Turoctocog alfa pegol (1) – Esperoct®

• Clinical trials
  • The Phase III PATHFINDER2 trial is an open-label, non--controlled trial with a parallel-group design, comparing on-demand treatment with prophylactic treatment using turoctocog alfa pegol in previously treated male patients aged 12 years and over with severe haemophilia A (factor VIII activity < 1 per cent).
  • In the single-arm, non-comparative, surgical Phase III trial PATHFINDER3, turoctocog alfa pegol was investigated for the management of bleeding during surgical procedures.

Patients aged 12 years and over with haemophilia A (congenital factor VIII deficiency)

• For the treatment and prophylaxis of bleeding in patients aged 12 years and over with haemophilia A (congenital factor VIII deficiency), the additional benefit compared with the appropriate comparator therapy is not proven.
• The additional benefit compared with the appropriate comparator therapy is therefore not proven.
• In its dossier for the assessment of the additional benefit of Turoctocog alfa pegol, the pharmaceutical manufacturer does not present any direct comparative studies against the appropriate comparator therapy.
• The before-and-after comparisons submitted are also unsuitable for addressing the benefit assessment question due to methodological limitations.
• Consequently, there are no suitable data available for the assessment of the additional benefit of Turoctocog alfa pegol for the treatment and prophylaxis of bleeding in previously treated patients aged 12 years and over with haemophilia A (congenital factor VIII deficiency).
• Overall, therefore, an additional benefit is not proven.
• Overall assessment
  • On balance, therefore, an additional benefit is not proven.

Courtesy translation only, please refer to the German original.