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Hemgenix (etranacogene dezaparvovec)
Australian Prescription Medicine Decision Summary
Published
Product name
Hemgenix
Active ingredient
Etranacogene dezaparvovec
Submission type
Type A - New biological entity
Decision
Hemgenix has been provisionally approved.
Decision date
Registration date
What this medicine was approved for
Hemgenix (etranacogene dezaparvovec) was provisionally approved for the following therapeutic use:
Hemgenix is an adeno-associated virus vector-based gene therapy indicated for treatment of adults with haemophilia B (congenital factor IX deficiency), without a history of factor IX inhibitors, who:
- currently use factor IX prophylaxis therapy, or
- have current or historical life-threatening haemorrhage, or repeated, serious spontaneous bleeding episodes.
The decision to approve this indication has been made on the basis of short-term efficacy and safety data from the clinical trial program. Continued approval of this indication depends on confirmation of longer-term benefit from ongoing clinical trials.
How this medicine works
Hemgenix is a treatment for haemophilia B, a disease which is caused by a defect in the gene which produces coagulation factor IX protein. This leads to insufficient production of factor IX, a protein that is critical for blood clotting. Hemgenix is composed of a functional Factor IX gene sequence packaged into a virus, which acts as a delivery vehicle for the gene. Hemgenix treats hemophilia B by introducing a copy of the fgactor IX gene into liver cells, which then produce functional factor IX. This increases the levels of functional factor IX protein in the blood. Higher factor IX levels can restore blood clotting, reducing bleeding episodes in people with haemophilia B.
Why the TGA approved or did not approve this medicine
The decision was based on quality (chemistry and manufacturing), nonclinical (pharmacology and toxicology), clinical (pharmacology, safety and efficacy) and risk management plan information submitted by the sponsor. The benefit-risk profile of Hemgenix was considered favourable for the therapeutic use approved.
Consumer Medicine Information (CMI)
The CMI leaflet offers guidance for consumers to support safe and effective use of the medicine. The CMI includes information on dose, how to use the medicine properly, potential side effects, safety precautions, storage instructions and more.
The Australian CMIs can be accessed through the ARTG details hyperlink below.
Product Information (PI)
The PI document provides essential prescribing information for health professionals, including details on dosage recommendations, pregnancy category, contraindications, precautions and potential side effects.
The Australian PIs can be accessed through the ARTG details hyperlink below.
Sponsor
Other resources
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