Hemgenix

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.