First Canadian patient receives gene therapy post-approval

Montreal, May 20, 2026 – The first Canadian patient received HEMGENIX, gene therapy for hemophilia B (following regulatory approval and commercialization), in London, Ontario, on May 13. Several other Canadian patients have previously been treated in hemophilia A and B clinical trials.

“This is a meaningful moment for the bleeding disorders community in Canada,” said Emil Wijnker, President of the Canadian Hemophilia Society. “For many, hemophilia B is a constant consideration in everyday life. Advances like gene therapy signal real progress, with the potential to ease that burden and meaningfully change how people manage their condition over time.”

HEMGENIX has performed well in clinical trials. After a single infusion, more than 90 percent of recipients show stable factor IX expression in the mild hemophilia range (5–50 percent) or normal range (greater than 50 percent), starting several weeks after treatment and lasting for a period of five years or more, and have stopped prophylaxis. For more information on the latest research on HEMGENIX, see this article.

In February and March 2026, Ontario and British Columbia agreed to reimburse HEMGENIX through their public drug plans. Decisions in the rest of the country are expected at any time. “We hope that the other provinces will quickly follow British Columbia’s and Ontario’s examples so that there is equity in access across Canada,” said Emil Wijnker. For details on Ontario and British Columbia drug plan reimbursement, see this update.

The Canadian Bleeding Disorder Registry, managed by the Association of Hemophilia Clinic Directors of Canada, records health outcomes in all patients with bleeding disorders. It has recently developed a gene therapy module to monitor the safety and efficacy of novel treatments like HEMGENIX over the long term — decades rather than years. A similar gene therapy registry, run by the World Federation of Hemophilia, collects data from countries around the world.

Note that HEMGENIX (etranacogene dezaparvovec) is approved by Health Canada for the treatment of adults (18 years of age or older) with hemophilia B (congenital Factor IX deficiency) who require routine prophylaxis to prevent or reduce the frequency of bleeding episodes. There is no clinical experience with HEMGENIX use in patients with mild or moderate hemophilia B (FIX activity > 2%). There are approximately 800 people with hemophilia B in Canada. Of these, 625 are over 18 years of age, and about 180 have severe or moderately severe disease (less than 2 percent FIX expression).

For more information, contact the CHS at chs@hemophilia.ca or (514) 848-0503, (800) 668-2686.