etranacogene dezaparvovec at a glance
Medicine class
Adeno-associated viral vector gene therapy and advanced therapy medicinal product.
Licensed use
Moderately severe or severe haemophilia B in adults without current or previous factor IX inhibitors.
Administration
Intravenous infusion in a specialist setting equipped to manage infusion reactions.
Follow-up
Ongoing monitoring of liver health, factor IX activity and bleeding control.
What is etranacogene dezaparvovec used for?
Its licence covers:
Severe haemophilia B in adults without a history of factor IX inhibitors.
Moderately severe haemophilia B in adults without a history of factor IX inhibitors.
How does etranacogene dezaparvovec work?
A non-replicating AAV5 vector carries instructions for the active Padua variant of factor IX into liver cells. The cells can then produce factor IX, increasing clotting activity and protection against bleeding. Response varies between individuals.
How to take etranacogene dezaparvovec
A specialist haemophilia team prepares and gives it as a diluted intravenous infusion. It is not self-administered or taken with food.
Assessment includes factor IX inhibitor testing, anti-AAV5 antibody assessment and detailed liver evaluation.
Staff monitor for reactions during and after the infusion.
Continue the existing haemophilia treatment plan until the specialist team confirms a change.
Keep the Patient Card and show it to healthcare professionals.
Do not donate blood, organs, tissues or cells for transplantation after treatment.
etranacogene dezaparvovec side effects
Common or expected effects
- Headache
- Flu-like illness
- Raised liver enzymes or other abnormal liver blood tests
- Infusion reactions such as flushing, itchy rash, fever, dizziness, chest discomfort or upper abdominal pain
- Nausea
- Fatigue or general malaise
Get urgent medical advice
- Severe hypersensitivity or anaphylaxis during or shortly after infusion
- Significant liver inflammation, which may reduce factor IX expression
- Possible abnormal clotting, with symptoms such as sudden chest pain, breathlessness or painful one-sided leg swelling
etranacogene dezaparvovec in pregnancy
There are no pregnancy data, and potential fetal harm is unknown. It should not be used during pregnancy and is not recommended for women who could become pregnant. Treated males and female partners who could become pregnant must use barrier contraception for 12 months. Treated males must not donate semen.
etranacogene dezaparvovec while breastfeeding
It is unknown whether it passes into human milk, and risk to a breastfed child cannot be excluded. It should not be used while breastfeeding; discuss feeding plans with the specialist team before treatment.
etranacogene dezaparvovec interactions
Formal in-vivo interaction studies have not been performed. Review all prescribed, over-the-counter and complementary products with the specialist team.
Potentially hepatotoxic medicines, herbal products or supplements
May increase liver reactions and potentially reduce gene-therapy effectiveness, requiring specialist review and closer monitoring.
Alcohol and other hepatotoxic substances
May add to liver injury risk, particularly during early follow-up.
CYP3A4 inhibitors or inducers
If corticosteroids are required, these may increase steroid adverse effects or reduce steroid effectiveness.
Common questions about etranacogene dezaparvovec
Answers are fully visible for fast scanning and source review.
Who can receive etranacogene dezaparvovec?
Adults with moderately severe or severe haemophilia B who have no current or previous factor IX inhibitors. Suitability also depends on liver health, infection status and specialist assessment.
Is it a factor IX replacement product?
No. It enables liver cells to produce factor IX. Replacement factor IX may still be needed while response develops or for later bleeding, trauma or procedures.
Can treatment be repeated?
Sources
These sources were used to prepare and review this medicine page.
Prepared and reviewed by the iatroX editorial team.