CSL Canada has reported that the first patient in Canada has received Hemgenix (etranacogene dezaparvovec), a one-time gene therapy for hemophilia B, at London Health Sciences Centre in Ontario. The treatment follows a pan-Canadian Pharmaceutical Alliance agreement, with Hemgenix now publicly reimbursed in Ontario and British Columbia for eligible adults who would otherwise require ongoing replacement therapy.
Hemophilia B is a rare genetic bleeding disorder caused by a deficiency of clotting Factor IX, often requiring lifelong management with regular medication, monitoring, and specialized multidisciplinary care through hemophilia treatment centers. People with hemophilia B can experience spontaneous or prolonged bleeding episodes that may lead to joint damage, chronic pain, reduced mobility, and other long-term complications, affecting daily activities and quality of life. In Canada, the condition affects an estimated one in 50,000 people.
Hemgenix is designed to address the underlying cause of hemophilia B by enabling the body to produce Factor IX endogenously. According to CSL Canada, clinicians, patient organizations, and treatment centers view the first administration of Hemgenix as an important step in expanding available treatment options and incorporating gene therapy into hemophilia care. Physicians involved in the first treatment noted that delivering gene therapy requires coordination across clinical teams and systems and indicated that this readiness is now beginning to translate into routine care for eligible patients.
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