Pfizer Phase III hemophilia A gene therapy trial meets goals

Positive top-line results from the Phase III AFFINE study (NCT04370054) evaluating giroctocogene fitelparvovec, an investigational gene therapy for the treatment of adults with moderately severe to severe hemophilia A, were released today by US pharma giant Pfizer (NYSE: PFE).

The drug is being jointly developed with biotech company Sangamo Therapeutics (Nasdaq: SGMO).

The AFFINE study achieved its primary objective of non-inferiority, as well as superiority, of total annualized bleeding rate (ABR) from Week 12 through at least 15 months of follow up post-infusion compared with routine Factor VIII (FVIII) replacement prophylaxis treatment. Following a single 3e13 vg/kg dose, giroctocogene fitelparvovec demonstrated a statistically-significant reduction in mean total ABR compared to the pre-infusion period (1.24 vs 4.73; one-sided p-value=0.0040).