Pfizer Canada has received approval from Health Canada for its adeno-associated viral (AAV) vector-based gene therapy Beqvez (fidanacogene elaparvovec) for the treatment of haemophilia B.
A rare genetic bleeding disorder predominantly affecting males, haemophilia B impairs blood clotting due to a deficiency in clotting factors.
Individuals with this condition are susceptible to excessive and recurrent bleeding, which can be life-threatening, especially in severe cases.
The treatment is indicated for adult patients aged 18 years or above with moderately severe to severe haemophilia B with congenital Factor IX (FIX) deficiency and specifically for those without neutralising antibodies to variant AAV rhesus serotype 74.
The approval is based on results from the Phase III BENEGENE-2 clinical trial.
This single-arm, open-label trial is designed to assess the safety and efficacy of fidanacogene elaparvovec in adult males aged between 18 and 65 years with moderately severe to severe haemophilia B.
The study's primary goal was to compare the annualised bleeding rate in subjects who received gene therapy to those on a FIX prophylaxis replacement regimen, which is part of standard care.
A total of 45 participants were enrolled in the study.
Beqvez employs a bioengineered AAV capsid to offer a high-activity human FIX gene.
This gene therapy aims to enable eligible individuals with haemophilia B to produce FIX on their own following a one-time treatment, potentially eliminating the need for frequent intravenous FIX infusions.
Standard treatment involves regular intravenous infusions of plasma-derived or recombinant FIX to manage and prevent bleeding episodes.
Pfizer Canada speciality business unit Canada lead Frédéric Lavoie stated: “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.”
Last month, Pfizer received regulatory approval to conclude the acquisition of biotechnology company Seagen for $43bn in cash.
