Pfizer Inc. (PFE) Wednesday said its Phase 3 AFFINE study of giroctocogene fitelparvovec, an investigational gene therapy for the treatment of adults with hemophilia A met primary goal.
Hemophilia A is a hereditary bleeding disorder caused by deficiency in blood clotting protein known as factor VIII, resulting in high susceptibility to bleeding, both internally and externally.
The Phase 3 AFFINE study was to evaluate the efficacy and safety of a single infusion of giroctocogene fitelparvovec in adult male participants with moderately severe to severe hemophilia A.
The primary endpoint measured the total annualized bleeding rate (ABR) from Week 12 through at least 15 months following treatment with giroctocogene fitelparvovec compared to total ABR on prior prophylaxis treatment.
Following a single dose, giroctocogene fitelparvovec demonstrated a statistically significant reduction in mean total ABR compared to the pre-infusion period.
Key secondary endpoints of the study were also met. Further, giroctocogene fitelparvovec was generally well tolerated.
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