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CSL Behring Signs First Commercial Agreement in Austria to Fund Haemophilia B Gene Therapy HEMGENIX®


MARBURG, GERMANY – March 4, 2024 – Global biotechnology leader CSL Behring (ASX: CSL) today announced that a commercial contract has been signed to fund HEMGENIX® (etranacogene dezaparvovec) in Austria, with the option to access a dedicated fund, established by the provinces for specialised medicines. This is the first commercial agreement in Austria for HEMGENIX® and will enable access to the gene therapy for haemophilia B patients.

HEMGENIX® is the first and only one-time gene therapy approved in Austria for the treatment of severe and moderately severe haemophilia B (congenital factor IX deficiency) in adult patients without a history of factor IX inhibitors.1

This pioneering funding solution plays an important role in the broader paradigm shift being seen in the treatment of haemophilia B. Expanded access to HEMGENIX® will continue to progress the move away from lifelong intravenous factor IX infusions, which can significantly impact the quality of life and wellbeing of patients.2

“Over the past two years we have worked diligently with all relevant stakeholders to enable access to HEMGENIX®,” said Dr Beate Pettinger-Natmeßnig, General Manager Commercial Operations Austria, CSL Behring. “The agreement signed for HEMGENIX® demonstrates the Austrian healthcare system's commitment to provide access to gene therapy and to break new ground in the financing of advanced therapy medicinal products."

With this agreement in place, eligible patients in Austria with severe and moderately severe haemophilia B can be treated with HEMGENIX® at the Haemophilia Comprehensive Care Centre at the Medical University of Vienna, the first HUB-centre in the country. 

“It is hugely encouraging to see pioneering funding solutions being developed and realised across Europe,” said Dr Lutz Bonacker, SVP and General Manager Commercial Operations Europe, CSL Behring. “The new commercial agreement in Austria is a strong signal to how innovative and collaborative thinking can bring a transformative impact for patients and their loved ones, for whom there is still significant unmet need in haemophilia B.”

About Haemophilia B

Haemophilia 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. Current treatments for moderate to severe haemophilia B include life-long prophylactic infusions of factor IX to temporarily replace or supplement low levels of the blood-clotting factor.  


HEMGENIX® is a gene therapy that reduces the rate of abnormal bleeding in eligible people with haemophilia B by enabling the body to continuously produce factor IX, the deficient protein in haemophilia 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. 

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 HEMGENIX®. Fifty-four adult haemophilia B patients classified as having moderately severe to severe haemophilia B and requiring prophylactic factor IX replacement therapy were enrolled in a prospective, six-month or longer observational period during which time they continued to use their current standard of care therapy to establish a baseline Annual Bleeding Rate (ABR). After the six-month lead-in period, patients received a single intravenous administration of HEMGENIX® at the 2x10^13 gc/kg dose. Patients were not excluded from the trial based on pre-existing neutralizing antibodies (NAbs) to AAV5.

A total of 54 patients received a single dose of HEMGENIX® in the pivotal trial, with 52 patients completing at least three years of follow-up. The primary endpoint in the pivotal HOPE-B study was ABR 52 weeks after achievement of stable factor IX expression (months 7 to 18) compared with the six-month lead-in period. For this endpoint, ABR was measured from month seven to month 18 after infusion, ensuring the observation period represented a steady-state factor IX transgene expression. Secondary endpoints included assessment of factor IX activity.

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. A serious adverse event of hepatocellular carcinoma was determined to be unrelated to treatment with HEMGENIX® by independent molecular tumour characterization and vector integration analysis. No inhibitors to factor IX were reported. 

Long-term three-year data presented at the 65th American Society of Hematology (ASH) 2023 Annual Meeting and Exposition continue to reinforce the potential long-lasting efficacy and safety of HEMGENIX® and the ongoing benefit of this treatment for people living with haemophilia B.

About CSL

CSL (ASX:CSL; USOTC:CSLLY) is a global biotechnology company with a dynamic portfolio of lifesaving medicines, including those that treat haemophilia and immune deficiencies, vaccines to prevent influenza, and therapies in iron deficiency 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 32,000 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 CSL.com.

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Media Contacts
Stephanie Fuchs
Mobile: +49 151 584 388 60
Email: Stephanie.Fuchs@cslbehring.com


1 European Medicines Agency. First Gene therapy to treat haemophilia B. Available at: https://www.ema.europa.eu/en/news/first-gene-therapy-treat-haemophilia-b. [Accessed March 2024].
2 Srivastava A et al. WFH Guidelines for the Management of Hemophilia, 3rd edition. Haemophilia 2020; 26(Suppl 6):1-158.