nailing surgery after suffering a left distal femur fracture secondary to a sports injury. Before the procedure, the patient was treated with HUMATE-P® [antihemophilic factor and von Willebrand factor (human)], ALPHANATE^® (antihemophilic factor/von Willebrand factor complex), and aminocaproic acid for hematologic control. Left femoral and popliteal nerve blocks were performed for postoperative pain control. The patient was discharged home uneventfully three days after the surgery. In this case, PNB proved to be a safe and effective alternative in the management of a vWD type I young adult patient with comorbidities. Given the lack of established guidelines, a multidisciplinary team should be involved in the pre and perioperative management of these patients due to the risk of delayed bleeding.

2022-01-01·Clinical and Applied Thrombosis/Hemostasis4区 · 医学

Clinical Efficacy and Safety of Fanhdi^®, a Plasma-Derived VWF/Factor VIII Concentrate, in von Willebrand Disease in Spain: A Retrospective Study

4区 · 医学

Article

作者: Jiménez-Yuste, Víctor ; Núñez, Ramiro ; Nieto Hernández, Maria del Mar ; Marzo Alonso, Cristina ; Varó Castro, Maria José ; Palomo Bravo, Ángeles ; Benítez Hidalgo, Olga ; Coll, Julia ; Galmes, Bernardo J. ; Alvarez-Román, María Teresa ; Mir, Roser ; Hernández García, Carmen ; Carrasco, Marina ; Pérez González, Noelia Florencia ; García Candel, Faustino ; Gonzalez-Porras, Jose Ramon

Objective To evaluate the efficacy and safety of a plasma-derived factor VIII concentrate containing von Willebrand Factor (pdVWF/FVIII) in standard clinical practice in von Willebrand Disease (VWD) patients. Methods A retrospective, multicentric, observational study of VWD patients treated with Fanhdi®, a pdVWF/FVIII concentrate, from January 2011 to December 2017 was conducted at 14 centers in Spain. Efficacy and safety were evaluated for acute bleeding episodes, for prevention of bleeding in surgeries, and for secondary long-term prophylaxis. Results Seventy-two eligible patients, type 1, 2, 3 VWD (25%/38.9%/36.1%) were treated for spontaneous and traumatic bleeding (140 episodes, n = 41 patients), to prevent surgical bleeding (69 episodes, n = 43 patients); and for secondary long-term prophylaxis (18 programs, n = 13 patients). Replacement therapy with pdVWF/FVIII showed an excellent to good clinical efficacy in 96.7% of the bleeding episodes, 100% during surgical procedures and 100% during prophylaxis. No adverse events (AEs), nor serious AEs related to the product were observed. Conclusions Fanhdi® was effective, safe and well tolerated in the management of bleeding episodes, the prevention of bleeding during surgeries, and for secondary long-term prophylaxis in VWD patients.

100 项与 Antihemophilic factor/von Willebrand factor complex(Grifols SA) 相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
-	Antihemophilic factor/von Willebrand factor complex(Grifols SA)	B02BD06	-

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
获得性因子 VIII缺乏症	美国	Grifols Biologicals, Inc.	1978-08-15
血友病A	美国	Grifols Biologicals, Inc.	1978-08-15
冯·维勒布兰德病	美国	-	1978-08-15

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
血友病A	临床2期	加拿大	Grifols Biologicals, Inc.	2018-01-03
血友病A	临床2期	西班牙	Grifols Therapeutics, Inc.	2018-01-03
血友病A	临床2期	印度	Grifols Biologicals, Inc.	2018-01-03
血友病A	临床2期	意大利	Grifols Biologicals, Inc.	2018-01-03
血友病A	临床2期	加拿大	Grifols Therapeutics, Inc.	2018-01-03
血友病A	临床2期	印度	Grifols Therapeutics, Inc.	2018-01-03
血友病A	临床2期	意大利	Grifols Therapeutics, Inc.	2018-01-03
血友病A	临床2期	俄罗斯	Grifols Biologicals, Inc.	2018-01-03
血友病A	临床2期	俄罗斯	Grifols Therapeutics, Inc.	2018-01-03
血友病A	临床2期	西班牙	Grifols Biologicals, Inc.	2018-01-03

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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


NCT03095287 (CTgov)	临床2期	血友病A	2	Alphanate	繭遞鹹選選壓餘衊願觸(鬱顧鬱鹹繭鏇窪網夢積) = 遞夢網遞簾夢憲廠願蓋顧顧簾願窪選齋範獵廠 (遞遞蓋膚膚鏇鬱鬱鑰壓, 鏇夢獵襯鏇壓蓋遞遞廠 ~ 顧顧顧鹽衊範壓顧積簾) 更多	-	2021-11-23
ISTH2020	N/A	冯·维勒布兰德病	-	Fanhdi®	餘鬱範簾襯窪窪憲餘製(壓選觸鹽鬱蓋夢簾壓觸) = 夢壓廠顧構積築願糧餘餘淵簾顧蓋醖積範繭遞 (製鑰鑰築鑰築積憲壓繭 ) 更多	积极	2020-07-12