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ReFacto AF

Phase 3

Hemophilia A | Small molecule | Hematology |Pfizer, Inc.|Last Updated: Jun 3, 2011

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
RandomizedDouble-BlindCONTROLLED
Total Trials4
Total Enrollment130
FDA Designations
No designations recorded
Clinical Trials (4)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT00243659Study Evaluating B-Domain Deleted Recombinant Factor VIII (BDDrFVIII, ReFacto AF) in Patients With Hemophilia A Undergoing Elective Major SurgeryPHASE3 COMPLETED 30Apr 1, 2006Jun 1, 2008Jun 3, 201117 United States, Australia +6
NCT00141843Study to Establish Bioequivalence of ReFacto AF (BDDrFVIII) With Advate (FLrFVIII) in Hemophilia APHASE3 COMPLETED 100Jul 1, 2005Nov 1, 2006Apr 22, 200841 United States, Australia +11
NCT00037544Study Evaluating ReFacto AF in Severe Hemophilia APHASE3 COMPLETED - -Aug 1, 2004Apr 22, 2008 -
NCT00038935Study Evaluating BDDRFVIII and ReFacto AF in Hemophilia APHASE3 COMPLETED - -Feb 1, 2003Apr 22, 2008 -
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Study Endpoints
Primary Endpoints
Number of Patients Who Achieved Hemostatic Efficacy After Surgery
6 weeks

Efficacy at the end of surgery as determined by the investigator and/or the surgeon using the 4 point ordinal scale (Excellent, Good, Moderate or None). Excellent: Achieved hemostatic comparable to non-hemophilic patients. Good: Prolonged time to hemostasis (with somewhat increased bleeding compared to non-hemophilic patients.

To determine the incidence rate of Factor VIII inhibitors in the study patient population.To establish bioequivalence of ReFacto AF as compared to Advate using the one stage Factor VIII activity assay.
Secondary Endpoints
Number of Patients Who Achieved Hemostatic Efficacy at Hospital Discharge
6 weeks
To characterize the efficacy of ReFActo AF in preventing and treating bleeding episodes during prophylaxis.To characterize PK of ReFacto AF as compared to Advate over time.
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Study Design & Arms
AllocationNON_RANDOMIZED
MaskingNONE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
AEXPERIMENTAL -
BEXPERIMENTAL -
Interventions
NameTypeDescription
ReFacto AFBIOLOGICAL -
B-Domain deleted Recombinant Factor VIIIGENETIC -
BDDrFVIIIGENETIC -
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Eligibility Criteria
Age Range12 Years — N/A
SexMALE
Healthy VolunteersNo
Study Sites17

* Male, at least 12 years old, with severe hemophilia A undergoing major elective major surgery requiring FVIII replacement for at least 6 days. Negative FVIII inhibitor test at screening and no past history of inhibitor. * Previously treated with at least 150 exposure days to any Factor VIII produc...

Countries:United StatesAustraliaAustriaHungaryNew ZealandPolandRomaniaSwedenBelgiumFinlandFranceGermanyItalyNetherlandsSpain
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