Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Also known as von Willebrand factor (Recombinant)
von Willebrand factor · 1 trial · 1 indication
ABR for treated spontaneous BEs while on prophylactic treatment with rVWF through month 12 of treatment period/observation period (in years), where an observation period = (date of completion/termination - date of first dose + 1)/365.2425. Bleeds occurred at the same anatomical location with the same etiology within 24 hours after onset of the first bleed were considered a single bleed. Bleeding occurred at multiple locations related to the same injury were considered as a single bleeding episode. Historical observation period for historical BEs was 365 days prior to first dose of study drug. On-study observation period started on the day of first administration of study drug and continuing through the date of completion/discontinuation from study. The comparison of the two ABRs (on-study and historical) for spontaneous bleeding episodes (not related to trauma) during prophylactic treatment with rVWF was reported as a ratio of mean ABRs (on-study ABR:historical ABR).
| Arm | Type | Description |
|---|---|---|
| All Study Participants | EXPERIMENTAL | Participants will receive prophylaxis with rVWF in two cohorts: on-demand (OD) cohort (previously treated with OD) and pdVWF switch cohort (participants switching from prophylactic treatment with pdVWF). |
| Name | Type | Description |
|---|---|---|
| von Willebrand factor (Recombinant) | BIOLOGICAL | OD participants will receive intravenous (IV) rVWF:RCo at an initial prophylactic dose of 50 +/- 10 International Unit per Kilogram (IU/kg) twice (two infusions) a week for at least 12 months up to 15 months and may be increased up to 80 IU/kg. pdVWF switch cohort participants will receive rVWF:RCo equivalent (± 10%) to the weekly VWF dose received during prophylactic treatment with pdVWF. |
| Antihemophilic Factor (Recombinant) | BIOLOGICAL | During prophylaxis period any bleeding episodes requiring substitution therapy with VWF concentrate to control bleeding will be treated with rVWF with or without ADVATE. Participants will receive rFVIII IV if necessary for OD treatment of breakthrough bleeds or for peri-operative. The dose will be according to the bleeding type and severity and it will be adjusted to the clinical response. |
Inclusion Criteria: 1. Participant has a documented diagnosis of severe von Willebrand disease (VWD) (baseline Von Willebrand factor: Ristocetin cofactor activity (VWF:RCo) less than (\<) 20 International Units/Deciliter \[IU/dL\]) with a history of requiring substitution therapy with von Willebran...
Top 4 of 5 competitors
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Takeda Pharmaceutical Co. Ltd. Sponsored ADR | TAK | 4 | PHASE3 | Vonvendi, ADVATE |
| Incyte Corporation | INCY | 2 | PHASE3 | VGA039 |
| Alnylam Pharmaceuticals, Inc | ALNY | 1 | PHASE2 | ALN-6400 |
| Hemab Therapeutics Holdings, Inc. | COAG | 2 | PHASE1 | HMB-002 |
Von Willebrand factor is being developed for Von Willebrand Disease, a bleeding disorder caused by missing or defective von Willebrand factor. The recombinant form is under study as a prophylaxis treatment in adults with the condition. It is an investigational product and is not approved for commercial use.
Takeda Pharmaceutical Company Limited, which trades under the ticker TAK, is the developer of recombinant von Willebrand factor. The company is running the clinical program for the product in Von Willebrand Disease.
Von Willebrand factor is in Phase 3 development. One Phase 3 trial has been completed, and no trials are currently active. The product remains investigational and has not been approved by the FDA for Von Willebrand Disease.
Von Willebrand factor has been studied in one completed Phase 3 trial, NCT02973087, titled rVWF IN PROPHYLAXIS. That trial enrolled 29 adults with Von Willebrand Disease across the United States, Canada, France, Germany, Italy, Netherlands, Russia, Spain, and Turkey.
Yes. Von Willebrand factor is also known as von Willebrand factor (Recombinant), so the two names refer to the same Takeda product. The recombinant designation distinguishes the manufactured form from plasma-derived von Willebrand factor.
NCT02973087 was an uncontrolled Phase 3 study of von Willebrand factor in Von Willebrand Disease. It was not randomized and not double blinded, and it enrolled 29 participants aged 18 years and older. The trial has been completed.