Gene therapy for hemophilia B one step closer: CSL Behring and pCPA agree on pricing

MONTREAL, October 7, 2025 – CSL Behring Canada and the pan-Canadian Pharmaceutical Alliance (pCPA) have announced signing of a Letter of Intent that sets pricing conditions for the reimbursement of Hemgenix, gene therapy for hemophilia B. This agreement applies to public drug plans with the exception of Québec. Details of the Letter of Intent between pCPA, representing governments, and CSL Behring are confidential; however, the Canadian Hemophilia Society (CHS) has learned that some countries in Europe have adopted novel models of reimbursement for gene therapy that include payments to the manufacturer spaced out over a number of years with risk-sharing arrangements in case the therapy is ineffective in certain cases. Such an approach has the potential to reduce short-term budget impact and, in the long term, be cost saving for the health system.

The Letter of Intent paves the way for the next step in making the decades-old dream of gene therapy a reality for Canadian patients—decisions by provinces and territories to add Hemgenix to their drug formularies.

“Having treatment options that meet the individual needs of people living with hemophilia B is important,” said Emil Wijnker, president of the CHS. “The Letter of Intent is an encouraging step forward, and we urge the provinces and territories to work together, and with CSL Behring, to make this therapy available to those who need it as soon as possible.”

Clinicians also welcomed the announcement. “The availability of gene therapy for hemophilia B represents an important achievement in treatment options for our Canadian patients, with the potential to reduce the burden of regular prophylactic intravenous factor IX infusion therapy and improve quality of life,” said Dr. Natasha Pardy, President of the Association of Hemophilia Clinic Directors of Canada (AHCDC). “We look forward to being able to offer our patients this innovative treatment option.”

Some key facts about Hemgenix:

• It is a one-time intravenous infusion.
• It was approved by Health Canada in October 2023 for adults with hemophiliaB who require routine prophylaxis to prevent or reduce the frequency of bleeding episodes.
• There have been no reports of serious adverse reactions related to Hemgenix.
• Fifty-four (54) patients enrolled in the HOPE-B phase 3 clinical trial achieved an average of 36.7 percent factor IX activity 24 months after infusion. That level has remained very stable through 48 months.
• Fifty-one (51) of the 54 people enrolled have remained off factor IX prophylaxis through four years.
• Twenty-five (25) of the 54 people have received no factor IX infusions since infusion.
• Overall, people reported improvements in mobility and reductions in joint pain.
• Statistical models predict that 80 percent of people receiving Hemgenix will remain off prophylaxis for 25 years.
• In addition to the participants in the HOPE-B clinical trial, people with hemophiliaB in the U.S. and a number of European countries have been treated with Hemgenix following market authorisations in those countries.

While no provinces or territories have yet to add Hemgenix to their formularies, these decisions may come in the next weeks or months.

To find out more about eligibility for gene therapy, see the booklet All About Hemophilia Gene Therapy, part of the CHS Gene therapy Education Program (www.hemophilia.ca/gene-therapy) or talk to your bleeding disorder treatment team.

Québec
Quebec’s Institut national d’excellence en santé et en services sociaux (INESSS) issued a negative recommendation for Hemgenix in June 2024, pending longer term clinical trial results. Data from 60 months post-infusion will soon be available. It is hoped that this new information will lead to a change in the guidance from INESSS, and that Quebecers with hemophilia B will also have access to gene therapy.

See also:

CSL Behring news release

pan-Canadian Pharmaceutical Alliance announcement