Canada approves Pfizer's gene therapy for bleeding disorder

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.

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

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

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

Hemophilia B is found in 1 in 40,000 people and represents about 15% 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.