First Patient Treated with HEMGENIX® (etranacogene dezaparvovec) Gene Therapy for Haemophilia B in Germany

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Marburg, Germany – 1 July 2025 - CSL Behring Germany today announced that the first haemophilia B patient in Germany was treated yesterday with the gene therapy HEMGENIX® (etranacogene dezaparvovec) at the Hemophilia Treatment Center at Hannover Medical School.

HEMGENIX® is the first one-time gene therapy approved in Europe for the treatment of adults with severe and moderately severe haemophilia B, an inherited bleeding disorder caused by the lack of Factor IX (a protein needed to produce blood clots to stop bleeding). It is used in adults without a history of Factor IX inhibitors.1

"Each advancement in hemophilia treatment, particularly through innovative therapies like gene therapy, brings us closer to a better and more carefree life," explains Christian Schepperle, Managing Director of the Interessengemeinschaft Hämophiler e.V. (IGH) in Germany. "Our objectives in hemophilia treatment align closely with economic considerations. Advanced therapies are not only medically essential but also make socio-economic sense by helping to limit long-term healthcare costs ‒ thus making us more economically and geopolitically independent," Schepperle continues.

HEMGENIX® is now available to patients in Germany under an innovative, success-based reimbursement model being implemented for the first time in the country.

"The treatment of the first patient in Germany with HEMGENIX® marks a milestone in hemophilia B therapy. We are proud to offer a gene therapy that not only provides a long-term solution for patients but also sets new standards for outcome-based reimbursement models. The agreement with the GVK-Spitzenverband addresses critical challenges such as the long-term efficacy of this one-time therapy and ensures that reimbursement is tied to the individual treatment success of each patient. Additionally, it guarantees that the treatment costs for HEMGENIX® remain cost-neutral for statutory health insurance (GKV)," explains Christian Wieszner, Managing Director of CSL Behring Germany.

HEMGENIX® was granted conditional marketing authorisation by the European Commission (EC) for the European Union and European Economic Area in February 2023, following approval from the U.S. Food and Drug Administration (FDA) in November 2022. HEMGENIX® has also received regulatory approval in Canada, the UK, Switzerland, Australia, Saudi Arabia, Taiwan, South Korea and Hong Kong.

Following these approvals, CSL Behring is working with relevant stakeholders to ensure eligible patients have access to HEMGENIX® across Europe and beyond, building on milestone access decisions in Germany, Denmark, Switzerland, Spain, the UK (including Scotland), and Austria.2-7 Patients in France, Denmark, Austria, Spain and the UK have already experienced treatment with HEMGENIX®.

Additionally, new four-year data from the HOPE-B study presented at the Annual Congress of the European Association for Haemophilia and Allied Disorders 2025 showed that a one-time infusion of HEMGENIX® continues to offer long-term durability and safety.8

“CSL Behring is dedicated to providing opportunities for people with rare genetic bleeding disorders to better manage the impact of their condition on their daily lives. Treatments such as gene therapy have the potential to enable people living with haemophilia B to move towards a haemophilia-free mind” concludes Christian Wieszner, Managing Director of CSL Behring Germany.

 

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.9 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.9-11 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.9 Many people with haemophilia find themselves continually confronted with the mental and emotional impact of managing their condition, and rarely have their minds free of haemophilia.10

 

About HEMGENIX®

HEMGENIX® (etranacogene dezaparvovec) is an in vivo 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 protein that is deficient in people with the disease.12 It uses a non-infectious viral vector derived from an adeno-associated virus (AAV5).12 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.13 These genetic instructions remain in the target cells, but generally do not become a part of a person’s own DNA.12 Once delivered, the new genetic instructions allow the cellular machinery to produce stable levels of Factor IX.14

 

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.15 A total of 54 adult patients with haemophilia B, classified as having moderately severe to severe haemophilia 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).15 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 neutralising antibodies (NAbs) to AAV5.15

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 haemophilia B, with long-term bleed protection provided by a one-time infusion.1,8,12 A total of 94% of patients (51/54) discontinued routine Factor IX prophylaxis and remained prophylaxis-free at 4 years post-treatment.8 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.8 Mean adjusted annualised bleeding rate (ABR) for all bleeds was reduced vs lead-in of Factor IX prophylaxis (4.16, n=54) as compared to year four (0.40, n=51).8 The results also showed that etranacogene dezaparvovec is clinically effective in eligible patients with pre-existing AAV5 NAbs (up to a NAb titre of 1:898 or equivalent).1,8,12,16,17

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.8 No serious treatment-related adverse reactions were reported.1,8,12 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.17 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 tumour characterisation and vector integration analysis.17 No inhibitors to Factor IX were reported.8

 

Additional trial data

CSL220 (formerly AMT-060) has a similar structure to etranacogene dezaparvovec, differing only in a single amino acid substitution in the F9 gene.18 In a Phase I/II study, the durability of CSL220 was assessed in two cohorts of adult patients with severe/moderately severe haemophilia B receiving different single-infusion doses.18 Mean Factor IX activity remained stable in both cohorts at 8 years (Cohort 1: n=3, Year 8: 4.9 IU/dL; Cohort 2: n=5, Year 8: 5.6 IU/dL), mean ABR was maintained at Year 8 (Cohort 1: 2.2; Cohort 2: 1.0) with no new safety events identified were identified during Year 8 and no patients returning to continuous Factor IX prophylaxis.18

In a Phase IIb study of adults (n=3) with haemophilia B receiving a single dose of etranacogene dezaparvovec, mean aPTT-based actor IX activity remained stable from Year 1 (40.7%) up to Year 5 (46.7%) and mean ABR for the cumulative follow-up was 0.14 (Years 0-5) for all bleeds. A favourable safety profile was maintained over 5 years.19 Despite the limited population size, these data may provide further evidence of the long-term effects of etranacogene dezaparvovec.18,19

 

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

 

References:
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  1. 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: June 2025].
  2. CSL Behring. CSL Behring and GKV-Spitzenverband Agree on Reimbursement Price for HEMGENIX® – Europe's First Gene Therapy for Haemophilia B. Available at: https://www.cslbehring.de/en-us/news/2025/pm-gkv-hemgenix. [Last accessed June 2025].
  3. 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 June 2025].
  4. Ministerio De Sanidad. Puntos destacados de la reunión de la Comisión Interministerial de Precios de los Medicamentos 26 de septiembre de 2024. Available at: https://www.sanidad.gob.es/areas/farmacia/precios/comisionInteministerial/acuerdosNotasInformativas/docs/NOTAINFORMATIVACIPM_SEPTIEMBRE2024.pdf [Last Accessed: June 2025].
  5. 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 June 2025].
  6. Scottish Medicines Consortium. Etranacogene dezaparvovec (Hemgenix). Available at: https://scottishmedicines.org.uk/medicines-advice/etranacogene-dezaparvovec-hemgenix-full-smc2649/. [Last accessed June 2025].
  7. 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 June 2025].
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  15. 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.
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  19. von Drygalski A, Giermasz A, Gomez E, et al. OC 02.3 Etranacogene dezaparvovec hemophilia B gene therapy phase 2b trial final Results: stable and durable FIX level expression over 5 years. ISTH 2024 Oral Communication Abstracts. Research and Practice in Thrombosis and Haemostasis 2024; 8: 116.

 

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