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.

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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.

Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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