FAQs

The image is used for illustrative purposes only and does not represent actual patients.

FREQUENTLY ASKED QUESTIONS

HEMGENIX® is the first gene therapy approved for haemophilia B.1 As you discuss HEMGENIX® with your patients, important questions can arise.

Here, you can find answers to some of these common questions about treatment with HEMGENIX®.

HOW DOES HEMGENIX® WORK?
WHY DOES HEMGENIX® USE THE AAV5 VECTOR?
DOES HEMGENIX® ELIMINATE HAEMOPHILIA B IN PATIENTS?
HOW LONG DOES HEMGENIX® LAST?
WOULD HEMGENIX® LIMIT PATIENTS FROM TAKING FIX ON DEMAND IF NEEDED?2
WHAT ARE THE MOST COMMON SIDE EFFECTS WITH HEMGENIX®?
WHICH PATIENTS WERE INCLUDED IN THE HOPE-B STUDY?
HOW IS HEMGENIX® DOSED?2
DOES HEMGENIX® AFFECT THE PATIENT’S DNA?2
WILL HEMGENIX® BE PASSED ON TO THE PATIENT’S CHILDREN?2

AAV5, adeno-associated viral vector serotype 5; ALT, alaline aminotransferase; FIX, factor IX; HIV, human immunodeficiency virus; NAb, neutralising antibody; TEAE, treatment-emergent adverse event; ULN, upper limit of normal.

References

1. 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. 2. HEMGENIX® (etranacogene dezaparvovec). Summary of product characteristics. 3. Perrin GQ et al. Update on clinical gene therapy for hemophilia. Blood. 2019;133(5):407–414. 4. Ronzitti G et al. Human Immune Responses to Adeno-Associated Virus (AAV) Vectors. Front Immunol. 2020;11:670. 5. Vance MA et al. AAV Biology, Infectivity and Therapeutic Use from Bench to Clinic. In: Hashad D, ed. Gene Therapy Principles and Challenges. 1st ed. IntechOpen; 2015:119–143. 6. Louis Jeune V et al. Pre-existing anti-adeno-associated virus antibodies as a challenge in AAV gene therapy. Hum Gene Ther Methods. 2013;24(2):59–67. 7. Pipe S et al. Clinical Considerations for Capsid Choice in the Development of Liver-Targeted AAV-Based Gene Transfer. Mol Ther Methods Clin Dev. 2019;15:170–178. 8. Zincarelli C et al. Analysis of AAV serotypes 1–9 mediated gene expression and tropism in mice after systemic injection. Mol Ther. 2008;16(6):1073–1080. 9. Nathwani AC. Gene therapy for hemophilia. Hematology Am Soc Hematol Educ Program. 2019;2019(1):1–8. 10. Kruzik A et al. Prevalence of Anti-Adeno-Associated Virus Immune Responses in International Cohorts of Healthy Donors. Mol Ther Methods Clin Dev. 2019;14:126–133. 11. Klamroth R et al. Global Seroprevalence of Pre-existing Immunity Against AAV5 and Other AAV Serotypes in People with Hemophilia A. Hum Gene Ther. 2022;33(7–8):432–441. 12. Falese L et al. Strategy to detect pre-existing immunity to AAV gene therapy. Gene Ther. 2017;24(12):768–778. 13. Pipe SW et al. Gene Therapy with Etranacogene Dezaparvovec for Hemophilia B. N Engl J Med. 2023;388(8):706–718. 14. Thornburg CD. Etranacogene dezaparvovec for hemophilia B gene therapy. Ther Adv Rare Dis. 2021;2:26330040211058896. 15. Data on file. Study CSL222_3001 study (HOPE-B): 3-year follow-up analysis. Database Extract Date: 06 June 2023.