Eftrenonacog alfa (2) – Alprolix®

• Clinical trials
  • In support of this, the pharmaceutical manufacturer cites the four single-arm, non-randomised, uncontrolled clinical studies B-LONG, Kids B-LONG, PUPs B-LONG and B YOND, involving male patients with severe haemophilia B (defined as ≤ 2 IU/dl endogenous factor IX activity).

Patients of all age groups with haemophilia B

• For patients of all age groups with haemophilia B, the additional benefit of eftrenonacog alfa compared with the appropriate comparator therapy is not proven.
• In its dossier for the assessment of the additional benefit of eftrenonacog alfa, 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 compared with the appropriate comparator therapy can be inferred for patients of all age groups with haemophilia B.
• Overall assessment
  • In summary, for patients of all age groups with haemophilia B, the additional benefit of eftrenonacog alfa compared with the appropriate comparator therapy is not proven.

Courtesy translation only, please refer to the German original.