Description of Medical ServiceEmicizumab is a monoclonal antibody that is bispecific for Factor IXa and Factor X. The two arms bind to each of these factors, mimicking the action of Factor VIII and allowing the normal clotting cascade to continue. Emicizumab offers the following advantages over the recombinant or plasma-derived factors for patients requiring prophylaxis (Shima 2016):
Emicizumab will be evaluated as a prescription medicine by the Therapeutic Goods Administration (TGA).
- Subcutaneous vs intravenous administration;
- Weekly vs every other day administration;
- Sustained plasma concentrations;
- No risk of FVIII inhibitor development;
- Activity irrespective of the presence of FVIII inhibitors.
Description of Medical ConditionHaemophilia A (HMA) is an X-linked congenital bleeding disorder caused by a deficiency of the coagulation factor VIII (FVIII). HMA can be mild, moderate or severe depending on the level of deficiency.
- Mild: usually bleed as a result of injury or major surgery;
- Moderate: bleed spontaneously but usually as a result of injury;
- Severe: frequent spontaneous bleeding into muscles and joints.
The mainstay of treatment for HMA is replacement of the deficient Factor VIII by intravenous (IV) recombinant or plasma-derived Factor VIII, either on demand (to treat a bleed) or as prophylaxis. Prophylaxis prevents bleeding and irreversible joint destruction and is considered advisable before HMA patients engage in activities with higher risk of injury. Development of inhibitors (antibodies that neutralise replacement FVIII) is considered the most severe treatment-related complication in HMA, with a lifetime risk of development of ~20–30% in severe HMA and 5–10% in mild or moderate disease (ACHDO 2016).
Reason for Application
Health Technology Assessment
Medical Service Type
Previous Application Number1510
Consultation SurveyFeedback and comments are welcome at any stage during the MSAC process. Please provide comments on Application 1579 via a maximum of two A4 pages to HTA@health.gov.au (making sure that you type the application number at the start of your comments and in the subject heading of your email).
Public Summary Document-
Meetings for this Application
PASCNot applicable, because 1510.1 is a re-submission of earlier application 1510 (relating to patients WITH factor VIII inhibitors)
ESC13 June 2019
MSAC1-2 August 2019