CSL Behring's HEMGENIX® Gene Therapy for Hemophilia B Approved in Canada
CSL Behring Canada Inc. has made significant strides in making HEMGENIX®, a groundbreaking one-time gene therapy for adults with hemophilia B, accessible to patients across Canada. Authorized by Health Canada, HEMGENIX® has garnered support from key stakeholders, with discussions ongoing to ensure public coverage.
The Canadian Drug Agency (CDA) recommended public drug plan reimbursement of HEMGENIX® in July 2024, following positive results from the Phase 3 HOPE-B trial. The trial showed that a single infusion of HEMGENIX® resulted in a mean factor IX activity of 36.7 percent at 24 months post-infusion, with 94 percent of patients able to discontinue prophylaxis. CSL Behring Canada Inc. signed a Letter of Intent (LOI) with the pan-Canadian Pharmaceutical Alliance (pCPA) for public reimbursement, allowing provinces and territories outside of Quebec to list HEMGENIX® in their formularies. Marie-Eve Jacques, General Manager of CSL Canada, expressed gratitude for the pCPA's recognition of HEMGENIX®'s potential and eagerness to ensure public coverage. Emil Wijnker, President of the Canadian Hemophilia Society (CHS), encouraged provinces and territories to make HEMGENIX® accessible as soon as possible. Dr. Natasha Pardy, President of the Association of Hemophilia Clinic Directors of Canada (AHCDC), welcomed the availability of gene therapy for hemophilia B as an important achievement in treatment options.
HEMGENIX®, the first one-time gene therapy authorized for treating adults with hemophilia B in Canada, is poised to transform the lives of those who require routine prophylaxis. With the CDA's recommendation, the LOI signed with the pCPA, and the support of key stakeholders, HEMGENIX® is on track to become widely accessible through public drug plans, offering a new hope for patients with hemophilia B.
