European Commission Grants Approval for Pfizer’s DURVEQTIX® Gene Therapy for Hemophilia B
Pfizer Inc. (NYSE: PFE) today announced that the European Commission (EC) has granted conditional marketing authorization for DURVEQTIX® (fidanacogene elaparvovec), a gene therapy for the treatment of severe and moderately severe hemophilia B (congenital factor IX deficiency) in adult patients without a history of factor IX inhibitors and without detectable antibodies to variant AAV serotype Rh74. DURVEQTIX is designed to enable people living with hemophilia B to produce factor IX (FIX) themselves via a one-time dose, rather than multiple intravenous FIX infusions weekly or biweekly with the current standard of care.1,2,3
“There is a substantial medical and treatment burden for people with hemophilia B that receive standard of care today, with frequent infusions and many remaining at risk of breakthrough bleeds that can lead to pain and restricted mobility,” said Alexandre de Germay, Chief International Commercial Officer and Executive Vice President, Pfizer. “DURVEQTIX has shown the potential to offer long-term bleed protection in a one-time dose, reducing or eliminating bleeds for the appropriate patients with hemophilia B. These outcomes and their impact could become potentially transformative for hemophilia B care in the European Union.”
Hemophilia B is a rare genetic bleeding disorder that prevents normal blood clotting because of a deficiency in FIX that causes those with the disease to bleed more frequently and longer than others.1,4 The standard of care for hemophilia B treatment is prophylactic infusions of FIX replacement therapy that temporarily replace or supplement low levels of blood-clotting factor.1,2 Despite prophylaxis and regular intravenous infusions, many people living with moderate to severe hemophilia B are at risk of spontaneous bleeding episodes.5,6,7 The current standard of care also places strain on healthcare systems’ budgets and resource utilization.6,8,9,10 According to the World Federation of Hemophilia, more than 42,000 people worldwide are living with hemophilia B.11
The conditional marketing authorization is based on results from the pivotal Phase 3 BENEGENE-2 study (NCT03861273) evaluating the efficacy and safety of DURVEQTIX in adult male participants (age 18–62) with moderately severe to severe hemophilia B. BENEGENE-2 met its primary efficacy endpoint of non-inferiority and demonstrated a statistically significant decrease in annualized bleeding rate (ABR) for total bleeds (treated and untreated) post-DURVEQTIX infusion versus prophylaxis regimen with FIX, administered as part of usual care. Efficacy, based on ABR, also remained stable during year two to year four after treatment. DURVEQTIX was generally well-tolerated, with a safety profile consistent with Phase 1/2 results.
This conditional marketing authorization is valid in all 27 European Union (EU) member states, as well as in Iceland, Liechtenstein, and Norway. The EC approval follows recent regulatory approvals by the U.S. Food and Drug Administration (FDA) and Health Canada, where it is marketed as BEQVEZTM.
This milestone builds on Pfizer’s more than 40-year commitment to delivering breakthrough solutions to improve the lives of people living with hemophilia. In addition to DURVEQTIX, Pfizer recently reported positive results from a Phase 3 program investigating a gene therapy in hemophilia A (giroctocogene fitelparvovec). Additionally, a Phase 3 trial is investigating marstacimab, a novel, investigational, anti-tissue factor pathway inhibitor for the treatment of people with hemophilia A and B with and without inhibitors. A Biologics License Application and European Marketing Authorization Application for marstacimab for eligible patients without inhibitors are currently under review with the FDA and European Medicines Agency (EMA), respectively.