Health Canada has approved Pfizer’s BEQVEZ (fidanacogene elaparvovec), an adeno-associated viral (AAV) vector-based gene therapy for the treatment of adults (aged 18 years or older) with moderately severe to severe hemophilia B (congenital Factor IX (FIX) deficiency) who are negative for neutralizing antibodies to variant AAV serotype Rh74.
“Pfizer has more than 30 years of experience in developing and commercializing therapies for hematological disorders, and a deep understanding of the significant challenges that people living with hemophilia continually face,” said Frédéric Lavoie, Canada Lead – Specialty Business Unit, Pfizer Canada ULC. “We are proud to introduce an innovative therapy for people living with hemophilia B in the form of gene therapy.”
Health Canada’s approval for BEQVEZ is based on the open label, single arm Phase 3 BENEGENE-2 study, to evaluate the efficacy and safety of fidanacogene elaparvovec in adult male participants (age 18–65) with moderately severe to severe hemophilia B (defined as FIX circulating activity of 2% or less).
The main objective of the study was to evaluate the annualized bleeding rate (ABR) for participants treated with gene therapy versus FIX prophylaxis replacement regimen, administered as part of usual care. The study enrolled 45 participants.
“Our team at McMaster University is proud to have been a clinical trial site in Canada that studied this therapy,” expressed Dr. Alfonso Iorio, Professor and Chair, Health Research Methods, Evidence, and Impact at McMaster University. “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.”
Based on the World Federation of Hemophilia, more than 38,000 people worldwide were living with hemophilia B in 2021. In Canada, hemophilia B affects over 700 individuals.
“The Canadian Hemophilia Society welcomes the approval of BEQVEZ, the second gene therapy for hemophilia B to receive its Notice of Compliance from Health Canada in recent months,” said Wendy Quinn, Canadian Hemophilia Society (CHS) President. “Our hope is that gene therapy will be made available to those who are eligible and have made an informed decision to proceed.
“We are committed to ensuring education and support for our community as gene therapies have the potential to fundamentally transform treatment and improve quality of life for some individuals. The CHS will work closely with health authorities in the provinces and territories to ensure that gene therapies for hemophilia B become accessible to eligible patients across Canada as soon as possible.”
