First patient dosed in Phase III hemophilia A trial could narrow gap between Pfizer/Sangamo and BioMarin

Pfizer and Sangamo dosed the first patient in their Phase III AFFINE study of giroctocogene fitelparvovec, and with the trial’s endpoint follow-up at 12 months, it could put the partners very close to the timing of key competitor BioMarin in the race for approval of the first hemophilia A gene therapy.

Giroctocogene fitelparvovec (formerly SB-525) will be tested in an open-label, multi-center, single-arm trial, and follow moderate-to-severe hemophilia A patients up to five years, with the primary endpoint, impact on annual bleed rate (ABR), collected through the first year. Investigators will compare ABR from giroctocogene fitelparvovec with ABR from Factor VIII, the standard of care, collected during the Phase III lead-in study...