The following is an excerpt from a statement from Pfizer. Read the statement in its entiretyÂ here.
AFFINE is a global Phase 3, open-label, multicenter, single arm study that will evaluate the efficacy and safety of giroctocogene fitelparvovec in patients with moderately severe to severe hemophilia A. The primary endpoint is impact on annual bleed rate (ABR) through 12 months following treatment with giroctocogene fitelparvovec, compared to ABR on Factor VIII (FVIII) replacement therapy collected in the Phase 3 lead-in study period. Participants will be analyzed throughout the 5-year study period following the single infusion to further assess the durability and efficacy.
“The initiation of the pivotal Phase 3 dosing study of giroctocogene fitelparvovec is a significant achievement for Pfizer as we continue our longstanding commitment to improving care for the hemophilia community,” said Brenda Cooperstone, Chief Development Officer, Rare Disease, Pfizer Global Product Development. “Enrollment in the lead-in study is progressing well and recruitment is on track for Phase 3. Given the Phase 1/2 study findings to date, we believe that giroctocogene fitelparvovec has the potential to sustain factor levels and reduce annual bleed rates, suggesting this one-time gene therapy could potentially transform the standard of care for eligible patients worldwide.”
Data from the Phase 3 lead-in study will provide a baseline for patients evaluated in the Phase 3 study. Updated Phase 1/2 data announced at a Pfizer investor event on September 15, 2020 demonstrated that giroctocogene fitelparvovec was generally well tolerated. Each of the five patients in the high dose cohort sustained FVIII activity levels without bleeds or the need for prophylactic factor through up to 85 weeks. Factor VIII activity levels were sustained at a clinically meaningful level, with a geometric mean of ~71% when measured between the weeks of 9 and 52.
Read the statement in its entiretyÂ here.