Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Vonicog Alfa · 2 trials · 2 indications
ABR during the study compared to historical ABR for each participant for both spontaneous and traumatic bleeding episodes as classified by the investigator during prophylactic treatment with vonicog alfa (rVWF) will be reported.
Treatment success for vonicog alfa-treated nonsurgical bleeding episodes (using a 4-point scale: Excellent, Good, Moderate, None).
| Arm | Type | Description |
|---|---|---|
| Cohort 1: Participants With Age >=12 to <18 years | EXPERIMENTAL | Participants with age greater than or equal to (\>=) 12 to less than (\<) 18 years who have received on-demand (OD) therapy or prophylactic treatment with a pdVWF product will receive vonicog alfa (rVWF) with an initial dose selected within the range of 40 to 60 international units per kilogram (IU/kg) vonicog alfa (rVWF), intravenous infusions, twice-weekly for 12 months. Participants may receive vonicog alfa (rVWF) with or without ADVATE intravenous infusions, when indicated (as deemed necessary for breakthrough bleeding episode treatment and perioperative bleeding management). |
| Cohort 2: Participants With Age >=6 to <12 years | EXPERIMENTAL | Participants with age \>=6 to \<12 years who have received OD therapy of VWF product or prophylactic treatment with a pdVWF product will receive vonicog alfa (rVWF) with an initial dose selected within the range of 40 to 60 IU/kg vonicog alfa (rVWF), intravenous infusions, twice-weekly for 12 months. Participants may receive vonicog alfa (rVWF) with or without ADVATE intravenous infusions, when indicated (as deemed necessary for breakthrough bleeding episode treatment and perioperative bleeding management). |
| Cohort 3: Participants With Age <6 years | EXPERIMENTAL | Participants with age \<6 years who have received OD therapy of VWF product or prophylactic treatment with a pdVWF product will receive vonicog alfa (rVWF) with an initial dose selected within the range of 40 to 60 IU/kg vonicog alfa (rVWF), intravenous infusions, twice-weekly for 12 months. Participants may receive vonicog alfa (rVWF) with or without ADVATE intravenous infusions, when indicated (as deemed necessary for breakthrough bleeding episode treatment and perioperative bleeding management). |
| On-demand Treatment | EXPERIMENTAL | Participants will receive vonicog alfa (recombinant von Willebrand factor \[rVWF\]) treatment for non-surgical bleeding episodes over a 12 to 18-month period. |
| Elective Surgery | EXPERIMENTAL | 12-24 hours prior to surgery and within 3 hours of surgery. Minor surgery: infuse every 12-24 hours for at least 48 hours based on post-operative dosing. Oral Surgery: infuse at least once within first 8-12 hours post-surgery based on post-operative dosing. Major Surgery: infuse every 12-24 hours for at least first 96 hours post-surgery based on post-operative dosing. |
| Emergency Surgery | EXPERIMENTAL | Within 3 hours prior to surgery. Minor surgery: infuse every 12-24 hours for at least 48 hours based on post-operative dosing. Oral Surgery: infuse at least once within first 8-12 hours post-surgery based on post-operative dosing. Major Surgery: infuse every 12-24 hours for at least first 96 hours post-surgery based on post-operative dosing. |
| Name | Type | Description |
|---|---|---|
| Vonicog Alfa | BIOLOGICAL | Vonicog Alfa administered by intravenous injection. |
| ADVATE | BIOLOGICAL | ADVATE administered by intravenous injection. |
| Antihemophilic Factor (Recombinant) | BIOLOGICAL | Packaged in single boxes with 2 glass vials, with one vial containing the lyophilized ADVATE and the second vial containing the diluent. |
1. The participant has a documented diagnosis of severe VWD (baseline von Willebrand factor ristocetin cofactor activity \[VWF:RCo\] \<20 international units per deciliter \[IU/dL\]) with a history of replacement therapy with VWF concentrate required to control bleeding and a diagnosis of VWD type 1...
| 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 |
Vonicog Alfa is used for the treatment of Von Willebrand Disease (VWD), a bleeding disorder. It is being studied in children with severe VWD, both in completed and ongoing Phase 3 clinical trials. The drug is administered to help manage bleeding episodes in this patient population.
Vonicog Alfa targets von Willebrand factor, a protein essential for normal blood clotting. It is a recombinant form of this factor, designed to replace the deficient or defective protein in patients with Von Willebrand Disease. This helps restore proper hemostasis and reduce bleeding risk.
Vonicog Alfa is developed by Takeda Pharmaceutical Company Limited, a global biopharmaceutical company. Takeda is conducting clinical trials to evaluate the safety and efficacy of Vonicog Alfa in pediatric patients with severe Von Willebrand Disease.
Vonicog Alfa is in Phase 3 clinical development. It is an investigational drug, meaning it has not yet been approved by regulatory authorities. Two Phase 3 trials have been conducted or are ongoing, including one completed study and one currently recruiting participants.
Vonicog Alfa is being studied in two Phase 3 clinical trials. The first, NCT02932618, is a completed study of recombinant von Willebrand factor with or without ADVATE in children with severe VWD. The second, NCT05582993, is an ongoing study of Vonicog Alfa in children with severe VWD.
Vonicog Alfa is a recombinant von Willebrand factor (rVWF). It is the same drug referred to as rVWF in clinical trial titles. The completed trial NCT02932618 studied rVWF with or without ADVATE, and the ongoing trial NCT05582993 specifically evaluates Vonicog Alfa.