Applications

1728 – Etranacogene dezaparvovec for the treatment of Haemophilia B

Find out about the service or technology in this application and the medical condition it addresses. You can also view the application documents, the deadlines for providing consultation input and the outcome of the application when the MSAC process is complete.

  • Status Complete
  • Type Referral
  • Pre-PASC consultation Closed
  • Pre-MSAC consultation Closed
  • Outcome Not supported

Application details

Reason for application

Referral from the National Blood Authority.

Service or technology in this application

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

Medical condition this application addresses

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.

Consultation survey and deadlines

  • PASC consultation: Closed
  • MSAC consultation: Closed Friday 14 June 2024

Meetings to consider this application

  • PASC meeting: 13–14 April 2023
  • ESC meeting: 13–14 June 2024
  • MSAC meeting: 1–2 August 2024