ALHEMO (concizumab) - Haemophilia A or B with FIX inhibitors and aged 12 years and over

The clinical benefit of ALHEMO (concizumab), solution for injection in pre-filled pen, is substantial in prophylaxis for the prevention of bleeding episodes in patients suffering from haemophilia B (congenital factor IX deficiency) with FIX inhibitors and aged 12 and over. The Committee has made the maintenance of the substantial clinical benefit conditional on its reassessment within a maximum period of three years, based on the final results of the EXPLORER 7 study assessing the longer-term efficacy and safety of ALHEMO (end of study planned for February 2027), the data from the PUT-RD (Therapeutic Use Protocol-Data Collection) and any new data available.

Considering:

• the evidence of the superiority of prophylaxis with concizumab alone versus no prophylaxis (on-demand treatment with bypassing agents) in reducing the number of bleeding episodes in 52 randomised adult and adolescent patients aged 12 years and over with haemophilia A or B with inhibitors;
• the sub-optimal quality of this demonstration, raising uncertainties about the observed benefit, which may be overestimated;
• the lack of robust comparative data relative to prophylaxis using bypass agents;
• uncertainties regarding the safety profile of concizumab, especially with regard to thromboembolic risk;
• the absence of long-term data;

The Committee considers that ALHEMO (concizumab), solution for injection in pre-filled pen, provides no clinical added value (CAV V) in the current care pathway for prophylaxis for the prevention of bleeding episodes in patients with haemophilia B (congenital factor IX deficiency) with FIX inhibitors and aged 12 years or more, which includes the relevant comparators (NOVOSEVEN (rFVIIa) and FEIBA (CCPa)).