The approval was based on the results from the Phase 3 BENEGENE-2 study in which Beqvez demonstrated non-inferiority against current standard of care
FDA approves Pfizer's Beqvez for haemophilia B. (Credit: Pfizer Inc.)
Pfizer has received the US Food and Drug Administration (FDA) approval for Beqvez (fidanacogene elaparvovec-dzkt) to treat certain adult patients with moderate to severe haemophilia B.
Beqvez is a one-time, adeno-associated virus (AAV)-based gene therapy. It was approved in Canada for the same indication in January.
The FDA approval is for individuals undergoing factor IX (FIX) prophylaxis therapy, experiencing current or historical life-threatening haemorrhage, or facing repeated, serious spontaneous bleeding episodes.
Additionally, these patients should not have neutralising antibodies to adeno-associated virus serotype Rh74var (AAVRh74var) capsid, as detected by an FDA-approved test.
Pfizer US chief commercial officer and executive vice president Aamir Malik said: “This milestone is a testament to Pfizer’s continued effort to advance the standard of care for people living with haemophilia, with the delivery of a medicine that has the potential to offer both long-term bleed protection and value to the healthcare system because of its one-time administration.
“We are leveraging our expertise that comes with more than 40 years of experience in the haemophilia space, and are proactively working with treatment centres, payers, and the haemophilia community to appropriately help ensure the healthcare system is prepared to readily deliver Beqvez to the patients who can benefit from it.”
The FDA’s approval was based on the results from the Phase 3, open-label, single-arm BENEGENE-2 study.
The study assessed the efficacy and safety of Beqvez in 45 adults aged between 18–65.
The late-stage trial achieved its primary endpoint of demonstrating non-inferiority in the annualised bleeding rate (ABR) of total bleeds following Beqvez infusion compared to the prophylaxis regimen with FIX.
During the efficacy evaluation period, patients who received Beqvez experienced a mean ABR of 2.5 against a mean ABR of 4.5 observed during the lead-in pre-treatment period lasting at least six months.
Additionally, 60% of patients achieved elimination of bleeds, compared to 29% in the prophylaxis group. The median ABR was zero during the efficacy evaluation period and was 1.3 in the prophylaxis group.
The participants in the trial will be monitored for a cumulative period of 15 years. This includes six years within the BENEGENE-2 study and an additional nine years as part of a different Phase 3 study to understand the long-term safety and effectiveness of Beqvez.