Concizumab (1) – Alhemo®

• Clinical trials
  • In addition, the pharmaceutical manufacturer presents the multicentre, partially randomised, open-label Explorer7 trial, which formed the basis for marketing authorisation. This trial compared routine prophylaxis with concizumab and on-demand treatment with bypassing agents, and included male patients aged 12 years and over with congenital haemophilia A or B of any severity, as well as factor VIIIor factor IX inhibitors.

Adults and adolescents aged 12 years and over with haemophilia A (congenital factor VIII deficiency) and factor VIII inhibitors, for whom there is an indication for routine prophylaxis

• For adults and adolescents aged 12 years and over with haemophilia A (congenital factor VIII deficiency) and factor VIII inhibitors, who have an indication for routine prophylaxis, additional benefit is not proven.
• In its dossier for the assessment of the additional benefit of Concizumab, the pharmaceutical manufacturer does not present any direct comparative studies against the appropriate comparator therapy.
• Overall, on the basis of the study submitted, no added benefit of Concizumab compared with the appropriate comparator therapy can be concluded for adults and adolescents aged 12 years and over with haemophilia A (congenital factor VIII deficiency) and factor VIII inhibitors, with an indication for routine prophylaxis, no additional benefit for Concizumab can be inferred compared with the appropriate comparator therapy.
• Overall assessment
  • In the overall assessment, for adults and adolescents aged 12 years and over with haemophilia A (congenital factor VIII deficiency) and factor VIII inhibitors, for whom routine prophylaxis is indicated, the additional benefit of Concizumab compared with the appropriate comparator therapy is not proven.

Courtesy translation only, please refer to the German original.