etranacogene dezaparvovec
Hemgenix · ATC code B02BD
Also known as: Etranacogen dezaparvovec, Etranakogen dezaparvovek
- Key facts
- Active substance
- etranacogene dezaparvovec
- ATC code
- B02BD
- Authorisation
- centrally authorised across the EU (EMA)
- Dosage forms
- Hemgenix 1 x 1013 genome copies/mL concentrate for solution for infusion
- Marketing authorisation holder
- CSL Behring GmbH
- Information as of
- 2026-10-28
Key facts
Etranacogene dezaparvovec (Hemgenix) is a one-time AAV5-based gene therapy that delivers a factor IX gene to the liver, used for severe and moderately severe haemophilia B in adults.
What it is used for
- Treatment of severe and moderately severe haemophilia B (congenital factor IX deficiency) in adult patients without a history of factor IX inhibitors.
Dosing
- A single dose of 2 × 10¹³ genome copies/kg body weight (2 mL/kg) as an intravenous infusion after dilution; Hemgenix can be administered only once.
- Before treatment: absence of factor IX inhibitors must be shown, and liver health and pre-existing neutralising anti-AAV5 antibody titres must be assessed.
- Exogenous factor IX may be needed in the first weeks after infusion; factor IX activity should be monitored (e.g. weekly for 3 months), using the same assay over time.
- Given where staff and equipment can immediately treat infusion reactions. No dose adjustment in the elderly or in renal impairment; for hepatic impairment see the full text.
Contraindications
- Hypersensitivity to the active substance or to any of the excipients.
- Active infections, acute or uncontrolled chronic.
- Known advanced hepatic fibrosis or cirrhosis.
Important warnings
- Pre-existing neutralising anti-AAV5 antibodies above a titre of 1:898 may reduce efficacy.
- Infusion reactions including hypersensitivity and anaphylaxis are possible; patients are monitored during and for at least 3 hours after the infusion.
- Liver transaminase elevations may occur, mostly in the first 3 months, and may reduce efficacy; transaminases should be closely monitored after treatment.
- Vector integration into human genomic DNA has been observed; there is a theoretical risk of malignancy.
Side effects
- Very common: headache (31.6% of patients), ALT elevations (22.8%), AST elevations (17.5%), influenza-like illness (14%), C-reactive protein increased, infusion-related reactions.
- Common: dizziness, nausea, fatigue, malaise, blood creatine phosphokinase increased, blood bilirubin increased.
Interactions
- Existing medicines should be reviewed before treatment; ALT and factor IX activity should be monitored closely when a new medicine is started.
- Hepatotoxic medicines or substances (including alcohol, herbal products and supplements) may reduce efficacy and increase the risk of liver reactions, particularly in the first year.
Pregnancy and breastfeeding
- Not recommended in women of childbearing potential and should not be used during pregnancy.
- For 12 months after treatment, men and their female partners must use barrier contraception, and men must not donate semen.
Summary of the official product information, written with AI on 2026-10-29. Every number was checked automatically against the original text. Not reviewed by a physician – the full official text is authoritative.
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For patients
Package leaflet, verbatim
This product has no separate US patient information.
Authorisation and brand names by country
Medicines with exactly this active-substance composition, as listed in the national registers – alphabetical and without ranking. Whether a product is currently available is not shown here.
EU – centrally authorised (all member states)
- HemgenixCSL Behring GmbH · EMEA/H/C/004827
Source: European Medicines Agency (EMA), product information
Brand names in 3 more European countries
- Iceland
- Hemgenix
- Liechtenstein
- Hemgenix
- Norway
- Hemgenix
EMA – Article 57 product data (EU/EEA), 2026-10-29
Medelix receives no money from pharmaceutical companies. Brand names are listed alphabetically and without ranking.
Other substances in the same group
Another question about this substance?
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Sources
- European Medicines Agency. Hemgenix – EPAR, Product Information. 2026-10-28. https://www.ema.europa.eu/en/documents/product-information/hemgenix-epar-product-information_en.pdf
Editorial: Medelix editorial team · last updated 2026-10-28 · How these pages are made · Report an error
Source: European Medicines Agency (EMA), product information. Not advertising, not a treatment recommendation. For questions about your own treatment, ask your doctor or pharmacist.