Pfizer's gene therapy for hemophilia treatment approved by Health Canada, company says

On Wednesday, Pfizer announced that its gene therapy for treating a rare inherited bleeding disorder known as hemophilia B had been given approval by Health Canada. The endorsement was decided on the back of late-stage trials that demonstrated the single-dose therapy, to be marketed as Beqvez, outperformed the current standard treatment involving the replacement of a blood clotting protein named factor IX, according to a company statement. The therapy is intended for patients over 18 who suffer from moderate to severe hemophilia B. In November 2022, the U.S. Food and Drug Administration (FDA) sanctioned CSL’s drug, Hemgenix, thus becoming the first gene therapy for hemophilia B which can be administered once. The global rights for Hemgenix were acquired by CSL from uniQure NV, a gene therapy manufacturer, in 2021. Meanwhile, Pfizer is looking to get U.S. approval for an experimental antibody, marstacimab, for the treatment of both hemophilia A and B. Hemophilia is an inherited disorder where blood does not normally clot, the Canadian Hemophilia Society explains. Hemophilia patients may bleed spontaneously, unusually or for more extended periods. Hemophilia B affects 1 in 40,000 individuals and constitutes approximately 15 per cent of all hemophilia patients, the FDA clarifies. The U.S. health regulator is anticipated to announce its verdict on Pfizer’s therapy in the second quarter of 2024.