<p>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.</p><p>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: <a href="/en/medicines/human/orphan-designations/eu-3-18-1999" data-entity-type="node" data-entity-uuid="eb64bd39-a669-4ecb-81c0-513908971e6d" data-entity-substitution="canonical">EU/3/18/1999</a> </p><p>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.</p>
Therapeutic Indication
<div class="subsection mt-3-5"> <h3>Therapeutic indication</h3><p>Treatment of severe and moderately severe Haemophilia B (congenital Factor IX deficiency) in adult patients without a history of Factor IX inhibitors.</p> </div>
Therapeutic Area (MeSH)
ATC Code
B02BD
ATC Item
N/A
Pharmacotherapeutic Group
Blood coagulation factors
Active Substance (Summary)
INN / Common Names
| Substance | CAS | Monograph |
|---|---|---|
| Etranacogene dezaparvovec | N/A | Etranacogene dezaparvovec |
EMA Name
Hemgenix
Medicine Name
Hemgenix
Aliases
N/ANo risk management plan link.
