Emicizumab (2) – Hemlibra®

• Clinical trials
  • The HAVEN 3 study is an open-label, multicentre, parallel-group study with three randomised arms and one non-randomised arm.
  • The SPINART study is a randomised, open-label, multicentre, parallel-group study comparing routine prophylaxis with recombinant factor VIII preparations (Octocog alfa) with on-demand treatment with recombinant factor VIII preparations (Octocog alfa).

Patients with severe haemophilia A (hereditary factor VIII deficiency, FVIII < 1 %) without factor VIII inhibitors who are eligible for routine prophylaxis

• For patients with severe haemophilia A (hereditary factor VIII deficiency, FVIII < 1 %) without factor VIII inhibitors, the additional benefit of emicizumab as routine prophylaxis compared with the appropriate comparator therapy is not proven.
• The additional benefit compared with the appropriate comparator therapy is therefore not proven.
• Morbidity – number of treated bleeds
  • The results of the adjusted, indirect comparison of bleeding events were taken into account to estimate the differences in annualised bleeding rates.
  • These show no statistically significant differences between routine prophylaxis with emicizumab and prophylaxis with a recombinant factor VIII preparation, neither for all treated bleeds nor for treated joint bleeds.
  • However, these data on bleeding rates allow conclusions to be drawn solely regarding morbidity.
• Furthermore, no usable data are available for the quality of life category, for other morbidity endpoints, or for mortality.
• Conclusion
  • It is not possible, on the basis of the data presented, to weigh up any additional benefit or less benefit against the appropriate comparator therapy; consequently, no additional benefit over and above the appropriate comparator therapy can be inferred.

Courtesy translation only, please refer to the German original.