Health Canada approves Altuviiio
MONTRÉAL, March 31, 2025 – Health Canada has granted marketing approval to Altuviiio (efanesoctocog alfa) for the treatment of hemophilia A in children, adolescents and adults. It was approved by the U.S. FDA and by the European Medicines Agency in 2024 and is currently available to patients in the U.S. and many European countries.
Altuviiio is an ultra-extended half-life recombinant FVIII concentrate. It is infused intravenously. Its half-life is approximately 40 hours, compared to 12-14 hours for standard half-life FVIIIs and 18-20 hours for other extended half-life FVIIIs.
To extend half-life, efanesoctocog alfa connects three components to the FVIII molecule:
- Fc fusion to help FVIII recirculate;
- XTEN technology to shield FVIII from breaking down; and
- von Willebrand factor fragments to keep FVIII in the bloodstream longer.
Results from Phase 3 clinical trials showed:
- an average annual bleeding rate of 0.7 (lower than rates with prior FVIII prophylaxis);
- in adolescents and adults, after a dose of 50 IUs/kg once a week, an average FVIII level of 40% after 4 days, and 15% after 7 days;
- in children under 12 years, after a dose of 50 IUs/kg once a week, an average FVIII level of 40% for 2 to 3 days, and 10% after 7 days;
- 97% of bleeds resolved with one infusion;
- improvements in physical health, pain intensity and joint health;
- no new inhibitors;
- an acceptable side effect profile.
The next steps on the road to patient access are health technology assessments by the Institut national d’excellence en santé et en services sociaux (INESSS) for Quebec and by Canada’s Drug Agency (CDA) for the rest of Canada. Their recommendations to governments on reimbursement and pricing are expected in the coming weeks. It will then be up to Héma-Québec and Canadian Blood Services to negotiate contracts.
“The Canadian Hemophilia Society (CHS) welcomes Health Canada’s approval of Altuviiio,” said Emil Wijnker, CHS President. “Its ultra-extended half-life offers the possibility of once-a-week IV infusions, higher FVIII trough levels and better protection from bleeding. In turn, this can potentially result in improved joint health, quality-of-life and peace of mind for people with hemophilia A. The CHS calls on health authorities to make this product accessible across Canada in the very near future.”