Posted by Michael Wonder on 26 Apr 2024
US FDA approves Pfizer’s Beqvez (fidanacogene elaparvovec-dzkt), a one-time gene therapy for adults with hemophilia B
26 April 2024 - Pfizer announced today that the US FDA has approved Beqvez (fidanacogene elaparvovec-dzkt) for the treatment of adults with moderate to severe haemophilia B who currently use factor IX (FIX) prophylaxis therapy, or have current or historical life-threatening haemorrhage, or have repeated, serious spontaneous bleeding episodes, and do not have neutralising antibodies to adeno-associated virus serotype Rh74var capsid as detected by an FDA-approved test.
The FDA approval is based on the results from the pivotal BENEGENE-2 study, a Phase 3, open-label, single-arm study to evaluate the efficacy and safety of Beqvez in adult male participants (age 18–65) with moderately severe to severe haemophilia B (defined as FIX circulating activity of 2% or less).
