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

HemorrhageVon Willebrand Disease, Type 3Factor 8 Deficiency, Acquired+ [2]

Inactive Indication-

Originator Organization

Grifols SA

Active Organization

Grifols SAGrifols Biologicals, Inc.Alpha 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]

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]

NCT01051544

/ WithdrawnNot ApplicableIIT

Randomised Study of First TIME Immunotolerance Induction in Patients With Severe Type A Haemophilia With Inhibitor at High Risk of Failure: Comparison of Induction of Immune Tolerance With FVIII Concentrates With or Without Von Willebrand Factor Acronym: RES.I.S.T.- Naive

This is a prospective, controlled, randomized, open label study, aimed at comparing FVIII/VWF concentrates with FVIII concentrates at 200 IU/kg daily in their ability to induce immune tolerance in Haemophilia A patients with high responding inhibitors and poor prognosis for success.

Start Date25 Sep 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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50

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

01 Aug 2026·JOURNAL OF THROMBOSIS AND HAEMOSTASIS

Inhibitor development according to FVIII concentrates in previously untreated patients with severe hemophilia A: update from the PedNet registry

Article

Author: Nolan, Beatrice ; Fischer, K. ; Holzhauer, S. ; Chambost, H. ; Oudot, C. ; Pinto, Fernando ; Rosa Cid, A. ; Blatný, J. ; Mathews, N. ; Klamroth, R. ; Fischer, Kathelijn ; Långström, S. ; Motwani, Jayashree ; Carvalho, M. ; Pinto, F. ; Labarque, V. ; Male, C. ; Van Geet, Chris ; Bührlen, M. ; Zapotocka, Ester ; Nolan, B. ; Pergantou, H. ; Blatný, Jan ; Andersson, Nadine G. ; de Kovel, Marloes ; Labarque, Veerle ; Benitez Hidalgo, O. ; Königs, C. ; Castaman, G. ; Van Geet, C. ; Kenet, G. ; Kenet, Gili ; Andersson, N.G. ; Olivieri, Martin ; Kartal-Kaess, M. ; Zápotocká, E. ; Alvarèz Román, M.T. ; Ranta, Susanna ; Oldenburg, J. ; Königs, Christoph ; Carcao, M. ; Ranta, S. ; Levy-Mendelovich, S. ; d’Oiron, R. ; Carcao, Manuel ; Knudsen, H. ; Stamm Mikkelsen, T. ; Eckhardt, C. ; Ljung, R. ; Motwani, J. ; Molinari, A.C. ; Escuriola-Ettingshausen, C. ; Olivieri, M.

BACKGROUND:

Treatment of severe hemophilia A (SHA) with FVIII concentrates is complicated by the development of neutralizing inhibitors in about 30% during the first 50 exposure days (EDs). Despite extensive research, inhibitor risk of different FVIII concentrates remains to be elucidated.

OBJECTIVES:

This study aimed to analyze inhibitor development according to (classes of) individual FVIII concentrates in previously untreated patients (PUPs) with SHA.

METHODS:

PUPs with SHA born between 2000 and 2024 were followed until inhibitor development or 50 EDs. Inhibitor development according to classes of FVIII concentrates (ie, plasma-derived [pdFVIII] vs recombinant [rFVIII] and standard vs extended half-life [SHL-rFVIII vs EHL-rFVIII]) and individual concentrates used by ≥40 PUPs were compared using multivariable Cox regression (generating rate ratios [RR] with 95% CIs).

RESULTS:

One thousand five hundred three PUPs were included. Inhibitors developed in 444 PUPs after a median of 12 EDs (cumulative incidence, 31.0%; CI, 28.6%-33.4%). Compared to SHL-rFVIII, inhibitor risk was similar for pdFVIII (RR, 0.89; CI, 0.69-1.14) and EHL-rFVIII (RR, 1.10; CI, 0.75-1.61). Nine individual FVIII concentrates (5 SHL-rFVIII, 1 EHL-rFVIII, and 3 pdFVIII) were compare to Advate (n = 392): only KogenateFS/HelixateNexGen (n = 307; RR, 1.40; CI, 1.07-1.82, P, .013) and Fanhdi (n = 50; RR, 1.72; CI, 1.11-2.68; P, .024) showed increased inhibitor risk.

CONCLUSION:

Inhibitor development occurred in 31.0% of PUPs, with similar incidence across SHL-rFVIII, EHL-rFVIII, and pdFVIII. Analysis of individual concentrates showed increased inhibitor risk for Kogenate FS/Helixate NexGen (SHL-rFVIII) and for the first time for Fanhdi (pdFVIII). In the absence of formal PUP studies, PedNet will continue evaluating the inhibitor risk according to individual FVIII concentrates.

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. ; Wilson, Michele R. ; Linden, Stephan ; Barrientos, Laura Vidal ; 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 ; Vidal Barrientos, Laura ; Wilson, Michele R. ; 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.

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
Hemorrhage	United States	Grifols Biologicals, Inc.	31 Jul 2026
Von Willebrand Disease, Type 3	United States	Grifols Biologicals, Inc.	31 Jul 2026
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


NCT00555555 (CTgov)	Phase 4	Hemorrhage \| Von Willebrand Disease, Type 3	4	Alphanate	fqkhaimgep = ppkwwsbuor qrokfinumg (ynpkwyacmx, rdymphzghp - vrxalbhzfw) View more	-	30 Sep 2026
ISTH2024	Not Applicable	Von Willebrand Diseases	26	Alphanate	psxdnqlatb(oreygdnfqk) = mluvmsuhqr mhkfbomsic (jnmseumtbh ) View more	Positive	23 Jun 2024
NCT00323856 (CTgov)	Phase 4	Hemophilia A	51	Alphanate+Factor VIII (FVIII)	piucdcndpk = dvnjvfkwqz bgouguxolq (fgtflmfgyf, cuspjmlitg - wzjqmzklwq) View more	-	09 Jun 2022
NCT03095287 (CTgov)	Phase 2	Hemophilia A	2	Alphanate	ocveoqyeps(fvgeuxptam) = qgbkbyyjqf dxuzgihbba (xnwytitobx, ncpjqeutrj - aqwrhsfyit) View more	-	23 Nov 2021

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