1728.1 – Etranacogene dezaparvovec for the treatment of Haemophilia B

Referral from the National Blood Authority.

Etranacogene dezaparvovec is a gene therapy designed to introduce a copy of the Factor IX (FIX) gene to address the lack of functional FIX protein expression in a haemophila B patient. The therapy is delivered by infusion into the bloodstream.

Type: Therapeutic technology

Haemophilia is a congenital lifelong bleeding disorder caused by deficiencies in coagulation factors as a result of mutations in clotting factor genes. Haemophilia B is less common than Haemophilia A, accounting for approximately 15% of total cases, and the majority of people affected are male due to the associated coagulation factor being found on the X chromosome.

The following consultation deadlines apply:

• Pre-PASC consultation deadline: Bypassing PASC 
• Pre-MSAC consultation deadline: Friday 13 June 2025 11:59 pm AEST  

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This application will be considered at the following meetings:

• PASC meeting: Bypassing PASC
• ESC meeting: 12-13 June 2025
• MSAC meeting: 31 July - 1 August 2025

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The outcome of this application is pending.

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• the MSAC process
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