Nonacog beta pegol (1) – Refixia®

• Clinical trials
  • The marketing authorisation studies NN7999-3747 and NN7999-3775 described in the dossier are open-label, multicentre, uncontrolled studies designed to investigate the efficacy and safety of Nonacog beta pegol in previously treated male patients aged 13 to 70 years with moderate or severe haemophilia B.

Treatment and prophylaxis of bleeding in patients aged 12 years and over with haemophilia B (congenital factor IX deficiency)

• For the treatment and prophylaxis of bleeding in patients aged 12 years and over with haemophilia B (congenital factor IX 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.
• The pharmaceutical manufacturer has not submitted any relevant data for the assessment of the additional benefit of nonacog beta pegol.
• The G-BA considers that, taken as a whole, the results presented are not sufficient to allow patient-relevant effects to be inferred regarding the additional benefit of Nonacog beta pegol compared with the appropriate comparator therapy.
• Conclusion
  • The pharmaceutical manufacturer has not provided any relevant data for the assessment of the additional benefit of Nonacog beta pegol.
  • The G-BA considers that, taken as a whole, the results presented are not suitable for deriving patient-relevant effects regarding the additional benefit of Nonacog beta pegol compared with the appropriate comparator therapy.
  • The additional benefit compared with the appropriate comparator therapy is therefore not proven.

Courtesy translation only, please refer to the German original.