Haemevolution

This European website contains promotional content developed by CSL Behring for European healthcare professionals and includes references to CSL Behring medicines. Prescribing information and Adverse Event reporting can be found at the bottom of the page.

Haemevolution

Are You a Healthcare Professional?

This European website, initiated and developed by CSL Behring, contains product specific information and promotional content on gene therapy in haemophilia B and is intended for an international audience of European healthcare professionals only. For more detailed information on the use of the product in your country, please visit www.cslbehring.com.

Yes, I am a healthcare professional*

Enter Site

*CSL Behring is legally obliged to restrict this website to healthcare professionals only. By clicking “Yes, I am a healthcare professional”, you confirm this statement is true and you accept all liability related. If you are not a healthcare professional you can visit our disease awareness webpage: www.haemevolution.eu

HEMGENIX® is the first and only approved gene therapy for haemophilia B in europe, addressing the root cause of haemophilia B and reducing its impact on patients.3

HEMGENIX® uses the non-replicating recombinant adeno-associated virus 5 (AAV5) vector for liver-directed introduction of a therapeutic FIX-Padua gene.1 This highly active FIX Padua protein variant is shown to generate 5-8 times higher mean endogenous FIX activity than the more common wild-type FIX protein.4,5,6

Gene Therapy For Haemophilia Explained

Haemophilia A and B are both monogenic, X-linked genetic disorders that are suitable for gene therapy.2,7 Gene therapy for haemophilia aims to increase clotting Factor VIII levels (haemophilia A) or Factor IX levels (haemophilia B), by providing a new functional gene or coding sequence, leading to improved health outcomes and reducing or eliminating the need for routine factor prophylaxis.2,8,9

Mechanism Of Action Infographic

Gene Therapy Mechanism Of Action Visual Infographic

See gene therapy for haemophilia broken down into 7 steps in this visual mechanism of action infographic, from AAV vector attachment to clotting factor protein synthesis.

Download Mechanism Of Action Infographic Download Mechanism Of Action Infographic

HOPE-B: A pioneering Phase 3 trial

The robust efficacy, safety and tolerability profile of HEMGENIX® was demonstrated in the multinational, pivotal Phase 3 HOPE-B trial.1,2

HOPE-B included 54 male patients ≥18 years of age with congenital haemophilia B (FIX activity ≤2% of normal), currently on continuous FIX prophylaxis for ≥2 months prior to screening.1,2

Primary Endpoint:1,2

Comparing annualized bleeding rate (ABR) for all bleeds between HEMGENIX® and prophylaxis for non-inferiority between the 6-month lead-in period and the 52 weeks following stable FIX expression (month 7-18).

Key secondary endpoints:1,2

  • FIX activity at 6, 12, and 18 months after dosing
  • Proportion of patients remaining free of continuous prophylaxis
  • Occurrence and resolution of target joints, and proportion of patients with zero bleeds
  • Correlation of FIX activity levels to pre-existing AAV5 NAb titre
  • Adverse events

The primary endpoint has been completed, with patient data evaluated for the first 24 months following HEMGENIX® treatment. Patient follow up will continue for 5 years after administration of HEMGENIX®.1,2

Previous

Home

Return to the homepage to see the HEMGENIX® therapy overview

Next

Clinical Evidence

Find out more about the efficacy, safety and tolerability data behind HEMGENIX®

References

  1. EU SmPC HEMGENIX® (European Medicine Agency, 2023)
  2. Pipe SW, et al. Gene Therapy with Etranacogene Dezaparvovec for Hemophilia B. N Engl J Med. 2023;388:706-718.
  3. CSL Behring. First Gene Therapy for Hemophilia B, CSL's HEMGENIX®, Approved by the European Commission. Press release. 20 Feb 2023. Available at: https://newsroom.csl.com/2023-02-20-First-Gene-Therapy-for-Hemophilia-B,-CSLs-HEMGENIX-R-,-Approved-by-the-European-Commission. Accessed March 2023.
  4. Nathwani AC. Gene therapy for Haemophilia. Hematology Am Soc Hematol Educ Program. 2019;2019(1):1-8.
  5. Thornburg CD. Etranacogene dezaparvovec for hemophilia B gene therapy. Ther Adv Rare Dis. 2021;2:1–14.
  6. Von Drygalski A, et al. Etranacogene dezaparvovec (AMT-061 phase 2b): normal/near normal FIX activity and bleed cessation in hemophilia B Blood Advances. 2019;3(21):3241-47.
  7. Rodríguez-Merchán EC, et al. Gene Therapy in Hemophilia: Recent Advances. Int. J. Mol. Sci. 2021;22:7647-67.
  8. Miesbach W, et al. Gene therapy with adeno-associated virus vector 5–human factor IX in adults with hemophilia B. Blood. 2018;131(9):1022-31.
  9. Perrin GQ, Herzog RW, Markusic DM. Update on clinical gene therapy for Haemophilia. Blood. 2019;133(5):407-414.