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Recombinant Factor VIIa BI

Phase 3

Hemophilia A | Monoclonal antibody | Hematology |Takeda Pharmaceutical Company Limited|Last Updated: May 11, 2021

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
RandomizedCONTROLLEDDMC
Total Trials1
Total Enrollment40
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT01757405Recombinant Factor VIIa BI (rFVIIa BI) Treatment of Acute Bleeding Episodes Per an On-demand RegimenPHASE3 COMPLETED 40Feb 20, 2013Nov 11, 2014May 11, 202116 United States, Japan +7
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Study Endpoints
Primary Endpoints
Percentage of Bleeding Episode With "Treatment Success"
within 12 hours of first dose

No additional hemostatic product required within 12 hours of first dose other than the prescribed dosing regimen.

Secondary Endpoints
Treatment Response for Each Bleeding Episode
within 24 hours of infusion
Percentage of Clinical Responders (Sustained Bleeding Control) for All Acute Bleeding Episodes
24 hours post infusion
Safety and Tolerability of Treatment Regimens by Clinical Assessment of Percentage of Participants With Adverse Events (AEs)
6 months (throughout study period)
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Study Design & Arms
AllocationRANDOMIZED
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
≤ 3 doses of 90 µg/kg rFVIIa BIEXPERIMENTAL -
One dose of 270 µg/kg rFVIIa BIEXPERIMENTAL -
Interventions
NameTypeDescription
Recombinant Factor VIIa BI (rFVIIa BI)BIOLOGICALAdministered approximately every 3 hours as an intravenous bolus injection on-demand
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Eligibility Criteria
Age Range12 Years — 65 Years
SexMALE
Healthy VolunteersNo
Study Sites16

Main Inclusion Criteria: * Participant is male with hemophilia A or B with inhibitors, with a high titer (≥5 Bethesda unit (BU)) or a historical high anamnestic response. * Participant is 12 to 65 years old at the time of screening. * Participant is currently using or has used bypassing agents for ...

Countries:United StatesJapanPolandRomaniaRussiaSerbiaSpainTaiwanUkraine
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