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

Iptacopan (3) Fabhalta® Novartis Pharma GmbH Hematopoietic diseases Paroxysmal nocturnal haemoglobinuria 290–945 100% additional benefit not proven Orphan (turnover limit)
Concizumab (4) Alhemo® Novo Nordisk GmbH Hematopoietic diseases Haemophilia B, aged ≥ 12 years, without factor IX inhibitors 290–490 100% additional benefit not proven
Concizumab (3) Alhemo® Novo Nordisk GmbH Hematopoietic diseases Haemophilia A, aged ≥ 12 years, without factor VIII inhibitors 1,800–2,000 100% additional benefit not proven
Andexanet alfa (2) Ondexxya® AstraZeneca GmbH Hematopoietic diseases Repeal of anticoagulant therapy 9,900–32,600 50% additional benefit not proven
Concizumab (2) Alhemo® Novo Nordisk GmbH Hematopoietic diseases Haemophilia B, aged ≥ 12 years, with factor IX inhibitors 2–12 57% Hint for considerable additional benefit
Concizumab (1) Alhemo® Novo Nordisk GmbH Hematopoietic diseases Haemophilia A, aged ≥ 12 years, with factor VIII inhibitors 80–125 100% additional benefit not proven
Marstacimab (1) Hympavzi® Pfizer Pharma GmbH Hematopoietic diseases Severe haemophilia A, ≥ 12 years, without factor VIII inhibitors 1,900–2,000 100% additional benefit not proven
Marstacimab (2) Hympavzi® Pfizer Pharma GmbH Hematopoietic diseases Severe haemophilia B, ≥ 12 years, without factor IX inhibitors 300–310 100% additional benefit not proven
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 130–330 100% Hint for non-quantifiable additional benefit Orphan
Exagamglogen autotemcel (1) 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 (1) Piasky® Roche Pharma AG Hematopoietic diseases Paroxysmal nocturnal haemoglobinuria, ≥ 12 years, ≥ 40 kg 260–749 100% additional benefit not proven
Iptacopan (1) Fabhalta® Novartis Pharma GmbH Hematopoietic diseases Paroxysmal nocturnal hemoglobinuria 0
290–945
100% Hint for non-quantifiable additional benefit Orphan repealed
Danicopan (1) 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
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 (1) Vafseo® MEDICE Arzneimittel Pütter GmbH & Co. KG Hematopoietic diseases Symptomatic anemia in chronic kidney disease (CKD) 60,800–71,400 100% additional benefit not proven
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 (1) Altuvoct® Swedish Orphan Biovitrum GmbH Hematopoietic diseases Hemophilia A n.d. 100% additional benefit not proven Orphan
Nonacog beta pegol (2) Refixia® Novo Nordisk Pharma GmbH Hematopoietic diseases Hemophilia B, < 12 years 100–110 100% additional benefit not proven
Eftrenonacog alfa (2) Alprolix® Swedish Orphan Biovitrum GmbH Hematopoietic diseases Hemophilia B 560–720 100% additional benefit not proven Orphan (turnover limit)
Luspatercept (4) Reblozyl® Bristol-Myers Squibb GmbH & Co. KGaA Hematopoietic diseases Β-thalassaemia, transfusion-dependent anaemia 250–330 100% additional benefit not proven Orphan (turnover limit)
Luspatercept (5) Reblozyl® Bristol-Myers Squibb GmbH & Co. KGaA Hematopoietic diseases Myelodysplastic syndrome with transfusion-dependent anaemia, pre-treated 790–1,860 100% additional benefit not proven Orphan (turnover limit)
Etranacogen Dezaparvovec (1) 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% additional benefit not proven
Sutimlimab (1) Enjaymo® Sanofi-Aventis Deutschland GmbH Hematopoietic diseases Cold agglutinin disease (CAD) 370–1,510 100% Hint for minor additional benefit Orphan
Valoctocogen Roxaparvovec (1) Roctavian® BioMarin International Ltd. Hematopoietic diseases Hemophilia A 690–800 100% Hint for non-quantifiable additional benefit Orphan
Voxelotor (1) 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 (1) Aspaveli® Swedish Orphan Biovitrum GmbH Hematopoietic diseases Paroxysmal nocturnal haemoglobinuria (PNH), pre-treated patients 190–520 100% Hint for non-quantifiable additional benefit Orphan
Lusutrombopag (1) Mulpleo® Shionogi GmbH Hematopoietic diseases Thrombocytopenia in chronic liver disease 1,790–24,130 100% additional benefit not proven
Mepolizumab (5) Nucala® GlaxoSmithKline GmbH & Co. KG Hematopoietic diseases Hypereosinophilic syndrome 100–400 100% Hint for considerable additional benefit
Albutrepenonacog alfa (2) Idelvion® CSL Behring GmbH Hematopoietic diseases Hemophilia B, congenital factor IX deficiency 560–720 100% additional benefit not proven Orphan (turnover limit)
Ravulizumab (3) Ultomiris® Alexion Pharma Germany GmbH Hematopoietic diseases Paroxysmal hemoglobinuria, pediatric patients 0–49 100% additional benefit not proven
Roxadustat (1) Evrenzo® Astellas Pharma GmbH Hematopoietic diseases Symptomatic anemia in chronic kidney disease (CKD) 151,000–195,000 100% additional benefit not proven
Avatrombopag (1) Doptelet® Swedish Orphan Biovitrum GmbH Hematopoietic diseases Thrombocytopenia in chronic liver disease 1,790–24,130 100% additional benefit not proven
Avatrombopag (2) Doptelet® Swedish Orphan Biovitrum GmbH Hematopoietic diseases Immune thrombocytopenia (ITP) 4,260–10,830 100% additional benefit not proven
Crizanlizumab (1) 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 repealed
Ravulizumab (2) Ultomiris® Alexion Pharma Germany GmbH Hematopoietic diseases Atypical hemolytic uremic syndrome (aHUS) 210–700 100% additional benefit not proven
Luspatercept (1) Reblozyl® Celgene GmbH Hematopoietic diseases Beta thalassemia 0
170–300
100% Hint for non-quantifiable additional benefit Orphan repealed
Luspatercept (2) Reblozyl® Celgene GmbH Hematopoietic diseases Myelodysplastic syndrome (MDS) 0
840–1,870
100% Hint for non-quantifiable additional benefit Orphan repealed
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 (1) Tavlesse® Grifols Deutschland GmbH Hematopoietic diseases Chronic immune thrombocytopenia 4,200–9,700 100% additional benefit not proven
Betibeglogene Autotemcel (1) Zynteglo® Bluebird bio (Germany) GmbH Hematopoietic diseases Beta thalassemia 50 100% Hint for non-quantifiable additional benefit Orphan
Andexanet alfa (1) Ondexxya® Portola Deutschland GmbH Hematopoietic diseases Antidote for reversal of anticoagulation (factor Xa) 0
4,200–27,600
100% additional benefit not proven repealed
Turoctocog alfa pegol (1) Esperoct® Novo Nordisk Pharma GmbH Hematopoietic diseases Hemophilia A 2,840–3,190 100% additional benefit not proven
Ravulizumab (1) Ultomiris® Alexion Pharma Germany GmbH Hematopoietic diseases Paroxysmal hemoglobinuria 260–700 100% additional benefit not proven
Emicizumab (2) Hemlibra® Roche Pharma AG Hematopoietic diseases Hemophilia A, without factor VIII inhibitors 2,000 100% additional benefit not proven
Damoctocog alfa pegol (1) Jivi® Bayer Vital GmbH Hematopoietic diseases Hemophilia A, ≥ 12 years 2,840–3,190 100% additional benefit not proven
Caplacizumab (1) Cablivi® Sanofi-Aventis Deutschland GmbH Hematopoietic diseases Thrombotic thrombocytopenic purpura (TTP) 150 100% non-quantifiable additional benefit Orphan
Rurioctocog alfa pegol (1) Adynovi® Shire Deutschland GmbH Hematopoietic diseases Hemophilia A, ≥ 12 years 2,840–3,190 100% additional benefit not proven
Emicizumab (1) Hemlibra® Roche Pharma AG Hematopoietic diseases Hemophilia A 100 50% Hint for non-quantifiable additional benefit
Nonacog beta pegol (1) Refixia® Novo Nordisk Pharma GmbH Hematopoietic diseases Hemophilia B, ≥ 12 years 500–570 100% additional benefit not proven
Lonoctocog alfa (1) Afstyla® CSL Behring GmbH Hematopoietic diseases Hemophilia A 3,190–3,590 100% additional benefit not proven
Eftrenonacog alfa (1) Alprolix® Swedish Orphan Biovitrum GmbH Hematopoietic diseases Hemophilia B 0
580–660
100% non-quantifiable additional benefit Orphan repealed
Albutrepenonacog alfa (1) Idelvion® CSL Behring GmbH Hematopoietic diseases Hemophilia B 0
580–660
100% non-quantifiable additional benefit Orphan repealed
Ticagrelor (2) Brilique® AstraZeneca GmbH Hematopoietic diseases Prevention of atherothrombotic events in patients with myocardial infarction and high risk for an atherothrombotic event 105,000 100% Hint for minor additional benefit
Efmoroctocog alfa (1) Elocta® Swedish Orphan Biovitrum GmbH Hematopoietic diseases Hemophilia A 3,190–3,590 100% additional benefit not proven
Edoxaban (1) Lixiana® Daiichi Sankyo Deutschland GmbH Hematopoietic diseases Prophylaxis of thromboembolic events 1,167,000–1,334,000 81% Indication of minor additional benefit
Simoctocog alfa (1) Nuwiq® Octapharma GmbH Hematopoietic diseases Hemophilia A 3,190–3,585 100% additional benefit not proven
Apixaban (3) Eliquis® Bristol-Myers Squibb GmbH & Co. KGaA / Pfizer Deutschland GmbH Hematopoietic diseases Treatment and prophylaxis of venous thrombosis and pulmonary embolism 241,000 47% Indication of minor additional benefit
Turoctocog alfa (1) NovoEight® Novo Nordisk Pharma GmbH Hematopoietic diseases Hemophilia A 3,190–3,585 100% additional benefit not proven
Apixaban (2) Eliquis® Bristol-Myers Squibb GmbH & Co. KGaA / Pfizer Deutschland GmbH Hematopoietic diseases Prophylaxis of strokes and systemic embolisms 926,000–1,093,000 100% Indication of minor additional benefit
Apixaban (1) Eliquis® Bristol-Myers Squibb GmbH & Co. KGaA / Pfizer Deutschland GmbH Hematopoietic diseases Prophylaxis of venous thromboembolism 390,000 58% Indication of minor additional benefit