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rVWF

Phase 3

Von Willebrand Disease (VWD) | Monoclonal antibody | Hematology |Takeda Pharmaceutical Company Limited|Last Updated: Sep 3, 2025

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
CONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment38
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT03879135A Study of Recombinant Von Willebrand Factor (rVWF) in Pediatric and Adult Participants With Severe Von Willebrand Disease (VWD)PHASE3 COMPLETED 38Apr 1, 2019Jan 30, 2025Sep 3, 202533 United States, Austria +7
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Study Endpoints
Primary Endpoints
Spontaneous Annualized Bleeding Rate (sABR)
Up to 12 months

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.

Secondary Endpoints
Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious TEAEs
Up to 5.8 years
Number of Participants Based on Severity of TEAEs
Up to 5.8 years
Number of Participants Based on Causality of TEAEs
Up to 5.8 years
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Study Design & Arms
AllocationNON_RANDOMIZED
MaskingNONE
ModelPARALLEL
PurposePREVENTION
Treatment Arms
ArmTypeDescription
On-DemandEXPERIMENTALParticipants will receive recombinant von Willebrand factor (rVWF) (with or without ADVATE).
ProphylaxisEXPERIMENTALParticipants will receive recombinant von Willebrand factor (rVWF).
Interventions
NameTypeDescription
rVWFBIOLOGICALRecombinant von Willebrand factor
rFVIIIBIOLOGICALRecombinant Factor VIII
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Eligibility Criteria
Age Range0 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites33

Inclusion Criteria: The participant will not be considered eligible for the study without meeting all of the criteria below. Participants who have completed Study 071301 or Study 071102 (or participants who have completed the surgery arm treatment in Study 071102 and want to continue to receive on...

Countries:United StatesAustriaFranceGermanyItalyNetherlandsRussiaSpainTurkey (Türkiye)
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