Description of Medical ServiceEtranacogene 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 ConditionHaemophilia 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 ApplicationReferral from the National Blood Authority
Medical Service TypeTherapeutic technology
Previous Application Number/sNot Applicable
Application FormApplication Form (PDF 1133 KB)
Application Form (Word 185 KB)
Consultation SurveyConsultation Survey (PDF 658 KB)
Consultation Survey (Word 27 KB)
MSAC consultation TBA – please subscribe to the MSAC Email Bulletin to be notified when consultation opens for this application
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 ConfirmationPICO Confirmation (PDF 1523 KB)
PICO Confirmation (Word 328 KB)