About etranacogene dezaparvovec
Adeno-associated virus gene therapy
Used for
- Treatment of adults with hemophilia B who use factor IX prophylaxis or have serious bleeding.
Usual adult dose
One-time intravenous infusion: 2 × 10¹³ genome copies per kilogram.
Common side effects
- Headache
- Flu-like symptoms, including fatigue and fever
- Infusion reactions
- Elevated liver enzymes
Important warnings
- Serious liver injury can occur; liver tests are monitored before and after infusion.
- Factor IX levels may decline over time, and bleeding treatment may still be needed.
- Avoid alcohol and medicines that can harm the liver during the monitoring period.
- Not suitable for people with factor IX inhibitors or certain pre-existing liver conditions.
Pregnancy
Effects on pregnancy are unknown. Treatment is not recommended during pregnancy; discuss pregnancy plans with a specialist.
Breastfeeding
It is unknown whether this treatment passes into breast milk. Breastfeeding decisions require specialist advice.
Interactions
- Medicines that harm the liver may increase liver-injury risk.
- Corticosteroids or other immune-suppressing medicines may be needed if liver enzymes rise.
AI-generated from general medical knowledge, not from this product's official leaflet. It may be incomplete or out of date. Not medical advice — ask a pharmacist or doctor before making any decision.