Pfizer Inc. (NYSE: PFE), a US pharmaceutical giant, has announced positive topline results from the Phase III AFFINE study for its gene therapy candidate, giroctocogene fitelparvovec, for the treatment of hemophilia A. With these results, Pfizer is now poised to prepare for regulatory submission for this one-time gene therapy, which is a product of its collaboration with Sangamo Therapeutics. This development complements the recent approval of Beqvez (fidanacogene elaparvovec), another one-time gene therapy for hemophilia B, acquired from Spark Therapeutics and approved in the US in April this year.
The AFFINE study (NCT04370054) aimed to establish the non-inferiority or superiority of giroctocogene fitelparvovec in reducing the total annualized bleeding rate (ABR) from week 12 to at least 15 months post-infusion, compared to routine Factor VIII (FVIII) treatment, with a focus on patients with moderate-to-severe hemophilia A. Pfizer’s candidate demonstrated a statistically significant reduction in mean total ABR compared to the pre-infusion period (1.24 bleeds versus 4.73; p-value=0.0040). Notably, only one patient (1.3%) reverted to Factor VIII treatment during the study. The treatment was generally well-tolerated, with 10 patients (13.3%) experiencing 13 serious adverse events considered related to the treatment, all of which were generally resolved with clinical management.
Pfizer’s goal, as outlined in its press release, is for a single infusion of giroctocogene fitelparvovec to enable hemophilia A patients to produce FVIII for an extended period, offering protection against bleeding episodes and reducing the reliance on routine prophylaxis through intravenous (IV) infusions or injections. The company plans to engage in discussions with regulatory authorities regarding the results in the coming months and will present further data at upcoming medical conferences.- Flcube.com
