The approval was based on the open-label, single-arm Phase 3 BENEGENE-2 study in which a single dose of the therapy was found superior to the current standard of care
Pfizer has secured Health Canada approval for haemophilia B therapy. (Credit: MasterTux from Pixabay)
Pfizer Canada has received Health Canada’s approval for Beqvez (fidanacogene elaparvovec) to treat adult patients with moderately severe to severe haemophilia B ahead of the US approval.
Beqvez is an adeno-associated viral (AAV) vector-based gene therapy.
Health Canada has approved the therapy to treat haemophilia B patients aged 18 years or older who tested negative for neutralising antibodies to variant AAV serotype Rh74.
Pfizer Canada speciality business unit Canada lead Frédéric Lavoie said: “Pfizer has more than 30 years of experience in developing and commercialising therapies for haematological disorders, and a deep understanding of the significant challenges that people living with haemophilia continually face.
“We are proud to introduce an innovative therapy for people living with haemophilia B in the form of gene therapy.”
The approval was based on the open-label, single-arm Phase 3 BENEGENE-2 study which enrolled 45 people.
The trial assessed the efficacy and safety of fidanacogene elaparvovec in adult male participants aged 18–65 with haemophilia B.
The primary objective of the study was to compare the annualised bleeding rate (ABR) of individuals receiving gene therapy against those receiving factor IX prophylactic replacement regimens as standard care.
According to the results, a single dose of the therapy was found superior to the current standard of care.
McMaster University Health Research Methods, Evidence, and Impact professor and chair Alfonso Iorio said: “Our team at McMaster University is proud to have been a clinical trial site in Canada that studied this therapy.
“This new treatment shows potential in reducing the burden of frequent infusions by providing, with a single infusion, a persistent liver production of factor IX, which could help improve the life of those affected.”
The US Food and Drug Administration (FDA) is anticipated to deliver its approval on Pfizer’s therapy in the second quarter of this year.
In November 2022, the FDA approved CSL’s Hemgenix, making it the first one-time gene treatment for haemophilia B.
Pfizer also plans to secure approval in the US for marstacimab, an investigational antibody, to treat haemophilia A and B.