Hemgenix
What Hemgenix 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.
Approved use, as published by the European Medicines Agency.
What the labels mean
- Orphan medicine: for a rare disease (affecting no more than 5 in 10,000 people in the EU).
- Advanced therapy: a gene, cell or tissue-engineered medicine.
- PRIME: a priority medicine that the EMA supported early because it may meet an unmet need.
- Conditional approval: approved on less complete data because the need is high; more data must follow.
Key facts
- Brand
- Hemgenix
- Active substance
- etranacogene dezaparvovec
- Disease area
- Hemophilia B
- Medicine group
- Blood coagulation factors
- ATC code
- B02BD
- Marketing authorisation holder
- CSL Behring GmbH
- Authorised in the EU since
- 2023
Official information
In the USA
Same active substance: Hemgenix
Specialist medicine: started and supervised by a specialist doctor or hospital. This page is a plain-language summary of public official information, not medical advice; follow your doctor and the official product information.
Common questions
What is Hemgenix used for?
Treatment of severe and moderately severe Haemophilia B (congenital Factor IX deficiency) in adult patients without a history of Factor IX inhibitors.
What is the active substance in Hemgenix?
Hemgenix contains etranacogene dezaparvovec.
Who makes Hemgenix?
The EU marketing authorisation holder is CSL Behring GmbH.
Is Hemgenix an orphan medicine?
Yes. Hemgenix is designated an orphan medicine in the EU, for a rare disease (Hemophilia B).
Source: European Medicines Agency medicines data (ema.europa.eu), reproduced with acknowledgement of the source. Page made 5 November 2026 by Satya Babu.
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