Basic Information
Hemgenix
Regulatory Information
EMEA/H/C/004827
February 20, 2023
December 15, 2022
6
January 15, 2025
Company Information
Germany
Emil-von-Behring-Straße 76 35041 Marburg
Csl Behring Gmbh
Drug Classification
Active Substances Detail
EMA Resources
Detailed Information
Therapeutic Indication
### Therapeutic indication Treatment of severe and moderately severe Haemophilia B (congenital Factor IX deficiency) in adult patients without a history of Factor IX inhibitors.
Overview Summary
Hemgenix is a medicine used to treat adults with severe and moderately severe haemophilia B, an inherited bleeding disorder caused by the lack of factor IX (a protein needed to produce blood clots to stop bleeding). It is used in adults who have not developed inhibitors (proteins made by the body’s natural defenses) against factor IX. Haemophilia B is rare, and Hemgenix was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 21 March 2018. Further information on the orphan designation can be found here: [EU/3/18/1999](/en/medicines/human/orphan-designations/eu-3-18-1999) Hemgenix contains the active substance etranacogene dezaparvovec and is a type of advanced therapy medicine called a ‘gene therapy product’. This is a type of medicine that works by delivering genes into the body.