How HEMGENIX® Works
WHAT IS HEMGENIX®?
People with haemophilia B are born with an altered form of a gene needed to make FIX, an essential protein required for blood to clot and stop any bleeding.1,2
People with haemophilia B have insufficient levels of FIX and are prone to internal or external bleeding episodes.1,2
HEMGENIX® is a gene therapy treatment that contains the active substance etranacogene dezaparvovec. A gene therapy treatment works by delivering a gene into the body to correct a genetic defect.1
HEMGENIX® is used for the treatment of severe and moderately severe haemophilia B (congenital FIX deficiency) in adults who do not have current or past inhibitors (neutralising antibodies) against the FIX protein.1
HOW DOES HEMGENIX® WORK?
HEMGENIX® works by giving you a working version of the F9 gene. The gene is put inside a virus, which carries it to the liver.2,3
The virus used for gene therapy is inactivated. This means that it cannot spread in the body and does not cause illness in humans.2,3
Once the gene is delivered, the liver can start making its own FIX.3
The gene should stay in the liver cells, without becoming part of your DNA.3
This allows the liver to produce the FIX protein and raise the levels of working FIX in the blood, helping the blood to clot more normally and prevent or reduce bleeding episodes.1,2
FIX, Factor IX.
References
1. HEMGENIX® (etranacogene dezaparvovec). Summary of product characteristics. 2. Nathwani AC. Gene therapy for hemophilia. Hematology Am Soc Hematol Educ Program. 2019;2019(1):1–8. 3. Nathwani AC. Gene therapy for hemophilia. Hematology Am Soc Hematol Educ Program. 2022;2022(1):569–578.
Reporting side effects
If you experience 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. Adverse events should also be reported to CSL Behring UK Ltd on 01444 447 405 or at medinfo@cslbehring.com.
▼ This medicine is subject to additional monitoring. This will allow quick identification of new safety information. You can help by reporting any side effects you may get. See above for how to report side effects.
