Fidanacogene Elaparvovec-dzkt Approved by FDA for Hemophilia B

The US Food and Drug Administration (FDA) has approved fidanacogene elaparvovec-dzkt, a one-time gene therapy for hemophilia B developed by Pfizer, according to a press release.

Fidanacogene elaparvovec-dzkt is indicated for adult patients with moderate or severe hemophilia B who either are currently on factor IX (FIX) therapy, have current or historical life-threatening hemorrhage, or have repeated and serious spontaneous bleeding episodes. Potential patients must also not have neutralizing antibodies against adeno-associated virus serotype Rh74var capsid.

Gene Therapy Approved for Hemophilia B Treatment

“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,” stated Adam Cuker, MD, the Director of the Penn Comprehensive and Hemophilia Thrombosis Program. “A one-time treatment with [fidanacogene elaparvovec-dzkt] has the potential to be transformative for appropriate patients by reducing both the medical and treatment burden over the long term.”

The approval of fidanacogene elaparvovec-dzkt was supported by the phase III BENEGENE-2 study, which enrolled 45 adult male patients with moderately severe to severe hemophilia B defined as FIX circulating activity of 2% or less. In the study, fidanacogene elaparvovec-dzkt showed noninferiority in annualized bleeding rate of total bleeds post infusion compared with standard FIX prophylaxis.

Fidanacogene elaparvovec-dzkt recently received regulatory approval in Canada and is currently undergoing review by the European Medicines Agency for approval in the European Union.

Related: FDA Approves Expanded Indication for IXINITY to Include All Patients With Hemophilia B