The CHS pressures provinces to reimburse hemophilia B gene therapy

Montréal, December 17, 2025 – The Canadian Hemophilia Society (CHS) has written to the Provincial/Territorial Ministers of Health (excluding Quebec) to urge them to add etranacogene dezaparvovec (Hemgenix), gene therapy for hemophilia B, to their drug formularies.

In October, after months of negotiations, the pan-Canadian Pharmaceutical Alliance and the manufacturer of Hemgenix, CSL Behring, agreed on pricing. This agreement applies to all public drug plans in Canada.

Unlike previous coagulation products, Hemgenix is not being supplied via Canadian Blood Services. Instead, it will be treated like many other drugs supplied via provincial drug formularies. As a result, each province or territory will make its own decision on reimbursement. An alternative pathway for provinces not planning to provide gene therapy locally is to enter into agreements with other jurisdictions to send patients out-of-province for administration.

“With the goal of equity,” said CHS President Emil Wijnker, “the CHS is calling on all provinces and territories to agree to reimburse.”

The CHS letter to Ministers of Health highlighted five-year data on Hemgenix published in the New England Journal of Medicine on December 7, 2025. The Final analysis of A Study of Etranacogene Dezaparvovec for Hemophilia B provides strong evidence on the safety, efficacy and durability of the therapy. Key points include:

1. Fifty (50) of the 54 patients enrolled completed 60 months of follow-up. One person withdrew efficacy consent following low expression of factor IX (FIX). A second had a liver transplant and efficacy data collection ceased. Two people died of causes unrelated to the drug.
2. Only three of the remaining 50 subjects require continuous FIX prophylaxis (all previously reported).
3. The remaining 47 subjects do not require prophylaxis.
4. No one resumed FIX prophylaxis during Years 4 and 5.
5. The mean FIX activity level remained stable and greater than 36% during Years 1–5 post-gene therapy.

Year 1: 41.5%
Year 2: 36.7%
Year 3: 38.6%
Year 4: 37.4%
Year 5: 36.1%

6. The results for the 54 patients were as follows:

Missing data (see point 1): 4 people
Lack of efficacy: 2 people
5-12% FIX: 5 people
12-40% FIX: 26 people
40-100% FIX: 17 people

7. Similar outcomes were observed between AAV5 NAb-positive and NAb-negative individuals.
8. The Annual Bleeding Rates for all bleeds in all 54 patients were …

Year 1: 1.33
Year 2: 0.91
Year 3: 0.83
Year 4: 0.40
Year 5: 0.40

9. Most of these bleeds were in the three patients who returned to prophylaxis.
10. Mean annual consumption of FIX for the 54 people was as follows:

Lead-in period: 257,339 IUs
Year 1: 10,532 IUs
Year 2: 8,777 IUs
Year 3: 10,218 IUs
Year 4: 9, 421 IUs
Year 5: 10,900 IUs

The letter concluded that “Hemgenix will be a highly effective therapy for ten years and more in the vast majority of recipients and that, as a result, it will not only be life-changing for patients but will also result in long-term savings to the health system.”

Hemgenix is currently reimbursed in Austria, Denmark, France, Germany, Ireland, Italy, Spain, the United Kingdom and the U.S.

 

QUÉBEC

The health technology assessment (HTA) body in Quebec, INESSS, has not yet recognized the therapeutic value of Hemgenix. The Québec Chapter of the CHS continues its efforts to reverse this assessment. It believes that the five-year data just released make a strong and compelling case as to the safety, efficacy and durability of this major treatment advance.

 

For more information on gene therapy for hemophilia, please consult the Canadian Hemophilia Society Gene Therapy Education program at www.hemophilia.ca/gene-therapy.