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Pfizer's Hemophilia B Gene Therapy Approved in Canada

NEW YORK – Health Canada has approved Pfizer's Beqvez (fidanacogene elaparvovec) as a gene therapy treatment for hemophilia B, the drugmaker said Wednesday.

The gene therapy is currently under regulatory review in the US and EU.

In Canada, Beqvez has been approved as a treatment for adults with moderately severe to severe hemophilia B, a rare genetic bleeding disorder mostly seen in men and caused by a deficiency in the blood-clotting protein FIX, which is produced by the eponymous gene. Patients must also be negative for neutralizing antibodies to variant adeno-associated viral (AAV) serotype Rh74 to receive the gene therapy.

The one-time gene therapy uses an AAV vector to deliver a variant of the FIX gene, after which patients are expected to be able to produce FIX on their own without regular intravenous infusions of plasma-derived or recombinant FIX — the current standard care for the disease.

Health Canada approved Beqvez after reviewing data from Pfizer's open-label, single-arm Phase III study, dubbed BENEGENE-2, which enrolled 45 adult male patients with moderately severe to severe hemophilia B. Investigators compared the annualized bleeding rate in patients treated with the gene therapy against those who received standard FIX prophylaxis replacement regimen. Pfizer reported previously that the mean annualized bleeding rate over 12 months was 1.3 with Beqvez and 4.43 in those getting intravenous FIX infusions for at least six months during the lead-in pretreatment period. 

Beqvez is the second gene therapy approved for hemophilia B in Canada, following CSL Behring's Hemgenix (etranacogene dezaparvovec) in October.