CADTH recommends hemophilia B gene therapy reimbursement
Canadian panel's Hemgenix recommendation comes with conditions
The Canadian Agency for Drugs and Technologies in Health’s (CADTH) Canadian Drug Expert Committee (CDEC) recommended reimbursement for hemophilia B gene therapy Hemgenix (etranacogene dezaparvovec), according to the therapy’s developer, CSL Behring Canada.
Hemgenix in 2023 became the first gene therapy in the country approved for hemophilia B when the federal regulatory agency Health Canada approved it to treat adults 18 and older with hemophilia B who need routine prophylaxis to prevent or reduce the frequency of bleeding episodes.
The CDEC said its recommendation to make Hemgenix eligible for reimbursement by public drug programs for eligible patients with severe or moderately severe hemophilia B comes with conditions, including that its cost be reduced and that it be prescribed by “specialists and experts in treating hemophilia B.”
“We thank CADTH for their quick review and positive reimbursement recommendation, which signifies an important step forward in facilitating access of Hemgenix to eligible people with hemophilia B in Canada,” Philippe Hebert, general manager at CSL Behring Canada, said in a company press release. The company plans “to collaborate with all stakeholders in Canada to secure public formulary coverage for Hemgenix as quickly as possible,” Hebert said.
The decision was supported by results from the ongoing Phase 3 clinical trial, HOPE-B (NCT03569891), showing that a single infusion of Hemgenix increased factor IX activity levels and significantly reduced the annual rate of bleeding episodes in adult male patients. Factor IX is a protein that is necessary for proper blood clotting, leaving these patients vulnerable to spontaneous bleeding and other characteristic symptoms.
Testing required for treatment
People with hemophilia B have insufficient factor IX in their blood, due to mutations in the F9 gene.
The main source of factor IX is the liver. Hemgenix uses a virus, which is harmless to humans, to insert a functional copy of the F9 gene into liver cells. This therapeutic strategy is called gene replacement therapy, and with these new instructions, the liver produces enough factor IX to restore proper blood clotting and prevent bleeding.
Patients must undergo prior testing to ensure their immune system will not inhibit the function of the virus or the factor IX itself. These tests are included as eligibility criteria for reimbursement in the official CADTH Reimbursement Recommendation.
“We recognize that the CADTH recommendation comes with certain conditions, and we urge all parties to work together so that this therapy, which has the potential to fundamentally transform treatment and improve quality of life, becomes available across Canada as soon as possible,” said Emil Wijnker, president of the Canadian Hemophilia Society.
Hemgenix is given an infusion into the bloodstream. A one-time, single-dose treatment, it has the potential to reduce or even eliminate the need for regular treatment with regular factor IX replacement therapies that provide a functional version of the protein.
“The reimbursement recommendation from CADTH for Hemgenix brings us closer to having access to this therapy for our patients,” said David Lillicrap, MD, a professor in Queen’s University’s department of pathology and molecular medicine. “Living with hemophilia B requires life-long intravenous infusions of factor IX to maintain adequate levels and reduce the frequency of bleeding events,” Lillicrap said. “The potential for gene therapy as an option to help address unmet therapeutic needs is significant progress in this disease area.”
Hemgenix is also approved in the United States, the European Union, and Switzerland, and has provisional approval in Australia. In France, two patients recently received the gene therapy at hemophilia treatment centers.
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