01.05.2025
16.10.2025
Concizumab (2)
Alhemo®
Novo Nordisk GmbH
Hematopoietic diseases
Haemophilia B, ≥ 12 years, with factor IX inhibitors
2–12
57%
Hint for
considerable additional benefit
01.05.2025
16.10.2025
Concizumab
Alhemo®
Novo Nordisk GmbH
Hematopoietic diseases
Haemophilia A, ≥ 12 years, with factor VIII inhibitors
80–125
100%
no additional benefit
01.02.2025
17.07.2025
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
01.02.2025
17.07.2025
Marstacimab
Hympavzi®
Pfizer Pharma GmbH
Hematopoietic diseases
Severe haemophilia B, ≥ 12 years, without factor IX inhibitors
300–310
100%
no additional benefit
15.01.2025
03.07.2025
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
15.01.2025
03.07.2025
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
15.09.2024
06.03.2025
Crovalimab
Piasky®
Roche Pharma AG
Hematopoietic diseases
Paroxysmal nocturnal haemoglobinuria, ≥ 12 years, ≥ 40 kg
260–749
100%
no additional benefit
10.09.2024
20.02.2025
rADAMTS13
Adzynma®
Takeda GmbH
Hematopoietic diseases
ADAMTS13 deficiency in congenital thrombotic thrombocytopenic purpura (cTTP)
120–180
100%
Hint for
non-quantifiable additional benefit
Orphan
01.07.2024
19.12.2024
Iptacopan
Fabhalta®
Novartis Pharma GmbH
Hematopoietic diseases
Paroxysmal nocturnal hemoglobinuria
290–945
100%
Hint for
non-quantifiable additional benefit
Orphan
01.06.2024
22.11.2024
Pegcetacoplan (2)
Aspaveli®
Swedish Orphan Biovitrum GmbH
Hematopoietic diseases
Paroxysmal nocturnal hemoglobinuria, untreated patients
100–425
100%
Hint for
non-quantifiable additional benefit
Orphan
01.06.2024
22.11.2024
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
01.06.2024
22.11.2024
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
01.05.2024
17.10.2024
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)
15.07.2024
02.10.2024
Efanesoctocog alfa
Altuvoct®
Swedish Orphan Biovitrum GmbH
Hematopoietic diseases
Hemophilia A
0
100%
no additional benefit
Orphan
30.08.2023
15.02.2024
Nonacog beta pegol (2)
Refixia®
Novo Nordisk Pharma GmbH
Hematopoietic diseases
Hemophilia B, < 12 years
100–110
100%
no additional benefit
01.08.2023
01.02.2024
Eftrenonacog alfa (2, reassessment >€30m)
Alprolix®
Swedish Orphan Biovitrum GmbH
Hematopoietic diseases
Hemophilia B
560–720
100%
no additional benefit
Orphan (turnover limit)
15.05.2023
02.11.2023
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)
15.05.2023
02.11.2023
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)
01.05.2023
19.10.2023
Etranacogen Dezaparvovec
Hemgenix®
CSL Behring GmbH
Hematopoietic diseases
Hemophilia B
231–358
100%
Hint for
non-quantifiable additional benefit
Orphan
01.04.2023
21.09.2023
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)
01.03.2023
17.08.2023
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
02.01.2023
15.06.2023
Sutimlimab
Enjaymo®
Sanofi-Aventis Deutschland GmbH
Hematopoietic diseases
Cold agglutinin disease (CAD)
370–1,510
100%
Hint for
minor additional benefit
Orphan
15.09.2022
16.03.2023
Valoctocogene roxaparvovec
Roctavian®
BioMarin International Ltd.
Hematopoietic diseases
Hemophilia A
690–800
100%
Hint for
non-quantifiable additional benefit
Orphan
15.05.2022
03.11.2022
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
01.04.2022
15.09.2022
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
01.12.2021
19.05.2022
Mepolizumab (3)
Nucala®
GlaxoSmithKline GmbH & Co. KG
Hematopoietic diseases
Hypereosinophilic syndrome
100–400
100%
Hint for
considerable additional benefit
01.12.2021
19.05.2022
Lusutrombopag
Mulpleo®
Shionogi GmbH
Hematopoietic diseases
Thrombocytopenia in chronic liver disease
1,790–24,130
100%
no additional benefit
15.10.2021
07.04.2022
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)
01.10.2021
18.03.2022
Ravulizumab (3)
Ultomiris®
Alexion Pharma Germany GmbH
Hematopoietic diseases
Paroxysmal hemoglobinuria, pediatric patients
0–49
100%
no additional benefit
15.09.2021
03.03.2022
Roxadustat
Evrenzo®
Astellas Pharma GmbH
Hematopoietic diseases
Symptomatic anemia in chronic kidney disease (CKD)
151,000–195,000
100%
no additional benefit
01.04.2021
16.09.2021
Avatrombopag (2)
Doptelet®
Swedish Orphan Biovitrum GmbH
Hematopoietic diseases
Thrombocytopenia in chronic liver disease
1,790–24,130
100%
no additional benefit
01.04.2021
16.09.2021
Avatrombopag
Doptelet®
Swedish Orphan Biovitrum GmbH
Hematopoietic diseases
Immune thrombocytopenia (ITP)
4,260–10,830
100%
no additional benefit
01.12.2020
20.05.2021
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
01.08.2020
21.01.2021
Ravulizumab (2)
Ultomiris®
Alexion Pharma Germany GmbH
Hematopoietic diseases
Atypical hemolytic uremic syndrome (aHUS)
210–700
100%
no additional benefit
01.08.2020
21.01.2021
Luspatercept (2)
Reblozyl®
Celgene GmbH
Hematopoietic diseases
Beta thalassemia
0
170–300
100%
Hint for
non-quantifiable additional benefit
Orphan
01.08.2020
21.01.2021
Luspatercept
Reblozyl®
Celgene GmbH
Hematopoietic diseases
Myelodysplastic syndrome (MDS)
0
840–1,870
100%
Hint for
non-quantifiable additional benefit
Orphan
15.07.2020
07.01.2021
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
01.07.2020
17.12.2020
Fostamatinib
Tavlesse®
Grifols Deutschland GmbH
Hematopoietic diseases
Chronic immune thrombocytopenia
4,200–9,700
100%
no additional benefit
15.11.2019
14.05.2020
Betibeglogene autotemcel
Zynteglo®
bluebird bio (Germany) GmbH
Hematopoietic diseases
Beta thalassemia
50
100%
Hint for
non-quantifiable additional benefit
Orphan
01.09.2019
20.02.2020
Andexanet alfa
Ondexxya®
Portola Deutschland GmbH
Hematopoietic diseases
Antidote for reversal of anticoagulation (factor Xa)
4,200–27,600
100%
no additional benefit
01.08.2019
06.02.2020
Turoctocog alfa pegol
Esperoct®
Novo Nordisk Pharma GmbH
Hematopoietic diseases
Hemophilia A
2,840–3,190
100%
no additional benefit
01.08.2019
06.02.2020
Ravulizumab
Ultomiris®
Alexion Pharma Germany GmbH
Hematopoietic diseases
Paroxysmal hemoglobinuria
260–700
100%
no additional benefit
15.03.2019
05.09.2019
Emicizumab (2)
Hemlibra®
Roche Pharma AG
Hematopoietic diseases
Hemophilia A, without factor VIII inhibitors
2,000
100%
no additional benefit
01.01.2019
20.06.2019
Damoctocog alfa pegol
Jivi®
Bayer Vital GmbH
Hematopoietic diseases
Hemophilia A, ≥ 12 years
2,840–3,190
100%
no additional benefit
01.10.2018
22.03.2019
Caplacizumab
Cablivi®
Sanofi-Aventis Deutschland GmbH
Hematopoietic diseases
Thrombotic thrombocytopenic purpura (TTP)
150
100%
non-quantifiable additional benefit
Orphan
15.05.2018
01.11.2018
Rurioctocog alfa pegol
Adynovi®
Shire Deutschland GmbH
Hematopoietic diseases
Hemophilia A, ≥ 12 years
2,840–3,190
100%
no additional benefit
01.04.2018
20.09.2018
Emicizumab
Hemlibra®
Roche Pharma AG
Hematopoietic diseases
Hemophilia A
100
50%
Hint for
non-quantifiable additional benefit
01.11.2017
19.04.2018
Nonacog beta pegol
Refixia®
Novo Nordisk Pharma GmbH
Hematopoietic diseases
Hemophilia B, ≥ 12 years
500–570
100%
no additional benefit
01.02.2017
20.07.2017
Lonoctocog alfa
Afstyla®
CSL Behring GmbH
Hematopoietic diseases
Hemophilia A
3,190–3,590
100%
no additional benefit
15.06.2016
15.12.2016
Eftrenonacog alfa
Alprolix®
Swedish Orphan Biovitrum GmbH
Hematopoietic diseases
Hemophilia B
0
580–660
100%
non-quantifiable additional benefit
Orphan
01.06.2016
01.12.2016
Albutrepenonacog alfa
Idelvion®
CSL Behring GmbH
Hematopoietic diseases
Hemophilia B
0
580–660
100%
non-quantifiable additional benefit
Orphan
01.01.2016
16.06.2016
Efmoroctocog alfa
Elocta®
Swedish Orphan Biovitrum GmbH
Hematopoietic diseases
Hemophilia A
3,190–3,590
100%
no additional benefit
15.11.2014
07.05.2015
Simoctocog alfa
Nuwiq®
Octapharma GmbH
Hematopoietic diseases
Hemophilia A
3,190–3,585
100%
no additional benefit
15.01.2014
03.07.2014
Turoctocog alfa
NovoEight®
Novo Nordisk Pharma GmbH
Hematopoietic diseases
Hemophilia A
3,190–3,585
100%
no additional benefit