HEMGENIX (CSL Behring Australia Pty Ltd)
Product name
HEMGENIX
Sponsor
Date registered
Evaluation commenced
Decision date
Approval time
213 (255 working days)
Active ingredients
etranacogene dezaparvovec
Registration type
NCE/NBE
Indication
This medicine has provisional approval in Australia.
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.
Registration process
Provisional registration
Involves early access to vital and life-saving medicines through time-limited registration
Orphan drug
Sponsors receive a fee waiver to help bring medicines for a small population to market.