The positive decision from the
"Decades of intensive research around the world led to the development of this gene therapy for hemophilia B and the reimbursement recommendation from CADTH for HEMGENIX brings us closer to having access to this therapy for our patients," said leading Canadian hematologist, Dr.
Hemophilia B is a rare, lifelong bleeding disorder caused by a single gene defect, resulting in insufficient production of factor IX, a protein primarily produced by the liver that helps blood clots form. Treatments for moderate to severe hemophilia B include prophylactic infusions of factor IX replacement therapy to temporarily replace or supplement low levels of blood-clotting factor and, while these therapies are effective, those with hemophilia B must adhere to strict, lifelong infusion schedules. They may also still experience spontaneous bleeding episodes as well as limited mobility, joint damage or severe pain as a result of the disease. For appropriate patients, HEMGENIX allows people living with hemophilia B to produce their own factor IX, which can lower the risk of bleeding and reduce or eliminate the need for regular, scheduled factor IX replacement therapy.
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HEMGENIX is the first gene therapy for hemophilia B authorized by
"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
Full details on reimbursement recommendations for HEMGENIX including clinical criteria and conditions, are available on the CADTH website.
About Hemophilia B
Hemophilia B is a potentially life-threatening rare disease caused by a mutation on the F9 gene, resulting in the absence or deficiency of levels of factor IX (FIX), a protein produced primarily by the liver that helps the blood clot. People with hemophilia B are particularly vulnerable to bleeds in their joints, muscles, and internal organs, leading to pain, swelling, and joint damage. Current treatments for moderate to severe hemophilia B include recurrent prophylactic infusions of FIX to temporarily replace or supplement low levels of the blood-clotting factor.
About HEMGENIX®
HEMGENIX® is a gene therapy that reduces the rate of abnormal bleeding in eligible people with hemophilia B by enabling the body to continuously produce factor IX, the deficient protein in hemophilia B. It uses AAV5, a non-infectious viral vector, called an adeno-associated virus (AAV). The AAV5 vector carries the Padua gene variant of Factor IX (FIX-Padua) to the target cells in the liver, generating factor IX proteins that are 5x-8x more active than normal. These genetic instructions remain in the target cells, but generally do not become a part of a person's own DNA. Once delivered, the new genetic instructions allow the cellular machinery to produce stable levels of factor IX.
Important Information for Canada
Indications and clinical use:
HEMGENIX (etranacogene dezaparvovec) is indicated for treatment of adults (aged 18 years of age or older) with Hemophilia B (congenital Factor IX deficiency) who require routine prophylaxis to prevent or reduce the frequency of bleeding episodes.
There is no clinical experience of HEMGENIX use in patients with mild or moderate Hemophilia B (FIX activity > 2%).
Contraindications:
HEMGENIX is contraindicated in patients who are hypersensitive to this drug or to any ingredient in the formulation, including any non-medicinal ingredient, or component of the container.
Warnings and precautions:
- Shedding of HEMGENIX vector DNA
- Donation of blood, organs, tissues or cells
- Driving and operating machinery
- Thromboembolic events
- Hepatotoxicity
- Hepatocellular carcinogenicity
- Immune-mediated neutralization of the AAV5 vector capsid
- Infusion-related reactions
- Monitoring and laboratory tests
- Fertility
Other relevant warnings and precautions:
The most frequently reported adverse drug reactions in clinical studies related to HEMGENIX were ALT elevations (10/57 patients [17.5%]), headache (9/57 patients [15.8%]), influenza-like illness (8/57 patients [14%]) and AST elevations (5/57 patients [8.8%]).
For more information:
For more information and a complete risk/benefit profile, please refer to the Product Monograph available here. The product monograph is also available by calling us at 1-866-773-7721.
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