1579 - Emicizumab for routine prophylaxis to prevent bleeding or reduce the frequency of bleeding episodes in patients with moderate to severe Haemophilia A (congenital Factor VIII deficiency) WITHOUT Factor VIII inhibitors

Page last updated: 29 March 2019

Application Detail

Status

Open

Description of Medical Service

Emicizumab 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):

  • 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.

Emicizumab is registered as a prescription medicine by the Therapeutic Goods Administration (TGA).

Description of Medical Condition

Haemophilia 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

Therapeutic

Previous Application Number

Similar to earlier application 1510, but for a different population

Associated Documentation

Application Form

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Consultation Survey

Feedback 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).

PICO Confirmation

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Assessment Report

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Public Summary Document

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Meetings for this Application

PASC

The population in application 1579 (patients WITHOUT factor VIII inhibitors) was considered as part of PASC’s consideration of application 1510. Post PASC, the population in application 1579 was placed on hold, while the population in application 1510 progressed through ESC and MSAC (See 1510)

ESC

13 - 14 June 2019 (first ESC consideration of non-inhibitor population)

MSAC

1-2 August 2019 (first MSAC consideration of non-inhibitor population)