REDEFINING HOW YOU LIVE WITH HAEMOPHILIA B

INTRODUCING HEMGENIX®

The goal of HEMGENIX® is to offer protection from bleeds for people with haemophilia B, without the need for regular FIX (factor IX) infusions.1,2

While there have been advances in haemophilia B treatment, the usual treatment pathway is lifelong infusions of FIX replacement therapy.2

The aim of FIX prophylaxis is to reduce bleeding events by increasing FIX levels. However, the levels of plasma FIX can go up and down, which may put the patient at higher risk of breakthrough bleeds.3

Recurring spontaneous bleeding into the joints is the most common bleeding type, and can lead to:4–6
 

Icon joint damage

Joint damage

Icon pain

Pain

Icon reduced quality of life

Reduced quality of life

Quality of life is often impacted by long-term joint damage, which can cause:7

  • Chronic pain and discomfort
  • Restricted mobility and/or disability, such as joint deformity and early-onset arthritis (pain and swelling in the joints)
  • An inability to perform everyday activities, which may result in anxiety and depression

It’s clear that there is a need for new therapeutic options that could:8,9

  • Reduce or eliminate the need for routine FIX replacement
  • Reduce or eliminate bleeds
  • Provide high, sustained, and consistent FIX activity levels

After years of scientific research and clinical study, gene therapy with HEMGENIX® has arrived.

A ONE-TIME INFUSION THAT COULD REDEFINE HOW YOU LIVE WITH HAEMOPHILIA B BY PROVIDING POTENTIAL LONG-TERM BLEED PROTECTION1,10

Key facts about gene therapy:11

Gene therapy is not a cure for haemophilia B
Gene therapy does not change your genes
Gene therapy does not change the genes that you pass on to your children
The virus used in gene therapy will not make you ill
Gene therapy may affect intimacy with your partner
Gene therapy will affect immediate plans for children
You will still need to visit your haemophilia treatment centre after gene therapy
Some people may experience an allergic reaction during gene therapy infusion

WHAT IS HEMGENIX®?

HEMGENIX® is a gene therapy that aims to give your liver the instructions it needs to make its own working FIX. The goal of HEMGENIX® is to offer protection from bleeds, without the need for regular FIX infusions.1,12,13

HEMGENIX® is approved in the UK for the treatment of adults with severe or moderately severe haemophilia B (congenital factor IX deficiency) who do not have FIX inhibitors (proteins made by the body that stop FIX from working).1,14

At present, data from the HOPE-B clinical study cover up to 3 years after treatment.10 It shows that people in the trial have higher FIX levels and fewer bleeds, compared with when they were receiving regular FIX infusions.10 However, it is thought that the effects of HEMGENIX® will last longer than this. The trial is ongoing, and more time is needed to gauge how long the effects will continue to last.15

HOPE-B is a clinical trial looking at the use of HEMGENIX® in 54 people with haemophilia B.10 It has been running in Europe and the United States since 2018.2,15

FIX, factor IX.

References

1. HEMGENIX® (etranacogene dezaparvovec). Summary of product characteristics. 2. Pipe SW et al. Gene Therapy with Etranacogene Dezaparvovec for Hemophilia B. N Engl J Med. 2023;388(8):706–718. 3. Shapiro AD et al. Recombinant factor IX-Fc fusion protein (rFIXFc) demonstrates safety and prolonged activity in phase 1/2a study in hemophilia B patients. Blood. 2012;119(3):666–672. 4. Mulder K, Llinás A. The target joint. Haemophilia. 2004;10(Suppl 4):152–156. 5. Jiménez-Yuste V et al. Joint status in Spanish haemophilia B patients assessed using the Haemophilia Early Arthropathy Detection with Ultrasound (HEAD-US) score. Haemophilia. 2019;25(1):144–153. 6. Schnohr C et al. Joint Mobility and Physical Function of Danish Hemophilia Patients: A Three-Wave Panel Study Spanning 24 Years. Acta Haematol. 2018;140(4):240–246. 7. O’Hara J et al. The impact of severe haemophilia and the presence of target joints on health-related quality-of-life. Health Qual Life Outcomes. 2018;16(1):84. 8. Miesbach W et al. How to discuss gene therapy for haemophilia? A patient and physician perspective. Haemophilia. 2019;25(4):545–557. 9. Pipe SW. Delivering on the promise of gene therapy for haemophilia. Haemophilia. 2021;27(Suppl 3):114–121. 10. Pipe S 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(Suppl 1):1055. 11. CSL Behring, Haemophilia Society. 2024. BEYOND Gene Therapy in Haemophilia B: Information, Guidance and Support. 12. Miesbach W et al. Gene therapy with adeno-associated virus vector 5-human factor IX in adults with hemophilia B. Blood. 2018;131(9):1022–1031. 13. Nathwani AC. Gene therapy for hemophilia. Hematology Am Soc Hematol Educ Program. 2019;2019(1):1–8. 14. National Institute for Health and Care Excellence (NICE). Technology appraisal guidance: Etranacogene dezaparvovec for treating moderately severe or severe haemophilia B. (2024). TA989. Published 24 July 2024. Available at: https://www.nice.org.uk/guidance/ta989. Accessed May 2025. 15. ClinicalTrials.gov. HOPE-B: Trial of AMT-061 in Severe or Moderately Severe Hemophilia B Patients (NCT03569891). Available at: https://clinicaltrials.gov/study/NCT03569891. Accessed May 2025.

Reporting side effects
If you get any side effects, talk to your doctor, pharmacist or nurse. This includes any possible side effects not listed in the package leaflet. You can also report side effects directly via the Yellow Card Scheme. By reporting side effects, you can help provide more information on the safety of this medicine.