Update on novel therapies for hemophilia A and B

MONTRÉAL, September 19, 2025 – No announcements have been made over the summer on progress in gaining access to two novel hemophilia therapies.

Altuviiio for hemophilia A
In June, as previously reported, both Canada’s Drug Agency (CDA) and Quebec’s Institut national d’excellence en santé et en services sociaux (INESSS) released their final recommendations on Altuviiio, an ultra-extended-half-life factor VIII concentrate. They recommended that Altuviiio be reimbursed for adults and children with hemophilia A for …

• routine prophylaxis to prevent or reduce the frequency of bleeding episodes
• treatment and control of bleeding episodes, and
• perioperative management of bleeding (surgery).

Despite CHS pressure to accelerate the process, the timeline to gain access in the provinces and territories served by Canadian Blood Services (CBS) remains unchanged. Next steps include:

• price negotiations between CBS and the manufacturer, Sanofi
• a decision, following price negotiations, by the Provinces/Territories to add Altuviiio to the CBS National Formulary
• a formal procurement contract, and
• preparations for blood banks to handle the new product.

CBS has estimated that these steps will take at least one year, meaning that Altuviiio would not be available until the second or third quarter of 2026.

In Québec, it remains to be seen whether Altuviiio can be added to the Héma-Québec formulary as an “innovative therapy” or whether it will be included in the next product tender, which will not take effect until April 1, 2027.

For more information on Altuviiio, CLICK HERE to access the booklet called “All About Novel Therapies.”

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Hemgenix gene therapy for hemophilia B
Negotiations between CSL Behring Canada, the manufacturer of Hemgenix, and the pan-Canadian Pharmaceutical Alliance (pCPA), representing Canada’s public drug plans, have been going on for more than one year. There has been no news of a successful conclusion. If an agreement is reached, it will then be up to each province, one by one, to decide whether or not to add Hemgenix to its drug formulary.

Québec did not participate in the pCPA negotiations following a negative recommendation on Hemgenix access from INESSS. CSL Behring Canada is expected to make a new submission in the coming months with longer-term outcome data.

For more information on Hemgenix, CLICK HERE to access the booklet “All About Hemophilia Gene Therapy.”