CSL Behring Announces Positive Reimbursement Decision in Canada for HEMGENIX® (etranacogene dezaparvovec), the First Gene Therapy for Hemophilia B

The positive decision from the Canadian Agency for Drugs and Technologies in Health (CADTH) recognizes the importance of this one-time, single dose innovative treatment for adults with hemophilia B who require routine prophylaxis

OTTAWA, ON, July 16, 2024 /CNW/ -- CSL Behring Canada Inc., a business unit of global biotechnology leader CSL, today announced that the Canadian Agency for Drugs and Technologies in Health (CADTH) Canadian Drug Expert Committee (CDEC) recommended public drug plan reimbursement for HEMGENIX^® (etranacogene dezaparvovec) for the treatment of eligible adults with hemophilia B (congenital factor IX deficiency) who require routine prophylaxis to prevent or reduce the frequency of bleeding episodes. The recommendation is based on results from the ongoing Phase III HOPE-B trial, which showed a one-time infusion of HEMGENIX in adult males with hemophilia B elevated factor IX activity levels and significantly reduced the rate of annual bleeds compared to routine factor IX prophylaxis with a favorable safety profile.

"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. David Lillicrap. "Living with hemophilia B requires life-long intravenous infusions of factor IX to maintain adequate levels and reduce the frequency of bleeding events. The potential for gene therapy as an option to help address unmet therapeutic needs is significant progress in this disease area."

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.

"The Canadian Hemophilia Society (CHS) welcomes the recent recommendation by CADTH that HEMGENIX be reimbursed for the treatment of adults with severe or moderately severe hemophilia B," said Emil Wijnker, CHS President. "The CHS is committed to ensuring education and support to allow people with hemophilia B to make informed decisions about their treatment. 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." 

HEMGENIX is the first gene therapy for hemophilia B authorized by Health Canada. HEMGENIX has also been approved by the U.S. Food and Drug Administration, the European Commission (EC) for the European Union and European Economic Area, the United Kingdom's Medicines and Healthcare products Regulatory Agency (MHRA) and Swissmedic in Switzerland and was granted provisional approval in Australia.

"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," said Philippe Hebert, General Manager CSL Behring Canada. "At CSL Behring, we've been successful in working with governments, reimbursement bodies and the healthcare community to shape unique innovative payment models that reflect the value of this innovative therapy and look forward to continuing to collaborate with all stakeholders in Canada to secure public formulary coverage for HEMGENIX as quickly as possible."

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.

About CSL Behring
CSL Behring is a global biotherapeutics leader driven by our promise to save lives. Focused on serving patients' needs by using the latest technologies, we discover, develop and deliver innovative therapies for people living with conditions in the immunology, hematology, cardiovascular and metabolic, respiratory, and transplant therapeutic areas. We use three strategic scientific platforms of plasma fractionation, recombinant protein technology, and cell and gene therapy to support continued innovation and continually refine ways in which products can address unmet medical needs and help patients lead full lives.

CSL Behring operates one of the world's largest plasma collection networks, CSL Plasma. Our parent company, CSL (ASX: CSL; USOTC: CSLLY), headquartered in Melbourne, Australia, employs 32,000 people, and delivers its lifesaving therapies to people in more than 100 countries. For inspiring stories about the promise of biotechnology, visit CSL.com/Vita and follow us on Twitter.com/CSLBehring.

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SOURCE CSL Behring Canada Inc.