Application Detail

Description of Medical Service

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.

Description of Medical Condition

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.

Reason for Application

Referral from the National Blood Authority

Medical Service Type

Therapeutic technology

Previous Application Number/s

Not Applicable

Associated Documentation

Application Form

Application Form (PDF 1133 KB)
Application Form (Word 185 KB)

Consultation Survey

Consultation Survey (PDF 658 KB)
Consultation Survey (Word 27 KB)

PASC Consultation
Consultation closed

MSAC Consultation
MSAC consultation input closed Friday, 14 June 2024.

For further information please refer to PASC, ESC, MSAC Key Dates

For further information on the consultation process please refer to MSAC Consultation Process

PICO Confirmation

PICO Confirmation (PDF 1523 KB)
PICO Confirmation (Word 328 KB)

Assessment Report

-

Public Summary Document

Public Summary Document (PDF 918 KB)
Public Summary Document (Word 398 KB)

Meetings for this Application

PASC

13–14 April 2023

ESC

13-14 June 2024

MSAC

1-2 August 2024