CSL Canada Announces First Canadian Patient Receives HEMGENIX® (etranacogene dezaparvovec) Hemophilia B Gene Therapy in Ontario, Canada

• CSL Canada is proud to recognize this important milestone of the first Canadian patient to receive HEMGENIX^® (etranacogene dezaparvovec), the one-time gene therapy treatment for eligible patients living with hemophilia B, at London Health Sciences Centre in London, Ontario.
• Following a successful pan-Canadian Pharmaceutical Alliance (pCPA) agreement, HEMGENIX^® is now publicly reimbursed in Ontario and British Columbia via the Exceptional Access Program, with additional provinces under consideration.
• It is indicated for the treatment of adults (aged 18 years of age or older) with severe to moderately severe hemophilia B (congenital Factor IX deficiency) who require routine prophylaxis to prevent or reduce the frequency of bleeding episodes.¹ There is no clinical experience of HEMGENIX^® use in patients with mild or moderate hemophilia B (FIX activity > 2%).
• This milestone marks the first HEMGENIX^® treatment administered to a Canadian patient outside of a clinical trial, demonstrating real‑world access to gene therapy in Canada.   

OTTAWA, ON, May 20, 2026 /CNW/ - CSL Canada is proud to announce the first Canadian patient has received HEMGENIX^® (etranacogene dezaparvovec), a one-time hemophilia B gene therapy, at London Health Sciences Centre (LHSC). This milestone marks a significant advancement in hemophilia B care, reflecting how clinical innovation and health system collaboration are enabling real-world access to gene therapy for eligible patients in Canada.

Hemophilia B is a rare genetic bleeding disorder caused by a deficiency of clotting Factor IX, which prevents blood from clotting properly, often resulting in a lifelong dependence on medication to manage bleeding.² People living with hemophilia B may experience spontaneous or prolonged bleeding episodes that can lead to joint damage, chronic pain, reduced mobility, and other long-term complications that affect quality of life.³

For many patients and families, hemophilia B often means navigating constant vigilance around physical activity, injury risk, and disease management affecting all facets of everyday life. In Canada, hemophilia B affects approximately one in 50,000 people,⁴ typically requiring lifelong management through medical monitoring, specialized multidisciplinary care and sustained healthcare system resources through hemophilia treatment centres.⁵

HEMGENIX^® is a gene therapy addressing the underlying cause of hemophilia B by enabling the body to produce Factor IX on its own. This one-time treatment option, for patients who otherwise require ongoing replacement therapy, offers eligible adults the potential to reduce the routine burden of care and improve long-term disease management. Following a successful pan-Canadian Pharmaceutical Alliance (pCPA) agreement, HEMGENIX^® is now publicly reimbursed in Ontario and British Columbia.

"The first treatment with HEMGENIX^® in Canada reflects the tremendous importance of expanding treatment options and access to hemophilia B patients," said Marie-Ève Jacques, General Manager, CSL Canada.    

"This is a meaningful moment for the bleeding disorders community in Canada," said Emil Wijnker, President of the Canadian Hemophilia Society. "For many, hemophilia B is a constant consideration in everyday life. Advances like gene therapy signal real progress, with the potential to ease that burden and meaningfully change how people manage their condition over time."

"The first HEMGENIX^® treatment in Canada marks an important step forward in how we care for people living with hemophilia B," said Dr. Chai Phua, MD, FRCPC, Medical Director of the Adult Bleeding Disorders Program at London Health Sciences Centre. "Gene therapy introduces a new approach to treatment, and delivering it requires close coordination across clinical teams and systems. It's encouraging to see this level of readiness translating into real-world care for eligible patients."

CSL Canada continues to advance innovation and is working to help ensure all eligible Canadians can access and benefit from advances in hemophilia care and innovation.

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.⁶ 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.^6-8 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.⁶ 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.⁷

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.⁹ It uses a non-infectious viral vector derived from an adeno-associated virus (AAV5).⁹ The AAV5 vector carries the Padua gene variant of Factor IX to the target cells in the liver, generating Factor IX proteins that are 5x–8x more active than normal.¹⁰ These genetic instructions remain in the target cells, but generally do not become a part of a person's own DNA.⁹ Once delivered, the new genetic instructions allow the cellular machinery to produce stable levels of Factor IX.¹¹

About the Pivotal HOPE-B Trial
The pivotal Phase III HOPE-B trial was a multinational, open-label, single-arm study to evaluate the safety and efficacy of HEMGENIX^®.¹² 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). ¹² After the 6-month lead-in period, patients received a single intravenous administration of HEMGENIX^® at the 2x10¹³ gc/kg dose. Patients were not excluded from the trial based on pre-existing neutralizing antibodies (NAbs) to AAV5. ¹²

Additionally, new HOPE-B five-year data published in the New England Journal of Medicine and presented simultaneously at the American Society of Hematology (ASH) Annual Meeting 2025, confirmed the five-year durability and safety of HEMGENIX^®.^13,14

HEMGENIX^® was generally well-tolerated, with a total of 100 treatment-related adverse events (TRAEs) 5 years post-infusion, most of which occurred in the first six months post-treatment. ^13,14 No serious treatment-related adverse reactions were reported. ^9,15 Two deaths occurred during the trial due to non-treatment-related AEs: one at approximately 15 months post-dose due to cardiogenic shock and urosepsis, and another at approximately 54 months post-dose due to cardiac amyloidosis. Three previously reported serious adverse events (hepatocellular carcinoma, schwannoma and myelodysplastic syndrome) were determined to be unrelated to treatment with HEMGENIX^® by independent molecular tumour characterization and vector integration analysis.^14,16 No inhibitors to Factor IX were reported.^9,14

The five-year data marks the final analysis for the HOPE-B study, but participants who consent will continue to be monitored in the IX-TEND 222-3003 extended follow-up study (NCT05962398), which will track patients for up to 15 years post-treatment.^13,14

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
CSL (ASX:CSL; USOTC:CSLLY) is a leading global biopharma company with a dynamic portfolio of lifesaving medicines, including those that treat hemophilia and immune deficiencies, vaccines to prevent influenza, and therapies in iron deficiency, dialysis and nephrology. Since our start in 1916, we have been driven by our promise to save lives using the latest technologies. Today, CSL – including our three businesses, CSL Behring, CSL Seqirus and CSL Vifor – provides lifesaving products to patients in more than 100 countries and employs 29,000+ people. Our unique combination of commercial strength, R&D focus and operational excellence enables us to identify, develop and deliver innovations so our patients can live life to the fullest. For inspiring stories about the promise of biotechnology, visit CSL.com/Vita.

For more information about CSL, visit https://www.cslbehring.ca/

References

	1.	Product Monograph. Hemgenix-Product-Monograph.pdf
	2.	Harvard Health. Hemophilia (2025). https://www.health.harvard.edu/diseases-and-conditions/hemophilia-a-to-z. Last accessed February 18, 2026.
	3.	Konkle BA, et al. Hemophilia B. In: Adam MP, Mirzaa GM, Pagon RA, et al., editors. GeneReviews®. Seattle (WA): University of Washington, Seattle; 1993–2024. Available from: https://www.ncbi.nlm.nih.gov/books/NBK1495/ Accessed March 6, 2026.
	4.	Bleeding Disorders Canada. Hemophilia A and B. Montreal (QC): Bleeding Disorders Canada; 2024. https://www.hemophilia.ca/hemophilia-a-and-b/ Accessed March 6, 2026.
	5.	Hoffmann P, et al. Overview of gene therapy for hemophilia: questions and answers to navigate the innovation. Journal of Thrombosis and Haemostasis. 2025. https://www.sciencedirect.com/science/article/pii/S1538783625009158 Last accessed March 6, 2026.
	6.	Srivastava A, Santagostino E, Dougall A, et al. WFH Guidelines for the Management of Hemophilia, 3rd edition. Haemophilia 2020; 26 Suppl 6: 1-158.
	7.	Krumb E, Hermans C. Living with a "hemophilia-free mind" - The new ambition of hemophilia care? Res Pract Thromb Haemost 2021; 5: e12567.
	8.	Hermans C, Pierce GF. Towards achieving a haemophilia-free mind. Haemophilia 2023; 29: 951-953.
	9.	Pipe SW, Leebeek FWG, Recht M, et al. Gene Therapy with Etranacogene Dezaparvovec for Hemophilia B. N Engl J Med 2023; 388: 706-718.
	10.	Spronck EA, Liu YP, Lubelski J, et al. Enhanced Factor IX Activity following Administration of AAV5-R338L "Padua" Factor IX versus AAV5 WT Human Factor IX in NHPs. Mol Ther Methods Clin Dev 2019; 15: 221-231.
	11.	Thornburg CD. Etranacogene dezaparvovec for hemophilia B gene therapy. Ther Adv Rare Dis 2021; 2: 26330040211058896.
	12.	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.
	13.	Pipe, S, Miesback W, Recht, M, et al. End-of-study analysis of the HOPE-B Trial confirms the durable efficacy and safety of etranacogene dezaparvovec haemophilia b gene therapy over 5 years. ASH 2025. Abstract 25-4052.
	14.	Pipe SW, Miesbach W, Recht M, et al. Final Analysis of a Study of Etranacogene Dezaparvovec for Hemophilia B. New England Journal of Medicine.
	15.	Genezen MA, Inc. CSL Behring GmbH. Hemgenix^® (etranacogene dezaparvovec): Summary of Product Characteristics [online]. Available at: https://www.ema.europa.eu/en/documents/product-information/hemgenix-epar-product-information_en.pdf [Last accessed: February 2026].
	16.	Pipe SW, van der Valk P, Verhamme P, et al. Etranacogene dezaparvovec shows sustained efficacy and safety in adult patients with severe or moderately severe haemophilia B 3 years after administration in the hope-B Trial [EAHAD 2024 Oral Abstract OR09]. Haemophilia. 2024; 30: 25.

SOURCE CSL Canada

Media Contact: Victoria Bliss, Zeno Group (on behalf of CSL Canada), [email protected], + 1 647 333 5178