Approval Probability
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Adjusted LOA
ML Risk
Recombinant von Willebrand Factor · 2 trials · 1 indication
Hemostatic efficacy will be rated on a scale of excellent - good - moderate - none. Excellent: Intra-, and postoperative hemostasis achieved with rVWF with our without ADVATE was as good or better than that expected for the type of surgical procedure performed in a hemostatically normal subject. Good: Intra-, and postoperative hemostasis achieved with rVWF with or without ADVATE was probably as good as that expected for the type of surgical procedure performed in a hemostatically normal subject. Moderate: Intra-, and postoperative hemostasis with rVWF with or without ADVATE was clearly less than optimal for the type of procedure performed but was maintained without the need to change the rVWF concentrate. None: Participant experienced uncontrolled bleeding that was the result of inadequate therapeutic response despite proper dosing, necessitating a change of rVWF concentrate.
Treatment success was defined as the extent of control of bleeding episodes (BEs) using a mean efficacy rating score of \<2.5 for a participant's BEs treated with study product (recombinant von Willebrand Factor \[rVWF\] with or without recombinant factor VIII \[rFVIII\]) during the study period. Scores used: Excellent = 1 - actual infusions ≤ estimated number of infusions required to treat BE; no additional VWF required (all BEs); Good = 2 - \>1-2 infusions (minor/moderate BEs) or \<1.5 infusions (major BEs) greater than estimated required to control BE; no additional VWF required (all BEs); Moderate = 3 ≥ 3 infusions (minor/moderate BEs) or ≥ 1.5 infusions (major BEs) greater than estimated required to control BE; no additional VWF required (all BEs); None = 4 - severe uncontrolled bleeding or intensity of bleeding not changed; additional VWF required. Included participants with available primary efficacy rating (prospective-excluding gastrointestinal bleeds) in the Full Analysis Set.
| Arm | Type | Description |
|---|---|---|
| Recombinant von Willebrand Factor (rVWF) | EXPERIMENTAL | Surgery participants treated with Recombinant von Willebrand Factor (rVWF) |
| PK 80 Arm (minimum of 22 subjects with severe VWD) | EXPERIMENTAL | PK assessment (80 IU/kg rVWF) + 12-month treatment period |
| PK 50 Arm (14 subjects with type 3 VWD) | EXPERIMENTAL | Two single-blinded PK assessments (50 IU/kg rVWF + rFVIII/placebo) + 12-month treatment period |
| PK 50 Only Arm (minimum of 7 subjects with type 3 VWD) | EXPERIMENTAL | PK assessment (50 IU/kg rVWF) only, no treatment of bleeding episodes |
| Treatment Only (up to 7 subjects independent of VWD subtype) | EXPERIMENTAL | Treatment of bleeding episodes for a total of 12 months |
| Name | Type | Description |
|---|---|---|
| Recombinant von Willebrand Factor (rVWF) | BIOLOGICAL | rVWF will be administered by intravenous bolus infusion. Participants planned for major surgery will undergo a baseline pharmacokinetic assessment prior to surgery. The peri- and postoperative substitution regimen will be individualized according to the PK results, intensity and duration of the hemostatic challenge, and the institution´s standard of care. |
| Placebo | DRUG | Syringe supplied with physiologic saline solution for infusion |
| Recombinant factor VIIII (rFVIII) | BIOLOGICAL | Intravenous administration |
Inclusion Criteria: * Diagnosis of severe von Willebrand disease (VWD) as listed below and elective surgical procedure planned 1. Type 1 (Von Willebrand factor : Ristocetin cofactor activity (VWF:RCo) \<20 IU/dL), or 2. Type 2A (as verified by multimer pattern), Type 2B (as diagnosed by genoty...
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Takeda Pharmaceutical Co. Ltd. Sponsored ADR | TAK | 3 | PHASE3 | Vonicog Alfa, ADVATE |
| Alnylam Pharmaceuticals, Inc | ALNY | 1 | PHASE2 | ALN-6400 |
| Hemab Therapeutics Holdings, Inc. | COAG | 2 | PHASE1 | HMB-002 |
| Novo Nordisk A/S Sponsored ADR Class B | NVO | 1 | - | Undisclosed |
Recombinant von Willebrand Factor is used for the treatment of bleeding episodes in patients with Von Willebrand Disease (VWD), a bleeding disorder. It is also studied for use in subjects with severe Von Willebrand Disease undergoing surgery. The drug is developed by Takeda Pharmaceutical Company Limited.
Recombinant von Willebrand Factor is developed by Takeda Pharmaceutical Company Limited, which trades under the ticker TAK. The drug is being investigated for the treatment of Von Willebrand Disease, a hematologic condition.
Recombinant von Willebrand Factor is in Phase 3 clinical development. It is an investigational drug, not yet approved, and is being studied for the treatment of bleeding episodes in Von Willebrand Disease and for use in surgery for severe Von Willebrand Disease.
Recombinant von Willebrand Factor has completed two Phase 3 trials. NCT01410227 studied its pharmacokinetics, safety, and efficacy in treating bleeding episodes in Von Willebrand Disease with 49 participants. NCT02283268 studied its use in severe Von Willebrand Disease patients undergoing surgery with 24 participants.
Recombinant von Willebrand Factor is a monoclonal antibody modality. It is being developed for the treatment of Von Willebrand Disease, a bleeding disorder, and has completed Phase 3 clinical trials.