Delving into the Latest Updates on Antihemophilic factor/von Willebrand factor complex(Grifols SA) with Synapse

Overview

Basic Info

Drug Type

Non-recombinant coagulation factor

Synonyms

Alphanate, Factor VIII/von Willebrand factor, Fanhdi

+ [1]

Target

F10

Action

stimulants

Mechanism

F10 stimulants(Coagulation factor X stimulants), Blood coagulation pathway activation, Coagulation factor VIII replacements

Therapeutic Areas

Congenital DisordersHemic and Lymphatic DiseasesOther Diseases

Active Indication

Factor 8 Deficiency, AcquiredHemophilia AVon Willebrand Diseases

Inactive Indication-

Originator Organization

Grifols SA

Active Organization

Grifols Biologicals, Inc.

Grifols SAAlpha Therapeutic Corp.

Inactive Organization

Grifols Therapeutics, Inc.

License Organization-

Drug Highest PhaseApproved

First Approval Date

United States (15 Aug 1978),

Factor 8 Deficiency, AcquiredHemophilia AVon Willebrand Diseases

RegulationOrphan Drug (United States)

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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.

Start Date03 Jan 2018

Sponsor / Collaborator

Grifols Therapeutics, Inc.

[+1]

EUCTR2012-003450-92-ES

/ Not yet recruitingPhase 4

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

Start Date23 Nov 2013

Sponsor / Collaborator

Instituto Grifols SA

NCT01051076

/ CompletedNot ApplicableIIT

Immune Tolerance Induction Study in Patients With Severe Type A Haemophilia With Inhibitor After Failure of a Previous Induction of Immune Tolerance With FVII Concentrates Without Von Willebrand Factor Rescue

The purpose of this study is to evaluate whether a concentrate containing both FVIII and von Willebrand Factor (VWF) given at a high dose will induce immune tolerance in subjects who have already experienced and failed ITI with VWF-free FVIII concentrates. The treatment on this study is expected to last up to 33 months.

Start Date03 Nov 2009

Sponsor / Collaborator

City of Hope National Medical Center

[+4]

100 Clinical Results associated with Antihemophilic factor/von Willebrand factor complex(Grifols SA)

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100 Translational Medicine associated with Antihemophilic factor/von Willebrand factor complex(Grifols SA)

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100 Patents (Medical) associated with Antihemophilic factor/von Willebrand factor complex(Grifols SA)

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57

Literatures (Medical) associated with Antihemophilic factor/von Willebrand factor complex(Grifols SA)

31 Dec 2025·JOURNAL OF MEDICAL ECONOMICS

Cost-effectiveness and cost-utility analysis of Haemate-P versus other von Willebrand disease treatments in Spain

Article

Author: Panebianco, Marco ; Escolar, Gines ; Yan, Songkai ; McDade, Cheryl L. ; Barrientos, Laura Vidal ; Linden, Stephan ; Wilson, Michele R. ; Mendez, Pablo ; Tomic, Radovan ; Megias-Vericat, Juan E.

OBJECTIVE:

von Willebrand Disease (vWD) is the most common congenital bleeding disorder, with an estimated prevalence in Spain of 0.01%. The aim was to assess the cost-utility of Haemate-P compared with present alternatives in the treatment of vWD in Spain.

METHODS:

A Markov model was developed in Microsoft Excel to estimate the cost-effectiveness of various treatments for vWD over a lifetime horizon. Transition probabilities among health states were based on age and number of bleeding events. Treatment strategies compared included Haemate-P, Fanhdi, and Wilate in long-term prophylaxis (LTP) or on-demand treatment (ODT). Costs and quality-of-life were measured based on patient age, treatment, and number of bleeding events incurred. Both costs and utilities were discounted at 3%. One-way and probabilistic sensitivity analyses were performed.

RESULTS:

When comparing LTP regimens, Haemate-P was less costly and numerically more effective than both Fanhdi (incremental costs = -€1,313,845; incremental quality-adjusted life-years [QALY] = 0.13) and Wilate (incremental costs = -€2,233,940; incremental QALY = 0.29). For ODT, Haemate-P was assumed to have equal effectiveness as Fanhdi and Wilate but reduced the costs by €696,857 and €1,145,780, respectively. Haemate-P prophylaxis was more effective and less costly compared with Haemate-P on-demand in the base case (incremental costs = -€633,317; incremental QALY = 0.90). Results were generally robust to sensitivity analyses.

CONCLUSIONS:

In patients with severe vWD experiencing a high bleed rate, Haemate-P prophylaxis is a less costly and potentially more effective treatment strategy and Haemate-P is cost-saving among on-demand strategies.

31 Dec 2025·JOURNAL OF MEDICAL ECONOMICS

Budget impact analysis of Haemate- P as long-term prophylaxis and on-demand therapy for von Willebrand disease in Spain

Article

Author: Megías-Vericat, Juan E. ; Panebianco, Marco ; Yan, Songkai ; Wilson, Michele R. ; Vidal Barrientos, Laura ; Mendez, Pablo ; McDade, Cheryl L. ; Karampampa, Korinna ; Escolar, Gines

OBJECTIVES:

A decision-analytic model was developed to assess the Spanish healthcare system budget impact associated with increased use of Haemate-P in individuals with von Willebrand disease.

METHODS:

The model considered long-term prophylaxis and on-demand treatment with each of three von Willebrand factor (vWF) replacement products (Haemate-P, Fanhdi, and Wilate). Two budget scenarios were considered: a status quo scenario (no changes in uptake of products/strategy) and an alternative scenario (increased Haemate-P uptake). The model estimated total product costs and other medical costs for each budget scenario annually over 3 years. Total budget impact was estimated by subtracting the costs for the status quo scenario from the costs for the alternative scenario.

RESULTS:

The alternative Haemate-P uptake scenario was estimated to save the Spanish healthcare system €5.64 million over 3 years. Cost savings were driven primarily by a reduction in vWF product costs (-€5.62 million) resulting from a reduction in vWF product use (-€4.80 million) and bleed reductions (-€0.82 million). In univariate and multiway deterministic sensitivity analyses, the alternative scenario remained cost-saving.

CONCLUSION:

Haemate-P was found to be a cost-saving strategy, and increased use of Haemate-P over Fanhdi, and Wilate is expected to reduce overall costs to the healthcare system in Spain.

01 Jan 2025·CLINICAL AND APPLIED THROMBOSIS-HEMOSTASIS

Safety and Efficacy of the Use of pdVWF/FVIII-C in Patients with von Willebrand Disease: A Prospective, Observational, Post-Authorization Study

Article

Author: Lei, Alejhandra ; Hanna, Kim ; Afonso, Natalia ; Marzo Alonso, Cristina ; Carrasco, Marina ; Aragonés, Maria Esperança ; Oliveras, Juan ; Whyms, Dermot ; Torres, Mireia ; Nieto Hernández, Maria del Mar ; López Jaime, Francisco José ; Benítez Hidalgo, Olga

Introduction:

Plasma-derived von Willebrand factor containing FVIII concentrates (pdVWF/FVIII-C) are indicated as replacement therapy for patients with von Willebrand disease (VWD). This study assessed safety and efficacy associated with long-term real-world experience of the pdVWF/FVIII-C, Fanhdi ® , in patients with VWD.

Methods:

This observational, prospective, post-authorization cohort study was conducted at five centers in Spain. Patients with VWD were treated with the pdVWF/FVIII-C to achieve satisfactory hemostasis for on-demand (bleeding episodes and surgical/invasive procedures) and prophylaxis treatment. Clinical efficacy was evaluated as the response to treatment in both settings. Safety parameters were assessed.

Results:

Fifteen VWD patients received at least one dose of the pdVWF/FVIII-C and were followed for 12 months. Forty-six bleeding episodes were reported for 9 (60.0%) patients, and 6 surgical/invasive procedures for 5 (33.3%) patients. Most frequently reported bleedings were gastrointestinal (3 [33.0%] patients) and gynecological (3 [33.0%] patients). No complications nor bleeding episodes related to surgical/invasive procedures were reported. Overall clinical efficacy of treatment (including on-demand and prophylaxis) achieved 100% excellent and/or good (n = 15 patients), being excellent for 7 (46.7%) patients. There were 27 treatment-emergent adverse events in 8 (53.3%) patients, 11 serious adverse events in 3 (20.0%) patients, but none of them were drug-related. No clinical signs and symptoms of immunogenicity or thromboembolic events were reported.

Conclusions:

This real-world evidence study confirmed the efficacy of the pdVWF/FVIII-C as on-demand and/or prophylaxis treatment in patients with bleeding episodes or surgical procedures in VWD. Fanhdi ® was well tolerated without any safety concerns.

100 Deals associated with Antihemophilic factor/von Willebrand factor complex(Grifols SA)

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External Link

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

R&D Status

10 top approved records.

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Indication	Country/Location	Organization	Date
Factor 8 Deficiency, Acquired	United States	Grifols Biologicals, Inc.	15 Aug 1978
Hemophilia A	United States	Grifols Biologicals, Inc.	15 Aug 1978
Von Willebrand Diseases	United States	-	15 Aug 1978

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Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT00323856 (CTgov)	Phase 4	Hemophilia A	51	Factor VIII (FVIII)+Alphanate	pcikkhxpli = hcmzioiayt uraihdibsq (kappsdzxfd, qeixshsjwe - sklyfwagjj) View more	-	09 Jun 2022
NCT03095287 (CTgov)	Phase 2	Hemophilia A	2	Alphanate	igcbwxozrm(cdbcqleify) = feniarwaxm vjxttgphmv (qrwfwasqaz, hopuexbqbx - nqbedhthxv) View more	-	23 Nov 2021

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