Fidanacogene elaparvovec

Fidanacogene elaparvovec is a liver-specific adeno-associated virus (AAV) vector containing a codon-optimized human coagulation FIX gene that was investigated as a potential treatment for hemophilia B.^L46691 Hemophilia B is a rare X-linked genetic disorder characterized by abnormal coagulation due to dysfunctional coagulation factor IX with a male incidence estimated to be 1 in 30,000 male births worldwide.^A32552 Disease severity is linked to the level of factor IX activity in the blood plasma, ranging from increased bleeding after injuries and surgical operations to spontaneous bleeding, hemorrhages in soft tissues or joints, and severe subcutaneous hematomas. Current available treatments include substitution therapy involving the intravenous administration of standard factor IX once a week for bleeding or as prophylaxis and 2–3 times a week for severe hemophilia, although this carries a significant financial burden to the healthcare system. Other more radical approaches like liver transplants have also been considered.^A262929

On January 3, 2024, fidanacogene elaparvovec was approved by Health Canada under the brand name BEQVEZ for the treatment of adults with moderately severe to severe hemophilia B (congenital Factor IX (FIX) deficiency) who are negative for neutralizing antibodies to variant AAV serotype Rh74.^L49469 This approval was based on positive results demonstrated in the open-label, single-arm Phase 3 BENEGENE-2 study, where the annualized bleeding rate (ABR) was observed to be 1.3 for the 12 months from week 12 to month 15 compared to an ABR of 4.43 for the pre-treatment period.L49469,L46691 Fidanacogene elaparvovec was also approved by the FDA on April 26, 2024.^L50577