CSL Behring Signs Letter of Intent with pan-Canadian Pharmaceutical Alliance (pCPA) for Public Reimbursement of HEMGENIX® (etranacogene dezaparvovec), the First Gene Therapy for Hemophilia B

OTTAWA, Ontario, Oct. 6, 2025 /CNW/ -- CSL Behring Canada Inc., a business unit of global biopharma leader CSL, today announced that it has signed a Letter of Intent (LOI) with the pan-Canadian Pharmaceutical Alliance (pCPA) for the public reimbursement of HEMGENIX^® (etranacogene dezaparvovec). HEMGENIX is the first one-time gene therapy authorized by Health Canada for the treatment of adults with hemophilia B who require routine prophylaxis to prevent or reduce the frequency of bleeding episodes. The successful completion of the LOI agreement allows provinces and territories outside of Quebec to proceed with listing HEMGENIX as part of their formularies. CSL Behring remains in discussions with the province of Quebec to secure public reimbursement for HEMGENIX.

"We sincerely appreciate the pCPA for acknowledging the transformative potential of HEMGENIX for appropriate adults with hemophilia B," said Marie-Eve Jacques, General Manager, CSL Canada. "This milestone represents a significant advancement in making this revolutionary treatment accessible in Canada. We are eager to work with provinces and territories to ensure public coverage for HEMGENIX as quickly as possible."

In July 2024, the Canadian Drug Agency (CDA) recommended public drug plan reimbursement of HEMGENIX based on the results of the Phase 3 HOPE-B trial. The trial demonstrated that a one-time infusion of HEMGENIX in adult males with severe or moderately severe hemophilia B, with or without pre-existing AAV5 neutralizing antibodies, produced mean factor IX activity of 36.7 percent at 24 months post infusion.¹ In addition, 94 percent (51 out of 54) of patients treated with HEMGENIX discontinued use of prophylaxis and remained free of previous continuous routine prophylaxis therapy. Of the adverse events reported based on phase 2b and phase 3 trial (Hope-B), most frequently reported adverse drug reactions were ALT elevations, headache, influenza-like illness and AST elevations. The most common adverse reactions were infusion-related reactions.

"The availability of gene therapy for hemophilia B represents an important achievement in treatment options for our Canadian patients, with the potential to reduce the burden of regular prophylactic intravenous factor IX infusion therapy and improve quality of life," said Dr. Natasha Pardy, President of the Association of Hemophilia Clinic Directors of Canada (AHCDC). "We look forward to being able to offer our patients this innovative treatment option."

HEMGENIX allows people living with hemophilia B to produce their own factor IX, which can lower the risk of bleeding.

"Having treatment options that can meet the individual needs of people living with hemophilia B is important," said Emil Wijnker, President of the Canadian Hemophilia Society (CHS). "The Letter of Intent for reimbursement of HEMGENIX is an encouraging step forward, and we urge the provinces and territories to work together, and with CSL Behring, to make this therapy accessible as soon as possible to those who need it."

About Hemophilia B
Hemophilia B is a life-threatening rare disease. People with the condition are particularly vulnerable to bleeds in their joints, muscles, and internal organs, leading to pain, swelling, and joint damage.¹ The constant worry of a bleed means that their daily activities can be restricted, even for things as simple as going up and down stairs.^1-3 Current treatments for moderate to severe hemophilia B include life-long prophylactic infusions of Factor IX to temporarily replace or supplement low levels of the blood-clotting factor.¹ Many people with hemophilia find themselves continually confronted with the mental and emotional impact of managing their condition, and rarely have their minds free of hemophilia.

About HEMGENIX^®
HEMGENIX^® (etranacogene dezaparvovec) is an in vivo 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 protein that is deficient in people with the disease. It uses a non-infectious viral vector derived from an adeno-associated virus (AAV5). The AAV5 vector carries the Padua gene variant of Factor IX to the target cells in the liver, generating Factor IX proteins that are 5–8x 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.

About the Pivotal HOPE-B Trial 
The pivotal Phase III HOPE-B trial is an ongoing, multinational, open-label, single-arm study to evaluate the safety and efficacy of etranacogene dezaparvovec. A total of 54 adult patients with hemophilia B, classified as having moderately severe to severe hemophilia B and requiring prophylactic Factor IX replacement therapy, were enrolled in a prospective, 6-month or longer observational period. During this period, patients continued to use their current standard of care therapy to establish a baseline annual bleeding rate (ABR). After the 6-month lead-in period, patients received a single intravenous administration of etranacogene dezaparvovec at the 2x10¹³ gc/kg dose. Patients were not excluded from the trial based on pre-existing neutralizing antibodies (NAbs) to AAV5.²

The results of the Phase III HOPE-B trial demonstrated the long-lasting efficacy and safety of etranacogene dezaparvovec as well as the ongoing benefit of this treatment for people living with hemophilia B, with long-term bleed protection provided by a one-time infusion. A total of 94% of patients (51/54) discontinued routine Factor IX prophylaxis and remained prophylaxis-free at 4 years post-treatment. Etranacogene dezaparvovec demonstrated mean Factor IX activity levels of 37.4% (n=47), which were sustained at near normal levels at 4 years post-treatment. Mean adjusted annualized bleeding rate (ABR) for all bleeds was reduced vs lead-in (4.16, n=54) as compared to year four (0.40, n=51). The results also showed that etranacogene dezaparvovec is clinically effective in eligible patients with pre-existing AAV5 NAbs (up to a NAb titer of 1:898 or equivalent).³

Etranacogene dezaparvovec was generally well-tolerated, with a total of 96 treatment-related adverse events (AEs) 4 years post-infusion, 92 (96%) of which occurred in the first six months post-treatment. No serious treatment-related adverse reactions were reported. One death resulting from urosepsis and cardiogenic shock in a 77-year-old patient at 65 weeks following dosing was considered unrelated to treatment by investigators and the company sponsor. Three previously reported serious adverse events (hepatocellular carcinoma, schwannoma and myelodysplastic syndrome) were determined to be unrelated to treatment with etranacogene dezaparvovec by independent molecular tumor characterization and vector integration analysis. No inhibitors to Factor IX were reported.³

Important Information for Canada
HEMGENIX is indicated for the 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%).

Consult the product monograph available here for contraindications, warnings, precautions, adverse reactions, interactions, dosing, and conditions of clinical use. The product monograph is also available through our medical department. Call 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 donation networks, CSL Plasma. Our parent company, CSL (ASX:CSL; USOTC:CSLLY), headquartered in Melbourne, Australia, employs 29,000+ 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. 

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References

¹ Pipe SW, Leebeek FWG, Recht M, et al. Gene Therapy with Etranacogene Dezaparvovec for Hemophilia B. N Engl J Med 2023; 388: 706-718.
² Pipe S, van der Valk P, Verhamme P, et al. Long-Term bleeding protection, sustained FIX activity, reduction of FIX consumption and safety of hemophilia B gene therapy: results from the HOPE-B Trial 3 years after administration of a single dose of etranacogene dezaparvovec in adult patients with severe or moderately severe hemophilia B. Blood 2023; 142: 1055.
³ Leebeek FWG, von Drygalski A, et al. The phase 3 HOPE-B trial shows 4-year durability of sustained near-normal FIX activity, bleed protection and favourable safety in adults with severe or moderately severe haemophilia B. EAHAD 2025. EAHAD25-ABS-1255.

SOURCE CSL Behring