RARE Daily

Health Canada Approves Pfizer Hemophilia B Gene Therapy

January 4, 2024

Rare Daily Staff

Health Canada approved Pfizer Canada’s Beqvez, an AAV gene therapy for the treatment of adults with moderately severe to severe hemophilia B, a rare bleeding disorder.

Hemophilia B is a rare, genetic, life-threatening degenerative disease. People with the condition are particularly vulnerable to bleeds in their joints, muscles, and internal organs, leading to pain, swelling, and joint damage. Current treatment includes life-long prophylactic infusions of factor IX (FIX) to temporarily replace or supplement low levels of the blood-clotting factor. 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 more than 700 individuals.

Gene therapy has the potential to make more normal clotting ability possible in hemophilia B. In November, the U.S. Food and Drug Administration approved Hemgenix, the first gene therapy for adults with hemophilia B from CSL Behring and UniQure, which carries a $3.5 million price tag.

Pfizer’s Beqvez contains a bio-engineered adeno-associated virus (AAV) protein shell and a high-activity human coagulation FIX gene. The goal of this gene therapy for people living with hemophilia B, once treated, is that they will be able to produce FIX via this one-time treatment rather than having to regularly receive exogenous FIX.

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 Beqvez in adult male participants (age 18–65) with moderately severe to severe hemophilia B (defined as FIX circulating activity of 2 percent 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.

“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,” said Alfonso Iorio, professor and chair of Health Research Methods, Evidence, and Impact at McMaster University.

Photo: Alfonso Iorio, professor and chair of Health Research Methods, Evidence, and Impact at McMaster University

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