Concizumab (4) – Alhemo®

Haemophilia B, aged ≥ 12 years, without factor IX inhibitors

Characteristics

Start date 01.10.2025 – Marketing authorisation: 22.08.2025
Resolution 19.03.2026
INN Concizumab
Brand name Alhemo®
Pharm. company Novo Nordisk GmbH
G-BA Procedure ID D-1238
ATC code B02BX10 Other systemic hemostatics (B02BX)
ICD-10 codes (AIS) D67Hereditary factor IX deficiency
Alpha-ID codes (AIS) I27821Hemophilia B
Therapeutic area Hematopoietic diseases
Reason for procedure New therapeutic indication

Therapeutic indication of the resolution

Concizumab is used for the routine prophylaxis of bleeding in patients aged 12 years and over with moderate/severe haemophilia B (congenital factor IX deficiency; FIX ≤ 2 per cent) without FIX inhibitors.
Subpopulation Indication Comparator
Erwachsene und Jugendliche ab 12 Jahren mit schwerer und mittelschwerer Hämophilie B (FIX ≤ 2 %) ohne Faktor-IX-Inhibitoren mit Indikation zur Routineprophylaxe

Studies and Results

  • Clinical trials
    • The pharmaceutical manufacturer presents the multicentre, partially randomised, open-label Explorer8 trial, which formed the basis for marketing authorisation. This trial compared routine prophylaxis with concizumab against on-demand treatment with factor preparations, and included male patients aged 12 years and over with congenital severe haemophilia A (FVIII < 1 per cent) or moderate/severe haemophilia B (FIX ≤ 2 per cent) without factor VIII or factor IX inhibitors.

Adults and adolescents aged 12 years and over with severe and moderate haemophilia B (FIX ≤ 2 %) without factor IX inhibitors, with an indication for routine prophylaxis

  • For adults and adolescents aged 12 years and over with severe and moderate haemophilia B (FIX ≤ 2 %) without factor IX inhibitors, with an indication for routine prophylaxis, an additional benefit is not proven.
  • In its dossier for the assessment of the additional benefit of Concizumab, the pharmaceutical manufacturer does not present any direct comparative studies against the appropriate comparator therapy.
  • As it lacks a comparison with the appropriate comparator therapy, the study submitted is not suitable for assessing additional benefit.
  • Overall, for adults and adolescents aged 12 years and over with severe and moderate haemophilia B (FIX ≤ 2 per cent) without factor IX inhibitors, who have an indication for routine prophylaxis, the additional benefit of Concizumab compared with the appropriate comparator therapy is not proven.

Courtesy translation only, please refer to the German original.

Associated procedures



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