Nonacog beta pegol (2) – Refixia®

• Clinical trials
  • In addition, the pharmaceutical manufacturer has submitted the single-arm studies forming the basis for marketing authorisation: Paradigm 5 (previously treated male patients aged ≤ 12 years) and Paradigm 6 (previously untreated male patients aged < 6 years) involving patients with severe haemophilia B (≤ 2 % Factor IX activity).

Patients aged < 12 years with haemophilia B

• For patients aged < 12 years with haemophilia B, the additional benefit of nonacog beta pegol compared with the appropriate comparator therapy is not proven.
• In its dossier for the assessment of the additional benefit of Nonacog beta pegol, the pharmaceutical manufacturer does not present any direct comparative studies against the appropriate comparator therapy.
• The single-arm studies submitted are not suitable for assessing additional benefit due to the lack of comparison with the appropriate comparator therapy.
• Overall, on the basis of the studies submitted, no additional benefit can be inferred for patients aged < 12 years with haemophilia B compared with the appropriate comparator therapy.
• Overall assessment
  • In the overall review, the additional benefit of Nonacog beta pegol compared with the appropriate comparator therapy is not proven for patients aged < 12 years with haemophilia B.

Courtesy translation only, please refer to the German original.