Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Also known as Vonvendi, Vonicog alfa, TAK-577, von Willebrand factor (Recombinant), BAX 111, SHP677, Recombinant von Willebrand factor (rVWF)
rVWF · 4 trials · 2 indications
A TEAE is defined as an adverse event with an onset that occurs after receiving study drug. An SAE is any untoward medical occurrence that at any dose: results in death, life-threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent, significant disability/incapacity, a congenital abnormality/birth defect, an important medical event in the opinion of the healthcare provider, may jeopardize the participant or may require intervention to prevent one of the other outcomes listed in the definition above. TEAEs consist of both serious and non-serious adverse events.
Number of participants with severity of TEAE will be reported.
Number of participants with causality related TEAEs and SAEs will be reported.
Number of participants with thromboembolic events and severe hypersensitivity reactions will be reported.
Number of participants who develop neutralizing antibodies to VWF and FVIII will be reported.
Number of participants who develop total binding antibodies to VWF and FVIII will be reported.
Number of participants with clinically significant abnormalities from baseline values in laboratory parameters per investigator assessment will be reported.
Number of participants with clinically significant abnormalities from baseline values in ECG per investigator assessment will be reported.
Number of participants with clinically significant abnormalities from baseline values in vital sign parameters per investigator assessment will be reported.
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.
sABR was derived as \[number of treated bleeds\] / \[duration in years\]. Bleeds with unknown causality were considered as spontaneous. Bleeds were categorized based on the investigator assessment of cause. sABR during the first 12 months of prophylactic treatment with rVWF (vonicog alfa) was reported.
Treatment success for vonicog alfa-treated nonsurgical bleeding episodes (using a 4-point scale: Excellent, Good, Moderate, None).
| Arm | Type | Description |
|---|---|---|
| All Participants With VWD | EXPERIMENTAL | Participants will receive a single intravenous (IV) dose of VONVENDI at baseline PK assessment. During the 12-month on-demand (OD) treatment period, any bleeding episodes requiring replacement therapy with VWF will be treated with VONVENDI with or without ADVATE. Participants may also receive VONVENDI with or without ADVATE intravenous infusions, when indicated deemed necessary for perioperative bleeding management \[major, minor and oral surgery\]. Participants will receive initial dose of VONVENDI of 40 to 80 IU/kg of body weight. |
| 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 | EXPERIMENTAL | Participants will receive recombinant von Willebrand factor (rVWF) (with or without ADVATE). |
| Prophylaxis | EXPERIMENTAL | Participants will receive recombinant von Willebrand factor (rVWF). |
| 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 |
|---|---|---|
| VONVENDI | BIOLOGICAL | VONVENDI is administered by intravenous injection. |
| ADVATE | BIOLOGICAL | ADVATE is administered by intravenous injection. |
| Vonicog Alfa | BIOLOGICAL | Vonicog Alfa administered by intravenous injection. |
| rVWF | BIOLOGICAL | Recombinant von Willebrand factor |
| rFVIII | BIOLOGICAL | Recombinant Factor VIII |
| 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. |
Inclusion Criteria 1. Participant must voluntarily sign an institutional review board (IRB)/independent ethics committee-approved written informed consent form after all relevant aspects of the study have been explained and discussed with the participant. 2. Participant has a documented diagnosis o...
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 |
rVWF is a recombinant von Willebrand factor being studied for the treatment of Von Willebrand Disease (VWD), a bleeding disorder. It is intended for use in pediatric and adult patients with severe VWD. The drug is currently in Phase 3 clinical development and is not yet approved.
rVWF is being developed by Takeda Pharmaceutical Company Limited, which trades under the ticker TAK. The company is conducting clinical trials to evaluate the safety and efficacy of rVWF in patients with severe Von Willebrand Disease.
rVWF is in Phase 3 clinical development. A Phase 3 study has been completed, but the drug remains investigational and has not received regulatory approval. It is being studied for the treatment of severe Von Willebrand Disease in pediatric and adult patients.
rVWF has one completed Phase 3 clinical trial, identified as NCT03879135. This study evaluated rVWF in pediatric and adult participants with severe Von Willebrand Disease. The trial enrolled 38 participants and was conducted in multiple countries, including the United States, Austria, France, Germany, Italy, Netherlands, Russia, Spain, and Turkey.
Yes, rVWF is the abbreviation for recombinant von Willebrand factor. It is a laboratory-made version of the von Willebrand factor protein, which is deficient or defective in people with Von Willebrand Disease. The drug is being developed to replace this missing protein and help control bleeding.