Albutrepenonacog alfa (2) – Idelvion®

• Clinical trials
  • The pharmaceutical manufacturer presents the results of the open-label, uncontrolled, multicentre clinical studies CSL654_2004, CSL654_3001, CSL654_3002, CSL654_3003 and CSL654_5005 on albutrepenonacog alfa with the results from the open-label, uncontrolled, multicentre clinical studies on eftrenonacog alfa (B-LONG, Kids B-LONG, B-YOND).

Patients of all age groups with haemophilia B (congenital factor IX deficiency)

• For patients of all age groups with haemophilia B (congenital factor IX deficiency), additional benefit of albutrepenonacog alfa over the appropriate comparator therapy is not proven.
• Reasoning: For the assessment of the additional benefit of albutrepenonacog alfa in the treatment of patients of all age groups with haemophilia B (congenital factor IX deficiency) the pharmaceutical manufacturer did not submit a direct comparative study against the appropriate comparator therapy, but rather descriptive comparisons of the results from individual arms of various studies, as well as supplementary matching-adjusted indirectcomparisons(MAIC) of individual endpoints.
• The purely descriptive comparison of the results from individual arms of different studies is not suitable for deriving additional benefit.
• MAIC analyses without a bridge comparator do not, in principle, constitute an adequate means of adjusting for confounders.
• Consequently, the comparisons based on MAIC analyses submitted by the pharmaceutical manufacturer are not suitable for assessing the additional benefit of albutrepenonacog alfa.
• Overall, additional benefit for albutrepenonacog alfa compared with the appropriate comparator therapy is not proven for this patient population.
• Overall assessment
  • On balance, the additional benefit of albutrepenonacog alfa over the appropriate comparator therapy is not proven for this patient population.

Courtesy translation only, please refer to the German original.