Health Canada approves Pfizer’s gene therapy for treatment of hemophilia

Pfizer said on Wednesday that Canada’s health regulator approved its gene therapy for the treatment of a rare inherited bleeding disorder called hemophilia B ahead of a U.S. decision.

The approval was based on late-stage trials that showed a single dose of the therapy, to be sold under the brand name Beqvez, was superior to the current standard of care which involves replacing a blood-clotting protein called factor IX, according to the company’s release.

The therapy is for patients over 18 living with moderately severe to severe hemophilia B.

The U.S. Food and Drug Administration (FDA) had in November 2022 approved pharmaceutical company CSL’s product Hemgenix, making it the first one-time gene therapy for hemophilia B.

CSL had acquired exclusive global rights to Hemgenix from gene therapy maker uniQure NV in 2021.

Pfizer is also seeking U.S. approval for its experimental antibody, marstacimab, to treat hemophilia A and B.

Beqvez is listed as an approved product on Health Canada’s online database. CBC News reached out to Health Canada for comment on Pfizer’s announcement.

Hemophilia is a hereditary condition in which blood does not clot normally, according to the Canadian Hemophilia Society. Patients who have the condition bleed spontaneously, excessively or for a longer time than is typical.

Hemophilia B is found in 1 in 40,000 people and represents about 15 per cent of patients with hemophilia, according to the FDA.

The U.S. health regulator is expected to give its decision on Pfizer’s therapy in the second quarter of 2024.