Pfizer rings in New Year with hemophilia B gene therapy nod in Canada

After a pair of high-profile approvals for sickle cell disease gene therapies in the U.S. last month, Pfizer is ushering in the New Year with a regulatory endorsement for its hemophilia B gene therapy north of the border.

Health Canada has approved Pfizer's adeno-associated viral (AAV) vector-based gene therapy, Beqvez, for the treatment of certain adults with hemophilia B, the company said Wednesday.

The agency endorsed the drug to treat patients with moderate to severe disease who are negative for neutralizing antibodies to variant AAV serotype Rh74.

The approval is based on an open-label, single-arm study called BENEGENE-2, which enrolled 45 patients and met its primary goal. In the study, Pfizer's drug demonstrated noninferiority and superiority to the standard of care, a recurrent Factor IX regimen, on the measure of annualized bleeding rates.

Traditionally, patients with hemophilia B have needed to receive routine infusions of Factor IX treatment to prevent and control bleeding. But with Beqvez and its rival Hemgenix from CSL Behring, patients can receive a one-time treatment to produce their own Factor IX.

CSL's Hemgenix won its Canadian approval back in October 2023.

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