COPENHAGEN, DENMARK – October 30, 2024 – Global biotechnology leader CSL Behring (ASX: CSL) today announced that an innovative outcome-based agreement has been reached with Amgros, the Danish procurement authority, for the national reimbursement of HEMGENIX® (etranacogene dezaparvovec) in Denmark. Denmark is the first country in the Nordic region to reimburse HEMGENIX® under such a performance-based model, in which the regions only incur costs as long as the gene therapy proves effective over an agreed long-term period.
This agreement is based on the results of the pivotal HOPE-B trial, with 94% of patients discontinuing Factor IX prophylaxis and remaining prophylaxis-free at 3 years following the one-time infusion.1-3 This agreement recognises the potential long-term durability of the clinical effect of an Advanced Therapy Medicinal Product (ATMP) and means HEMGENIX® will be available to eligible haemophilia B patients in Denmark.
This decision follows the positive recommendation for reimbursement by the Danish Medicines Council in June 2024.4 This key milestone represents the shift away from lifelong intravenous Factor IX infusions, which can significantly impact the quality of life and wellbeing of patients.5
Recognizing the importance of monitoring treatment outcomes, Amgros has established a new digital platform to enable clinicians to report the effectiveness of HEMGENIX®, which is essential for implementing the outcome-based agreement.
“CSL Behring has been working in close collaboration with the Danish Medicines Council and Amgros since the positive recommendation was granted by the Danish Medicines Council in June this year, to develop an innovative agreement suitable for the new Danish ATMP approval process,” said Lise Grove, General Manager Denmark and Iceland, CSL Behring. “Now that we have signed this commercial agreement with Amgros, we are looking forward to seeing the first haemophilia B patients being offered this one-time innovative treatment in Denmark.”
CSL Behring is working with relevant stakeholders to continue the expansion of access to HEMGENIX® across Europe, building on recent milestone access decisions in Spain, UK, Scotland and Austria.6-9
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.
About HEMGENIX®
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®.5 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 2x1013 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 7 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 17th Annual Congress of the European Association for Haemophilia and Allied Disorders (EAHAD) 2024 continue to reinforce the potential long-lasting efficacy and safety of etranacogene dezaparvovec 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.
Media Contacts
Stephanie Fuchs
Mobile: +49 151 58438860
Email: stephanie.fuchs@cslbehring.com
References
- Pipe SW, Leebeek FWG, Recht M, et al. Gene Therapy with Etranacogene Dezaparvovec for Hemophilia B. N Engl J Med 2023; 388: 706-718.
- Pipe S 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(Suppl. 1):25.
- 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. [Last accessed October 2024].
- Medicinrådet. The Medical Council recommends the gene therapy Hemgenix following a new effect-based price agreement. Available at: https://medicinraadet.dk/nyheder/2024/medicinradet-anbefaler-genterapien-hemgenix-efter-ny-effektbaseret-prisaftale. [Last accessed October 2024].
- Srivastava A, Santagostino E, Dougall A, et al. WFH Guidelines for the Management of Hemophilia, 3rd edition. Haemophilia 2020; 26 Suppl 6: 1-158.
- CSL Behring. CSL Behring Signs First Commercial Agreement in Austria to Fund Haemophilia B Gene Therapy HEMGENIX®. Available at: https://www.cslbehring.de/en-us/news/2024/pm-hemgenix-agreement-austria. [Last accessed October 2024].
- National Institute for Health and Care Excellence. Final draft guidance: Etranacogene dezaparvovec for treating moderately severe or severe haemophilia B. Available at: https://www.nice.org.uk/guidance/gid-ta10699/documents/674. [Last accessed October 2024].
- CSL Behring. SMC accepts CSL Behring’s HEMGENIX (etranacogene dezaparvovec) for use on an interim basis in Scotland. Available at: https://www.cslbehring.de/news/2024/pm-hemgenix-scottland. [Last accessed October 2024]. .
- CSL Behring. CSL Behring granted positive reimbursement recommendation for Haemophilia B Gene Therapy, HEMGENIX® in Spain. Available at: https://www.cslbehring.de/en-us/news/2024/pm-hmgenix-reimbursement-spain [Last accessed October 2024].
