The Pfizer and Sangamo Therapeutics phase 3 AFFINE study assessing the efficacy and safety of giroctocogene fitelparvovec, a novel gene therapy for patients with moderately severe to severe hemophilia A, has reopened recruitment. The reopening, announced by news release, will resume enrollment this month and dosing is expected to restart in October.
Giroctocogene fitelparvovec is composed of a recombinant adeno-associated virus serotype 6 vector, which encodes the complementary DNA for B domain depleted human FVIII. It is designed to generate optimal expression of FVIII protein in patients.
In a phase 1/2 study of giroctocogene fitelparvovec (the Alta study), a single infusion in patients with severe hemophilia A was well tolerated with no sustained adverse events and minimal bleeding events.
Giroctocogene fitelparvovec was granted Orphan Drug, Fast Track, and regenerative medicine advanced therapy designations by the US Food and Drug Administration, and Orphan Medicinal Product status from the European Medicines Agency.
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The phase 3 study is an open-label, multicenter, single-arm study evaluating a single infusion of giroctocogene fitelparvovec in more than 60 male patients with hemophilia A. In order to gather pretreatment data on efficacy and safety, eligible patients will have completed at least 6 months of FVIII prophylaxis as part of the lead-in phase 3 study.
The primary study endpoint is the change in annualized bleeding rates (ABR) over a 15-month period after giroctocogene fitelparvovec treatment. This endpoint will be compared with the ABR on FVIII prophylaxis therapy alone. Secondary endpoints are the level of FVIII activity after steady state onset and over a 15-month period after the giroctocogene fitelparvovec infusion.
Pfizer and Sangamo Therapeutics announce phase 3 trial of investigational gene therapy for hemophilia A has re-opened recruitment. News release. Pfizer; September 22, 2022.