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Testing of hepatic AAV Vector integration

Phase 3

Hemophilia A | Unknown | Hematology |Pfizer, Inc.|Last Updated: May 27, 2026

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
CONTROLLEDDMC
Total Trials1
Total Enrollment173
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT05568719Safety and Effectiveness of Giroctocogene Fitelparvovec or Fidanacogene Elaparvovec in Patients With Hemophilia A or B RespectivelyPHASE3 RECRUITING 173Dec 28, 2022Feb 25, 2040May 27, 202623 United States, Australia +5
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Study Endpoints
Primary Endpoints
Incidence of thromboembolic events
Day 1 to 10 years
Incidence of factor inhibitor development
Day 1 to 10 years

FIX inhibitor development was defined as an inhibitor titer \>= 0.6 Bethesda units per milliliter (BU/mL).

Incidence of hepatic malignancy
Day 1 to 10 years
Incidence of liver abnormalities
Day 1 to 10 years
Factor activity level
Day 1 to 10 years

Factor activity level will be reported. Factor levels may be measured using different assay methods including a one-stage assay or by chromogenic substrate assay and a second one-stage assay.

Secondary Endpoints
Total ABR (treated or untreated; (excluding bleeds related to surgery)
Day 1 to 10 years
Incidence of and time from vector infusion to resumption of prophylaxis
Day 1 to 10 years
AIR of exogenous factor (excluding infusions related to surgery)
Day 1 to 10 years
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Study Design & Arms
AllocationNON_RANDOMIZED
MaskingNONE
ModelSINGLE_GROUP
PurposeOTHER
Treatment Arms
ArmTypeDescription
Hemophilia A / giroctocogene fitelparvovecOTHERParticipants have received treatment with giroctocogene fitelparvovec in a previous study and are not receiving any investigational product in this study
Hemophilia B / fidanacogene elaparvovecOTHERParticipants have received treatment with fidanacogene elaparvovec in a previous study and are not receiving any investigational product in this study
Interventions
NameTypeDescription
Testing of hepatic AAV Vector integrationDIAGNOSTIC_TESTEvaluation of AAV vector integration in participants for whom a sample of liver has been obtained through biopsy or surgical resection when clinically indicated
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Eligibility Criteria
Age Range18 Years — N/A
SexMALE
Healthy VolunteersNo
Study Sites23

Inclusion Criteria: -Only participants who received investigational giroctocogene fitelparvovec or fidanacogene eleparvovec and were enrolled in a Pfizer-sponsored study (C0371002, C0371003, C0371005, C3731001, C3731003) are eligible. Exclusion Criteria: -None

Countries:United StatesAustraliaCanadaSouth KoreaSwedenTaiwanTurkey (Türkiye)
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Recent Changes (Last 90 Days)
LOWMay 28, 2026NCT05568719lastUpdatePostDate: changed
LOWMay 28, 2026NCT05568719lastUpdatePostDate: changed
LOWMay 26, 2026NCT05568719primaryCompletionDate: changed
LOWMay 24, 2026NCT05568719studyFirstPostDate: changed