giroctocogene fitelparvovec + fidanacogene elaparvovec

This product is a **combination of two gene therapies** developed for hemophilia: giroctocogene fitelparvovec, for hemophilia A, and fidanacogene elaparvovec, for hemophilia B. Both use adeno-associated virus (AAV) vectors to deliver a functional gene to liver cells, enabling endogenous production of the missing clotting factor. - **Giroctocogene fitelparvovec** (PF-07055480, SB-525) is a recombinant AAV6-based gene therapy encoding a B-domain-deleted human factor VIII (F8) gene under control of a liver-specific promoter[2][8][11][17]. It aims to restore factor VIII production in hemophilia A patients, reducing or eliminating the need for regular infusions[5][8]. - **Fidanacogene elaparvovec** (PF-06838435, Spark100-hFIX-R338L, BEQVEZ) is an AAV-based gene therapy encoding a highly functional copy of the human factor IX (FIX) Padua gene for hemophilia B[16][20]. This therapy enables sustained FIX production, minimizing bleeding events and limiting the need for prophylactic infusions[4][20]. Both therapies are administered as a single intravenous infusion and have undergone Phase 3 trials with data showing significant reductions in annualized bleeding rates and sustained factor activity[17][20].