Hemgenix (etranacogene dezaparvovec)
This submission was evaluated under the provisional registration process.
The following table summarises the key steps and dates for this application.
| Description | Date |
|---|---|
| Submission dossier accepted and first round evaluation commenced | 31 March 2023 |
| First round evaluation completed | 30 August 2023 |
| Second round evaluation completed | 15 November 2023 |
| Delegate’s Overall benefit-risk assessment and request for Advisory Committee advice | 8 January 2024 |
| Advisory Committee meeting | February 2024 |
| Registration decision (Outcome) | 15 March 2024 |
| Completion of administrative activities and registration on ARTG | 19 March 2024 |
| Number of working days from submission dossier acceptance to registration decision | 213 |
- Sucrose
- Polysorbate 20
- Potassium chloride
- Monobasic potassium phosphate
- Sodium chloride
- Dibasic sodium phosphate
- Hydrochloric acid (pH adjustment)
36 × 10 mL multi-vial finished pack
24 X 10 mL multi-vial finished pack
12 × 10 mL multi-vial finished pack
10 mL vial
A single dose of 20000000000000 genome copies per kilogram (kg) of body weight, corresponding to 2.0 mL/kg body weight.
For further information refer to the Product Information.
Drugs which have been taken by only a limited number of pregnant women and women of childbearing age, without an increase in the frequency of malformation or other direct or indirect harmful effects on the human fetus having been observed.
Studies in animals are inadequate or may be lacking, but available data show no evidence of an increased occurrence of fetal damage.
The use of any medicine during pregnancy requires careful consideration of both risks and benefits by the treating health professional. This must not be used as the sole basis of decision making in the use of medicines during pregnancy. The TGA does not provide advice on the use of medicines in pregnancy for specific cases. More information is available from obstetric drug information services in your State or Territory.
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.
Hemgenix (etranacogene dezaparvovec) is to be included in the Black Triangle Scheme. The Product Information (PI) and Consumer Medicine Information (CMI) for Hemgenix must include the black triangle symbol and mandatory accompanying text for five years, which starts from the date that the sponsor notifies the TGA of supply of the product.
The Hemgenix Risk Management Plan includes routine pharmacovigilance and the submission of periodic safety update reports.
More information
The latest Product Information (PI) and Consumer Medicine Information (CMI) can be found by searching the Australian Register of Therapeutic Goods (ARTG).
Australian Public Assessment Reports (AusPARs) can be found by searching our AusPAR dataset.
The latest news and updates regarding therapeutic goods regulation can be found on our news page.