Fidanacogene Elaparvovec Gets European Approval for Hemophilia B
The European Commission granted conditional marketing authorization under the name Beqvez to the European Union.
Adam Cuker, MD
Credit: UPenn
The European Commission has granted conditional marketing authorization to Pfizer’s fidanacogene elaparvovecgene therapy under the name Durveqtix for the treatment of adults with severe and moderately severe hemophilia B without a history of factor IX (FIX) inhibitors and without detectable antibodies to variant adeno-associated virus (AAV) serotype Rh74.1
The approval comes a few months after the FDA approval of fidanacogene elaparvovec under the name Beqvez for patients with hemophilia B in the United States (US).2 The therapy costs $3.5 million in the US and was also approved in Canada in January 2024.4 Fidanacogene elaparvovec is a gene therapy that uses AAV to deliver a high-activity variant of human coagulation FIX gene to the liver in a single infusion.
“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,” Alexandre de Germay, Chief International Commercial Officer and Executive Vice President, Pfizer, said in a statement.1 “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.”
The approvals were based off ofdata from the phase 3 BENEGENE-2 clinical trial (NCT03861273) that assessed fidanacogene elaparvovec against the standard of care (SOC) FIX prophylaxis replacement regimen.The study treated 45 individuals with hemophilia B at a dose of 5x1011 vg/kg. It was deemed superior to SOC by investigators, reporting a mean ABR for all bleeds of 1.3 for 12 months—spanning from 12 weeks posttreatment to 15 months posttreatment—which was a 71% reduction from the lead-in study ABR (P <.0001).Investigators also found a 92% reduction in the annualized infusion rate of exogenous FIX following infusion with fidanacogene elaparvovec (P <.0001).3
Fidanacogene elaparvovec was well-tolerated, with a safety profile consistent with prior reports from the phase 1/2 studies. Overall, there were 14 serious adverse events (SAEs) observed in 7 patients (16%), none of which were related to infusion reactions, thrombotic events, or FIX inhibitors. Two of those SAEs occurred in a single patient and were deemed related to the treatment: a duodenal ulcer hemorrhage and anemia during corticosteroid use.3
“Many people with hemophilia B struggle with the commitment and lifestyle disruption of regular FIX infusions, as well as spontaneous bleeding episodes, which can lead to painful joint damage and mobility issues,” principal investigator Adam Cuker, MD, MS, the director of the Penn Comprehensive and Hemophilia Thrombosis Program, said in a statement about the US approval.2 “A 1-time treatment with BEQVEZ has the potential to be transformative for appropriate patients by reducing both the medical and treatment burden over the long term.”
