Emicizumab (3) – Hemlibra®

• Clinical trials
  • The HAVEN 6 trial is a single-arm trial that included patients of all age groups with mild (residual Factor VIII activity > 5 % and < 40 %) or moderate (residual Factor VIII activity ≥ 1% and ≤ 5%) hereditary haemophilia A without Factor VIII inhibitors, for whom the investigator deemed prophylaxis to be indicated.

Patients with moderate haemophilia A (hereditary factor VIII deficiency, FVIII ≥ 1% and ≤ 5%) and a severe bleeding phenotype without factor VIII inhibitors, who are eligible for routine prophylaxis

• For patients with moderate haemophilia A (hereditary factor VIII deficiency, FVIII ≥ 1% and ≤ 5%) and a severe bleeding phenotype without factor VIIIinhibitors, who are eligible for routine prophylaxis, the additional benefit of emicizumab as routine prophylaxis compared with the appropriate comparator therapy is not proven.
• Overall, therefore, additional benefit for emicizumab as routine prophylaxis against bleeding episodes in patients with moderate haemophilia A without factor VIII inhibitors and a severe bleeding phenotype is not proven, compared with the appropriate comparator therapy.
• morbidity
  • The primary endpoint of the study was treated bleeds, operationalised as the annualised bleeding rate.
• Overall assessment
  • In the overall review, additional benefit is not proven for emicizumab in the routine prophylaxis of bleeding episodes in patients with moderate haemophilia A without factor VIII inhibitors and a severe bleeding phenotype, compared with the appropriate comparator therapy.

Courtesy translation only, please refer to the German original.