Hematopoietic diseases

Substitute preparations for blood coagulation disorders (hemophilia, TTP, antidote, paroxysmal nocturnal hemoglobinuria (PNH)) belong to this category. To date, the G-BA has assessed xx such products in xx procedures. In the xx observed subpopulations, xx% have seen a major, XX% a considerable and XX% a minor additional benefit in relation to the comparative therapy. A non-quantifiable additional benefit has been proved for XX% of the subpopulations.

By contrast, no additional benefit has been proved for XX% of the subpopulations weighted by patient share per resolution. In total, this includes xx% of the xx million patients relevant for treatment with the assessed medicinal products.

All G-BA resolutions concerning hematopoietic diseases

Concizumab (2) Alhemo® Novo Nordisk GmbH Hematopoietic diseases Haemophilia B, ≥ 12 years, with factor IX inhibitors 2–12 57% Hint for considerable additional benefit
Concizumab Alhemo® Novo Nordisk GmbH Hematopoietic diseases Haemophilia A, ≥ 12 years, with factor VIII inhibitors 80–125 100% no additional benefit
Marstacimab (2) Hympavzi® Pfizer Pharma GmbH Hematopoietic diseases Severe haemophilia A, ≥ 12 years, without factor VIII inhibitors 1,900–2,000 100% no additional benefit
Marstacimab Hympavzi® Pfizer Pharma GmbH Hematopoietic diseases Severe haemophilia B, ≥ 12 years, without factor IX inhibitors 300–310 100% no additional benefit
Exagamglogen autotemcel (2) Casgevy® Vertex Pharmaceuticals (Germany) GmbH Hematopoietic diseases Sickle cell disease with recurrent vaso-occlusive crises; ≥ 12 years; no HLA-matched related stem cell donation available 130–1,230 100% Hint for non-quantifiable additional benefit Orphan
Exagamglogen autotemcel Casgevy® Vertex Pharmaceuticals (Germany) GmbH Hematopoietic diseases Β-thalassaemia, transfusion-dependent, ≥ 12 years, no HLA-compatible related stem cell donation available 20–150 100% Indication of non-quantifiable additional benefit Orphan
Crovalimab Piasky® Roche Pharma AG Hematopoietic diseases Paroxysmal nocturnal haemoglobinuria, ≥ 12 years, ≥ 40 kg 260–749 100% no additional benefit
rADAMTS13 Adzynma® Takeda GmbH Hematopoietic diseases ADAMTS13 deficiency in congenital thrombotic thrombocytopenic purpura (cTTP) 120–180 100% Hint for non-quantifiable additional benefit Orphan
Iptacopan Fabhalta® Novartis Pharma GmbH Hematopoietic diseases Paroxysmal nocturnal hemoglobinuria 290–945 100% Hint for non-quantifiable additional benefit Orphan
Pegcetacoplan (2) Aspaveli® Swedish Orphan Biovitrum GmbH Hematopoietic diseases Paroxysmal nocturnal hemoglobinuria, untreated patients 100–425 100% Hint for non-quantifiable additional benefit Orphan
Vadadustat Vafseo® MEDICE Arzneimittel Pütter GmbH & Co. KG Hematopoietic diseases Symptomatic anemia in chronic kidney disease (CKD) 60,800–71,400 100% no additional benefit
Danicopan Voydeya® Alexion Pharma Germany GmbH Hematopoietic diseases Paroxysmal hemoglobinuria in residual hemolytic anemia, add-on therapy to ravulizumab or eculizumab) 70–350 100% Hint for non-quantifiable additional benefit Orphan
Luspatercept (6) Reblozyl® Bristol-Myers Squibb GmbH & Co. KGaA Hematopoietic diseases Myelodysplastic syndromes with transfusion-dependent anemia, not pretreated, and without ring sideroblasts, pretreated 4,960–7,080 80% Hint for minor additional benefit Orphan (turnover limit)
Efanesoctocog alfa Altuvoct® Swedish Orphan Biovitrum GmbH Hematopoietic diseases Hemophilia A 0 100% no additional benefit Orphan
Nonacog beta pegol (2) Refixia® Novo Nordisk Pharma GmbH Hematopoietic diseases Hemophilia B, < 12 years 100–110 100% no additional benefit
Eftrenonacog alfa (2, reassessment >€30m) Alprolix® Swedish Orphan Biovitrum GmbH Hematopoietic diseases Hemophilia B 560–720 100% no additional benefit Orphan (turnover limit)
Luspatercept (5, reassessment >€30m) Reblozyl® Bristol-Myers Squibb GmbH & Co. KGaA Hematopoietic diseases Β-thalassaemia, transfusion-dependent anaemia 250–330 100% no additional benefit Orphan (turnover limit)
Luspatercept (4, reassessment >€30m) Reblozyl® Bristol-Myers Squibb GmbH & Co. KGaA Hematopoietic diseases Myelodysplastic syndrome with transfusion-dependent anaemia, pre-treated 790–1,860 100% no additional benefit Orphan (turnover limit)
Etranacogen Dezaparvovec Hemgenix® CSL Behring GmbH Hematopoietic diseases Hemophilia B 231–358 100% Hint for non-quantifiable additional benefit Orphan
Luspatercept (3) Reblozyl® Bristol-Myers Squibb GmbH & Co. KGaA Hematopoietic diseases Β-thalassaemia, non-transfusion-dependent anaemia 470–560 100% Indication of minor additional benefit Orphan (turnover limit)
Emicizumab (3) Hemlibra® Roche Pharma AG Hematopoietic diseases Moderate haemophilia A, without factor VIII inhibitors, with severe bleeding phenotype 220–240 100% no additional benefit
Sutimlimab Enjaymo® Sanofi-Aventis Deutschland GmbH Hematopoietic diseases Cold agglutinin disease (CAD) 370–1,510 100% Hint for minor additional benefit Orphan
Valoctocogene roxaparvovec Roctavian® BioMarin International Ltd. Hematopoietic diseases Hemophilia A 690–800 100% Hint for non-quantifiable additional benefit Orphan
Voxelotor Oxbryta® Global Blood Therapeutics Germany GmbH Hematopoietic diseases Haemolytic anaemia in sickle cell disease, monotherapy or combination with hydroxycarbamide, ≥ 12 years 1,590–2,580 100% Hint for non-quantifiable additional benefit Orphan
Pegcetacoplan Aspaveli® Swedish Orphan Biovitrum GmbH Hematopoietic diseases Paroxysmal nocturnal haemoglobinuria (PNH), pre-treated patients 190–520 100% Hint for non-quantifiable additional benefit Orphan
Mepolizumab (3) Nucala® GlaxoSmithKline GmbH & Co. KG Hematopoietic diseases Hypereosinophilic syndrome 100–400 100% Hint for considerable additional benefit
Lusutrombopag Mulpleo® Shionogi GmbH Hematopoietic diseases Thrombocytopenia in chronic liver disease 1,790–24,130 100% no additional benefit
Albutrepenonacog alfa (2, reassessment >€50m) Idelvion® CSL Behring GmbH Hematopoietic diseases Hemophilia B, congenital factor IX deficiency 560–720 100% no additional benefit Orphan (turnover limit)
Ravulizumab (3) Ultomiris® Alexion Pharma Germany GmbH Hematopoietic diseases Paroxysmal hemoglobinuria, pediatric patients 0–49 100% no additional benefit
Roxadustat Evrenzo® Astellas Pharma GmbH Hematopoietic diseases Symptomatic anemia in chronic kidney disease (CKD) 151,000–195,000 100% no additional benefit
Avatrombopag (2) Doptelet® Swedish Orphan Biovitrum GmbH Hematopoietic diseases Thrombocytopenia in chronic liver disease 1,790–24,130 100% no additional benefit
Avatrombopag Doptelet® Swedish Orphan Biovitrum GmbH Hematopoietic diseases Immune thrombocytopenia (ITP) 4,260–10,830 100% no additional benefit
Crizanlizumab Adakveo® Novartis Pharma GmbH Hematopoietic diseases Prevention of recurrent vasoocclusive crises in sickle cell anaemia, ≥16 years 0
390–1,690
100% Hint for minor additional benefit Orphan
Ravulizumab (2) Ultomiris® Alexion Pharma Germany GmbH Hematopoietic diseases Atypical hemolytic uremic syndrome (aHUS) 210–700 100% no additional benefit
Luspatercept (2) Reblozyl® Celgene GmbH Hematopoietic diseases Beta thalassemia 0
170–300
100% Hint for non-quantifiable additional benefit Orphan
Luspatercept Reblozyl® Celgene GmbH Hematopoietic diseases Myelodysplastic syndrome (MDS) 0
840–1,870
100% Hint for non-quantifiable additional benefit Orphan
Caplacizumab (2) Cablivi® Sanofi-Aventis Deutschland GmbH Hematopoietic diseases Thrombotic thrombocytopenic purpura (aTTP)Thrombotic thrombocytopenic purpura (TTP), 12 to < 18 years 2–3 100% Hint for non-quantifiable additional benefit Orphan
Fostamatinib Tavlesse® Grifols Deutschland GmbH Hematopoietic diseases Chronic immune thrombocytopenia 4,200–9,700 100% no additional benefit
Betibeglogene autotemcel Zynteglo® bluebird bio (Germany) GmbH Hematopoietic diseases Beta thalassemia 50 100% Hint for non-quantifiable additional benefit Orphan
Andexanet alfa Ondexxya® Portola Deutschland GmbH Hematopoietic diseases Antidote for reversal of anticoagulation (factor Xa) 4,200–27,600 100% no additional benefit
Turoctocog alfa pegol Esperoct® Novo Nordisk Pharma GmbH Hematopoietic diseases Hemophilia A 2,840–3,190 100% no additional benefit
Ravulizumab Ultomiris® Alexion Pharma Germany GmbH Hematopoietic diseases Paroxysmal hemoglobinuria 260–700 100% no additional benefit
Emicizumab (2) Hemlibra® Roche Pharma AG Hematopoietic diseases Hemophilia A, without factor VIII inhibitors 2,000 100% no additional benefit
Damoctocog alfa pegol Jivi® Bayer Vital GmbH Hematopoietic diseases Hemophilia A, ≥ 12 years 2,840–3,190 100% no additional benefit
Caplacizumab Cablivi® Sanofi-Aventis Deutschland GmbH Hematopoietic diseases Thrombotic thrombocytopenic purpura (TTP) 150 100% non-quantifiable additional benefit Orphan
Rurioctocog alfa pegol Adynovi® Shire Deutschland GmbH Hematopoietic diseases Hemophilia A, ≥ 12 years 2,840–3,190 100% no additional benefit
Emicizumab Hemlibra® Roche Pharma AG Hematopoietic diseases Hemophilia A 100 50% Hint for non-quantifiable additional benefit
Nonacog beta pegol Refixia® Novo Nordisk Pharma GmbH Hematopoietic diseases Hemophilia B, ≥ 12 years 500–570 100% no additional benefit
Lonoctocog alfa Afstyla® CSL Behring GmbH Hematopoietic diseases Hemophilia A 3,190–3,590 100% no additional benefit
Eftrenonacog alfa Alprolix® Swedish Orphan Biovitrum GmbH Hematopoietic diseases Hemophilia B 0
580–660
100% non-quantifiable additional benefit Orphan
Albutrepenonacog alfa Idelvion® CSL Behring GmbH Hematopoietic diseases Hemophilia B 0
580–660
100% non-quantifiable additional benefit Orphan
Efmoroctocog alfa Elocta® Swedish Orphan Biovitrum GmbH Hematopoietic diseases Hemophilia A 3,190–3,590 100% no additional benefit
Simoctocog alfa Nuwiq® Octapharma GmbH Hematopoietic diseases Hemophilia A 3,190–3,585 100% no additional benefit
Turoctocog alfa NovoEight® Novo Nordisk Pharma GmbH Hematopoietic diseases Hemophilia A 3,190–3,585 100% no additional benefit