Marstacimab (2) – Hympavzi®

Severe haemophilia B, ≥ 12 years, without factor IX inhibitors

Characteristics

Start date 01.02.2025 – Marketing authorisation: 18.11.2024
Resolution 17.07.2025
INN Marstacimab
Brand name Hympavzi®
Pharm. company Pfizer Pharma GmbH
G-BA Procedure ID D-1153
ATC code B02BX11 Other systemic hemostatics (B02BX)
ICD-10 codes (AIS) D67Hereditary factor IX deficiency
Alpha-ID codes (AIS) I27821Hemophilia B
Therapeutic area Hematopoietic diseases Hemophilia (Hemophilia A /Hemophilia B)
Reason for procedure Initial assessment
Specialty Bundling

Therapeutic indication of the resolution

Hympavzi is used for the routine prophylaxis of bleeding episodes in patients aged 12 years and older with a body weight of at least 35 kg with severe haemophilia B (congenital factor IX deficiency, FIX < 1%) without factor inhibitors.
Subpopulation Indication Comparator
Adults and adolescents aged 12 years and older with severe haemophilia B (congenital factor IX deficiency, FIX ˂ 1%) without factor IX inhibitors for routine prophylaxis Routine prophylaxis with recombinant or human plasma-derived coagulation factor IX preparations

Studies and Results

No. of studies
(best subpopulation) 		1 (BASIS (B7841005)) 0 (Data not accepted)
Study design
(best subpopulation) 		Data not accepted (Dossier: Single-arm + other comparison)

  • Clinical trials
    • The pharmaceutical manufacturer presents the open-label, single-arm Phase III BASIS trial (B7841005) – an open-label, single-arm Phase III trial forming the basis for marketing authorisation – featuring an intra-individual before-and-after comparison, in which male patients aged between 12 and 74 years with severe haemophilia A (factor VIII activity < 1 per cent) or moderate to severe haemophilia B (factor IX activity ≤ 2 per cent) and a body weight of at least 35 kg.

Adults and adolescents aged 12 years and over with severe haemophilia B <UL><LI>(congenital factor IX deficiency, FIX ˂ 1 %)</LI></UL> without factor IX inhibitors for routine prophylaxis

  • The additional benefit is not proven.
  • Overall, based on the study presented, for adults and adolescents aged 12 years and over with severe haemophilia B (congenital factor IX deficiency, FIX ˂ 1 %) without factor IX inhibitors, for routine prophylaxis, no additional benefit of marstacimab can be inferred compared with the appropriate comparator therapy.
  • Overall assessment
    • In the overall assessment, for adults and adolescents aged 12 years and over with severe haemophilia B without factor IX inhibitors, the additional benefit of marstacimab compared with the appropriate comparator therapy for routine prophylaxis is not proven.

Courtesy translation only, please refer to the German original.

Associated procedures



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