01.05.2025
16.10.2025
Ivacaftor / Tezacaftor / Elexacaftor (17)
Kaftrio®
Vertex Pharmaceuticals (Germany) GmbH
Metabolic diseases
Cystic fibrosis, combination treatment with ivacaftor, ≥ 2 years, non-class I mutation (no F508del mutation and no gating mutation)
375
64%
Hint for
major additional benefit
Orphan (turnover limit)
01.05.2025
16.10.2025
Ivacaftor / Tezacaftor / Elexacaftor (16)
Kaftrio®
Vertex Pharmaceuticals (Germany) GmbH
Metabolic diseases
Cystic fibrosis, combination treatment with ivacaftor, ≥ 2 years, non-class I mutation (one gating mutation and no F508del mutation)
100
100%
no additional benefit
Orphan (turnover limit)
01.05.2025
16.10.2025
Tiratricol
Emcitate®
Rare Thyroid Therapeutics International AB
Metabolic diseases
Peripheral thyrotoxicosis in monocarboxylate transporter deficiency 8
40–110
100%
Hint for
non-quantifiable additional benefit
Orphan
01.05.2025
16.10.2025
Serplulimab
Hetronifly®
Accord Healthcare GmbH
Oncological diseases
Small cell lung carcinoma, in combination with carboplatin and etoposide, first-line
3,210–7,719
100%
Hint for
non-quantifiable additional benefit
Orphan
15.04.2025
02.10.2025
Lisocabtagen maraleucel (3)
Breyanzi®
Bristol-Myers Squibb GmbH & Co. KGaA
Oncological diseases
Follicular lymphoma, after ≥ 2 prior therapies
370–840
100%
no additional benefit
Orphan
15.03.2025
04.09.2025
Seladelpar
Lyvdelzi®
Gilead Sciences GmbH
Digestive system diseases
Primary biliary cholangitis (combination with ursodeoxycholic acid)
6,000–13,000
100%
Indication of
minor additional benefit
Orphan
15.03.2025
04.09.2025
Fosdenopterin
Nulibry®
Sentynl Therapeutics
Nervous system diseases
Molybdenum cofactor deficiency type A
2
100%
Hint for
non-quantifiable additional benefit
Orphan
01.03.2025
21.08.2025
Blinatumomab (9)
Blincyto®
Amgen GmbH
Oncological diseases
Acute lymphoblastic B-cell leukaemia, high-risk first relapse, Ph-, CD19+, ≥1 month and <1 year
1
100%
Hint for
non-quantifiable additional benefit
Orphan
01.03.2025
21.08.2025
Blinatumomab (8)
Blincyto®
Amgen GmbH
Oncological diseases
Acute lymphoblastic B-cell leukaemia, Ph-, CD19+, newly diagnosed
160–270
100%
Hint for
considerable additional benefit
Orphan
01.03.2025
21.08.2025
Blinatumomab (7)
Blincyto®
Amgen GmbH
Oncological diseases
Acute lymphoblastic B-cell leukaemia, relapsed/refractory, ≥ 1 month to < 1 year, after ≥ 2 prior therapies or after allogeneic stem cell transplantation
1
100%
Hint for
non-quantifiable additional benefit
Orphan
01.03.2025
21.08.2025
Daratumumab (12, reassessment)
Darzalex®
Janssen-Cilag GmbH
Oncological diseases
Systemic light-chain amyloidosis, first-line, combination with cyclophosphamide, bortezomib and dexamethasone
220–515
100%
Hint for
considerable additional benefit
Orphan (turnover limit)
01.03.2025
21.08.2025
Fedratinib (2, reassessment)
Inrebic®
Bristol-Myers Squibb GmbH
Oncological diseases
Myelofibrosis
640–1,710
100%
Hint for
non-quantifiable additional benefit
Orphan
01.03.2025
21.08.2025
Setmelanotid (3)
Imcivree®
Rhythm Pharmaceuticals Inc
Metabolic diseases
Obesity and control of hunger, POMC, PCSK1, LEPR deficiency or Bardet-Biedl syndrome, ≥ 2 to < 6 years of age
24–67
100%
Hint for
non-quantifiable additional benefit
Orphan
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
01.01.2025
18.06.2025
Eliglustat (2)
Cerdelga®
Sanofi-Aventis Deutschland GmbH
Metabolic diseases
Gaucher disease type 1, ≥ 6 to < 18 years, ≥ 15 kg bw
10–30
100%
Hint for
non-quantifiable additional benefit
Orphan
15.12.2024
05.06.2025
Mirvetuximab Soravtansin
Elahere®
AbbVie Deutschland GmbH
Oncological diseases
Ovarian carcinoma, fallopian tube carcinoma or primary peritoneal carcinoma, FRα-positive, platinum-resistant, after 1 to 3 prior therapies
630–1,300
100%
Indication of
considerable additional benefit
Orphan
01.12.2024
15.05.2025
Daratumumab (11)
Darzalex®
Janssen-Cilag GmbH
Oncological diseases
Multiple myeloma, first-line, stem cell transplantation suitable, combination with bortezomib, lenalidomide and dexamethasone
1,750–1,910
100%
no additional benefit
Orphan (turnover limit)
01.12.2024
15.05.2025
Ciltacabtagen autoleucel (2, reassessment >€30m)
Carvykti®
Janssen-Cilag GmbH
Oncological diseases
Multiple myeloma, after at least 1 prior therapy, refractory to lenalidomide
2,280–3,640
62%
Hint for
considerable additional benefit
Orphan (turnover limit)
01.11.2024
17.04.2025
Artesunat
Artesunate Amivas®
Amivas Ireland Ltd
Infectious diseases
Severe malaria, from birth
87
100%
non-quantifiable additional benefit
Orphan
01.11.2024
17.04.2025
Zolbetuximab
Vyloy®
Astellas Pharma GmbH
Oncological diseases
Adenocarcinoma of the stomach or gastro-oesophageal junction, HER2-negative, first-line, combination with fluoropyrimidine- and platinum-containing chemotherapy
250–1,310
100%
Indication of
minor additional benefit
Orphan
15.10.2024
03.04.2025
Elafibranor
Iqirvo®
Ipsen Pharma GmbH
Digestive system diseases
Primary biliary cholangitis (combination with ursodeoxycholic acid)
6,000–13,000
100%
Hint for
minor additional benefit
Orphan
01.10.2024
20.03.2025
Polihexanid
Akantior®
SIFI S.p.A.
Eye diseases
Acanthamoebic keratitis; ≥ 12 years of age
70–410
100%
Hint for
non-quantifiable additional benefit
Orphan
01.10.2024
20.03.2025
Isavuconazol (2)
Cresemba®
Pfizer Pharma GmbH
Infectious diseases
Mucormycosis, ≥ 1 to ≤ 17 years
8
100%
Hint for
non-quantifiable additional benefit
Orphan
01.10.2024
20.03.2025
Isavuconazol (3)
Cresemba®
Pfizer Pharma GmbH
Infectious diseases
Aspergillosis, ≥ 1 to ≤ 17 years
40–215
100%
Hint for
non-quantifiable additional benefit
Orphan
15.09.2024
06.03.2025
Sotatercept
Winrevair®
MSD Sharp & Dohme GmbH
Cardiovascular diseases
Pulmonary arterial hypertension
580–7,850
100%
Hint for
minor additional benefit
Orphan
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
01.08.2024
06.02.2025
Sparsentan
Filspari®
Vifor Pharma Deutschland GmbH
Genitourinary system diseases
Immunoglobulin A - Nephropathy, primary
900–13,000
100%
Hint for
minor additional benefit
Orphan
01.08.2024
06.02.2025
Maralixibat (2)
Livmarli®
Mirum Pharmaceuticals International B.V.
Digestive system diseases
Progressive familial intrahepatic cholestasis (PFIC), ≥ 3 months
80–180
100%
Hint for
non-quantifiable additional benefit
Orphan
01.07.2024
19.12.2024
Axicabtagen-Ciloleucel (7, reassessment)
Yescarta®
Gilead Sciences GmbH
Oncological diseases
Diffuse large B-cell lymphoma, highly malignant B-cell lymphoma, after 1 prior therapy, relapse within 12 months or refractory
800–1,130
100%
Hint for
minor additional benefit
Orphan (turnover limit)
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
01.07.2024
19.12.2024
Tofersen
Qalsody®
Biogen GmbH
Nervous system diseases
Amyotrophic lateral sclerosis (ALS)
90–170
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
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
Dabrafenib (5, Finlee®)
Finlee®
Novartis Pharma GmbH
Oncological diseases
Malignant glioma, BRAF V600E mutation, ≥ 1 year, low-grade (LGG) first-line/higher-grade (HGG) after at least 1 prior therapy; combination with trametinib
7–115
41%
Hint for
considerable additional benefit
Orphan
01.05.2024
17.10.2024
Trametinib (4, Spexotras®)
Spexotras®
Novartis Pharma GmbH
Oncological diseases
Malignant glioma, BRAF V600E mutation, ≥ 1 year, low-grade (LGG) first-line/higher-grade (HGG) after at least 1 prior therapy; combination with dabrafenib
7–115
41%
Hint for
considerable 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
01.04.2024
19.09.2024
Efgartigimod alfa (2, reassessment >€30m)
Vyvgart®
Argenx Germany GmbH
Nervous system diseases
Myasthenia gravis, AChR antibodies+
6,300–19,000
100%
no additional benefit
Orphan (turnover limit)
01.04.2024
19.09.2024
Idecabtagen vicleucel (3)
Abecma®
Bristol-Myers Squibb GmbH & Co. KGaA
Oncological diseases
Multiple myeloma, at least 2 prior therapies
4,900–5,250
100%
no additional benefit
Orphan (turnover limit)
15.03.2024
19.09.2024
Omaveloxolon
Skyclarys®
Biogen GmbH
Nervous system diseases
Friedreich's ataxia, ≥ 16 years
970
100%
Hint for
non-quantifiable additional benefit
Orphan
01.03.2024
15.08.2024
Rozanolixizumab
Rystiggo®
UCB Pharma GmbH
Nervous system diseases
Myasthenia gravis, AChR antibodies+, MuSK antibodies+
6,470–19,300
98%
Hint for
considerable additional benefit
Orphan
15.02.2024
15.08.2024
Momelotinib
Omjjara®
GlaxoSmithKline GmbH & Co. KG
Oncological diseases
Myelofibrosis
210–1,160
50%
Hint for
minor additional benefit
Orphan
01.02.2024
01.08.2024
Rezafungin
Rezzayo®
Mundipharma
Infectious diseases
Invasive candida infections
31,800–34,600
100%
Hint for
non-quantifiable additional benefit
Orphan
15.01.2024
04.07.2024
Pegzilarginase
Loargys®
Immedica Pharma Germany GmbH
Metabolic diseases
Hyperargininemia (ARG1-D), ≥ 2 years
50
100%
Hint for
non-quantifiable additional benefit
Orphan
15.01.2024
04.07.2024
Vamorolon
Agamree®
Santhera Pharmaceuticals GmbH
Musculoskeletal system diseases
Duchenne muscular dystrophy, ≥ 4 years
740–3,670
100%
Hint for
non-quantifiable additional benefit
Orphan
01.01.2024
20.06.2024
Palopegteriparatid
Yorvipath®
Ascendis Pharma Endocrinology GmbH
Metabolic diseases
Chronic hypoparathyroidism, parathyroid hormone (PTH) substitution therapy
18,300–20,800
100%
no additional benefit
Orphan
01.01.2024
20.06.2024
Avapritinib (3)
Ayvakyt®
Blueprint Medicines GmbH
Oncological diseases
Indolent systemic mastocytosis (ISM)
715–1,000
100%
Indication of
minor additional benefit
Orphan
01.01.2024
20.06.2024
Polatuzumab (3, reassessment >€30m)
Polivy®
Roche Pharma AG
Oncological diseases
Diffuse large B-cell lymphoma, combination with rituximab, cyclophosphamide, doxorubicin and prednisone (R-CHP)
0
5,640–6,270
100%
no additional benefit
Orphan (turnover limit)
01.01.2024
20.06.2024
Polatuzumab (4, reassessment >€30m)
Polivy®
Roche Pharma AG
Oncological diseases
Diffuse large B-cell lymphoma, combination with bendamustine and rituximab
2,550–3,120
100%
no additional benefit
Orphan (turnover limit)
15.12.2023
06.06.2024
Letermovir (3, reassessment >€30m)
Prevymis®
MSD Sharp & Dohme GmbH
Infectious diseases
CMV reactivation/disease, prophylaxis after stem cell transplantation
1,400–1,800
100%
Hint for
non-quantifiable additional benefit
Orphan (turnover limit)
15.12.2023
06.06.2024
Letermovir (2)
Prevymis®
MSD Sharp & Dohme GmbH
Infectious diseases
CMV disease, prophylaxis after kidney transplantation
320
100%
no additional benefit
Orphan (turnover limit)
15.12.2023
06.06.2024
Lanadelumab (3)
Takhzyro®
Takeda GmbH
Other diseases
Hereditary angioedema, prophylaxis, 2 to < 12 years
1–30
100%
no additional benefit
Orphan (turnover limit)
01.12.2023
16.05.2024
Ivacaftor / Tezacaftor / Elexacaftor (15)
Kaftrio®
Vertex Pharmaceuticals (Germany) GmbH
Metabolic diseases
Cystic fibrosis, combination treatment with ivacaftor, from 2 to ≤ 5 years of age (heterozygous for F508del and MF mutation)
160
100%
Hint for
non-quantifiable additional benefit
Orphan (turnover limit)
01.12.2023
16.05.2024
Ivacaftor / Tezacaftor / Elexacaftor (14)
Kaftrio®
Vertex Pharmaceuticals (Germany) GmbH
Metabolic diseases
Cystic fibrosis, combination treatment with ivacaftor, 2 to ≤ 5 years (homozygous for F508del mutation)
250
100%
Hint for
non-quantifiable additional benefit
Orphan (turnover limit)
01.12.2023
16.05.2024
Ivacaftor / Tezacaftor / Elexacaftor (13)
Kaftrio®
Vertex Pharmaceuticals (Germany) GmbH
Metabolic diseases
Cystic fibrosis, F508del mutation, heterozygous and gating mutation, ≥ 2 to ≤ 5 years, combination with ivacaftor
13
100%
no additional benefit
Orphan (turnover limit)
01.12.2023
16.05.2024
Ivacaftor / Tezacaftor / Elexacaftor (12)
Kaftrio®
Vertex Pharmaceuticals (Germany) GmbH
Metabolic diseases
Cystic fibrosis, F508del mutation, heterozygous and RF mutation, ≥ 2 to ≤ 5 years, combination with ivacaftor
20
100%
no additional benefit
Orphan (turnover limit)
01.12.2023
16.05.2024
Ivacaftor / Tezacaftor / Elexacaftor (11)
Kaftrio®
Vertex Pharmaceuticals (Germany) GmbH
Metabolic diseases
Cystic fibrosis, F508del mutation, heterozygous and other or unknown mutation, ≥ 2 to ≤ 5 years, combination with ivacaftor
33
100%
no additional benefit
Orphan (turnover limit)
01.12.2023
16.05.2024
Cannabidiol (8, reassessment >€30m)
Epidyolex®
Jazz Pharmaceuticals Germany GmbH
Nervous system diseases
Dravet syndrome, ≥ 2 years, combination with clobazam
500–2,900
100%
no additional benefit
Orphan (turnover limit)
01.12.2023
16.05.2024
Cannabidiol (7, reassessment >€30m)
Epidyolex®
Jazz Pharmaceuticals Germany GmbH
Nervous system diseases
Lennox-Gastaut syndrome, ≥ 2 years, combination with clobazam
1,700–22,700
100%
no additional benefit
Orphan (turnover limit)
01.12.2023
16.05.2024
Cannabidiol (6, reassessment >€30m)
Epidyolex®
Jazz Pharmaceuticals Germany GmbH
Other diseases
Seizures associated with tuberous sclerosis, ≥ 2 years
200–2,700
100%
no additional benefit
Orphan (turnover limit)
01.12.2023
16.05.2024
Tebentafusp (2, reassessment >€30m)
Kimmtrak®
Immunocore Ireland Ltd.
Oncological diseases
Uveal melanoma, HLA-A*02:01-positive
100–130
100%
Hint for
considerable additional benefit
Orphan (turnover limit)
01.12.2023
16.05.2024
Patisiran (2, reassessment >€30m)
Onpattro®
Alnylam Germany GmbH
Metabolic diseases
Hereditary transthyretin amyloidosis with polyneuropathy (stage 1 or 2)
360
100%
Indication of
less benefit
Orphan (turnover limit)
01.12.2023
16.05.2024
Daratumumab (10, reassessment)
Darzalex®
Janssen-Cilag GmbH
Oncological diseases
Multiple myeloma, first-line, stem cell transplantation unsuitable, combination with bortezomib, melphalan and prednisone
3,450–2,680
100%
Indication of
considerable additional benefit
Orphan (turnover limit)
01.12.2023
16.05.2024
Vosoritid (3)
Voxzogo®
BioMarin International Limited
Other diseases
Achondroplasia, ≥ 4 months to < 2 years
35–49
100%
Hint for
non-quantifiable additional benefit
Orphan (turnover limit)
15.11.2023
02.05.2024
Midostaurin (3, reassessment >€30m)
Rydapt®
Novartis Pharma AG
Oncological diseases
Systemic Mastocytosis
300–400
100%
no additional benefit
Orphan (turnover limit)
15.11.2023
02.05.2024
Midostaurin (2, reassessment >€30m)
Rydapt®
Novartis Pharma AG
Oncological diseases
Acute myeloic Leukemia, FLT3-Mutation
380–1,040
100%
no additional benefit
Orphan (turnover limit)
01.11.2023
02.05.2024
Somapacitan
Sogroya®
Novo Nordisk Pharma GmbH
Metabolic diseases
Growth disturbance due to growth hormone deficiency, ≥ 3 years
9,150–11,600
100%
Hint for
non-quantifiable additional benefit
Orphan
15.10.2023
04.04.2024
Epcoritamab
Tepkinly®
AbbVie Deutschland GmbH & Co. KG
Oncological diseases
Diffuse large B-cell lymphoma (DLBCL), after ≥ 2 prior therapies)
1,050–1,900
100%
Hint for
non-quantifiable additional benefit
Orphan
01.10.2023
21.03.2024
Sirolimus
Hyftor®
Plusultra pharma GmbH
Skin diseases
Facial angiofibroma in tuberous sclerosis, ≥ 6 years.
1,500–5,000
100%
Hint for
non-quantifiable additional benefit
Orphan
15.09.2023
07.03.2024
Talquetamab
Talvey®
Janssen-Cilag GmbH
Oncological diseases
Multiple myeloma, at least 3 prior therapies
1,210–1,310
100%
Hint for
non-quantifiable additional benefit
Orphan
15.09.2023
07.03.2024
Lonapegsomatropin
Skytrofa®
Ascendis Pharma Endocrinology GmbH
Metabolic diseases
Growth disturbance due to growth hormone deficiency, ≥ 3 to < 18 years.
5,710–6,550
100%
Hint for
non-quantifiable additional benefit
Orphan
01.09.2023
15.02.2024
Vosoritid (2, reassessement >€30m)
Voxzogo®
BioMarin International Limited
Other diseases
Achondroplasia, ≥ 2 years
330–460
100%
Indication of
non-quantifiable additional benefit
Orphan (turnover limit)
01.09.2023
15.02.2024
Tisagenlecleucel (7, reassessment)
Kymriah®
Novartis Pharma GmbH
Oncological diseases
B-cell acute lymphoblastic leukemia, relapsed/refractory, 0 ≤ 25 years).
40–90
100%
Hint for
non-quantifiable additional benefit
Orphan
01.09.2023
15.02.2024
Tisagenlecleucel (6, reassessment)
Kymriah®
Novartis Pharma GmbH
Oncological diseases
Diffuse large B-cell lymphoma, relapsed or refractory, ≥ 2 prior therapies)
530–1,200
100%
Hint for
non-quantifiable additional benefit
Orphan
15.08.2023
15.02.2024
Migalastat (3, reassessment >€30m)
Galafold®
Amicus Therapeutics GmbH
Metabolic diseases
Fabry disease, ≥ 12 years
20–460
100%
no additional benefit
Orphan (turnover limit)
01.08.2023
01.02.2024
Bedaquilin (4, reassessment)
Sirturo®
Janssen-Cilag GmbH
Infectious diseases
Multidrug-resistant pulmonary tuberculosis
70–100
100%
Hint for
considerable additional benefit
Orphan
01.08.2023
01.02.2024
Glofitamab
Columvi®
Roche Pharma AG
Oncological diseases
B-cell lymphoma, diffuse large cell (DLBCL)
1,360–1,900
100%
Hint for
non-quantifiable additional benefit
Orphan
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)
15.07.2023
18.01.2024
Ivosidenib (2)
Tibsovo®
Servier Deutschland GmbH
Oncological diseases
Cholangiocarcinoma with IDH1-R132 mutation, after at least 1 prior therapy
80–160
100%
Hint for
non-quantifiable additional benefit
Orphan
15.07.2023
18.01.2024
Ivosidenib
Tibsovo®
Servier Deutschland GmbH
Oncological diseases
Acute myeloid leukemia with IDH1-R132 mutation, first-line, combination with azacitidine
45–125
100%
Indication of
major additional benefit
Orphan
01.08.2023
21.12.2023
Axicabtagen-Ciloleucel (6)
Yescarta®
Gilead Sciences GmbH
Oncological diseases
Follicular lymphoma, after ≥ 3 prior therapies
60–270
100%
no additional benefit
Orphan (turnover limit)
01.07.2023
21.12.2023
Axicabtagen-Ciloleucel (4, reassessment >€30m)
Yescarta®
Gilead Sciences GmbH
Oncological diseases
Diffuse large B-cell lymphoma and primary mediastinal large B-cell lymphoma, after at least 2 prior therapies
680–1,200
100%
no additional benefit
Orphan (turnover limit)
01.07.2023
21.12.2023
Axicabtagen-Ciloleucel (5)
Yescarta®
Gilead Sciences GmbH
Oncological diseases
Diffuse large B-cell lymphoma, highly malignant B-cell lymphoma, after 1 prior therapy, relapse within 12 months or refractory
800–1,130
1,600–2,260
50%
Hint for
non-quantifiable additional benefit
Orphan (turnover limit)
01.07.2023
21.12.2023
Selumetinib (2, reassessment)
Koselugo®
AstraZeneca GmbH
Oncological diseases
Neurofibromatosis (≥ 3 to < 18 years, type 1)
510–740
100%
Hint for
non-quantifiable additional benefit
Orphan
15.05.2023
02.11.2023
Setmelanotid (2)
Imcivree®
Rhythm Pharmaceuticals Inc.
Metabolic diseases
Obesity and control of hunger, Bardet-Biedl syndrome, ≥ 6 years
300–1,100
100%
Hint for
non-quantifiable additional benefit
Orphan
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
15.04.2023
05.10.2023
Tabelecleucel
Ebvallo®
Pierre Fabre Pharma GmbH
Oncological diseases
Relapsed or refractory Epstein-Barr virus positive posttransplant lymphoproliferative disease (EBV+ PTLD), pretreated, 2 years and older
7–30
100%
Hint for
non-quantifiable additional benefit
Orphan
01.04.2023
05.10.2023
Belantamab-Mafodotin (2, reassessment)
Blenrep®
GlaxoSmithKline GmbH & Co. KG
Oncological diseases
Multiple myeloma, at least 4 prior therapies, monotherapy
0
570–1,130
100%
Hint for
non-quantifiable additional benefit
Orphan
01.04.2023
21.09.2023
Pitolisant (3)
Wakix®
Bioprojet Deutschland GmbH
Nervous system diseases
Narcolepsy, with or without cataplexy (children and adolescents, 6-17 years)
120–650
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)
15.02.2023
17.08.2023
Ciltacabtagene Autoleucel
Carvykti®
Janssen-Cilag GmbH
Oncological diseases
Multiple myeloma, at least 3 previous therapies
0
1,210–1,310
100%
Hint for
non-quantifiable additional benefit
Orphan
15.02.2023
03.08.2023
Fenfluramin (2)
Fintepla®
Zogenix GmbH
Nervous system diseases
Lennox-Gastaut syndrome, add-on therapy, ≥ 2 years
2,100–22,700
100%
Hint for
considerable additional benefit
Orphan
15.01.2023
06.07.2023
Maralixibat
Livmarli®
Mirum Pharmaceuticals Germany GmbH
Digestive system diseases
Alagille-Syndrome, ≥ 2 months
139–377
100%
Hint for
non-quantifiable additional benefit
Orphan
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
01.12.2022
01.06.2023
Maribavir
Livtencity®
Takeda GmbH
Infectious diseases
Cytomegalovirus infection (CMI), refractory to therapy
90–130
100%
Hint for
minor additional benefit
Orphan
15.10.2022
06.04.2023
Vutrisiran
Amvuttra®
Alnylam Germany GmbH
Metabolic diseases
Hereditary transthyretin amyloidosis (hTTA) with polyneuropathy (stage 1 or 2)
360
100%
Indication of
minor additional benefit
Orphan
15.10.2022
06.04.2023
Lonafarnib
Zokinvy®
EigerBio Europe Ltd
Other diseases
Hutchinson-Gilford progeria syndrome or progeroid laminopathy, 12 months and over
5–6
100%
Hint for
non-quantifiable additional benefit
Orphan
01.10.2022
16.03.2023
Asciminib
Scemblix®
Novartis Pharma GmbH
Oncological diseases
Chronic myeloid leukaemia (CML), Ph+, after ≥ 2 prior therapies
840–1,150
100%
Indication of
minor additional benefit
Orphan
01.10.2022
16.03.2023
Olipudase alfa
Xenpozyme®
Sanofi-Aventis Deutschland
Metabolic diseases
Acid sphingomyelinase deficiency (ASMD) type A/B or type B
70–80
100%
Hint for
non-quantifiable additional benefit
Orphan
01.10.2022
16.03.2023
Brexucabtagene-Autoleucel (2)
Tecartus®
Gilead Sciences GmbH
Oncological diseases
B-cell acute lymphoblastic leukaemia (ALL), aged > 26
81–200
100%
Hint for
non-quantifiable 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
01.09.2022
16.02.2023
Burosumab (6)
Crysvita®
Kyowa Kirin GmbH
Metabolic diseases
FGF23-related hypophosphatemia in tumour-induced osteomalacia
60–140
100%
no additional benefit
Orphan (turnover limit)
01.09.2022
16.02.2023
Efgartigimod alfa
Vyvgart®
Argenx Germany GmbH
Nervous system diseases
Myasthenia gravis, AChR antibody+
0
14,000–16,800
100%
Hint for
considerable additional benefit
Orphan
01.09.2022
16.02.2023
Birkenrindenextrakt
Filsuvez®
Amryt Pharma GmbH
Other diseases
Wound treatment in epidermolysis bullosa (from 6 months)
270–860
100%
Hint for
minor additional benefit
Orphan
15.08.2022
02.02.2023
Eladocagene Exuparvovec
Upstaza®
PTC Therapeutics International Limited
Metabolic diseases
Aromatic L-amino acid decarboxylase (AADC) deficiency, ≥ 18 months
4–30
100%
Hint for
non-quantifiable additional benefit
Orphan
01.07.2022
15.12.2022
Mosunetuzumab
Lunsumio®
Roche Pharma AG
Oncological diseases
Follicular lymphoma (FL), after ≥ 2 prior therapies
650–690
100%
Hint for
non-quantifiable additional benefit
Orphan
01.07.2022
15.12.2022
Cerliponase alfa (2, reassessment)
Brineura®
BioMarin Deutschland GmbH
Metabolic diseases
Neuronal ceroid lipofuscinosis (NCL) type 2
40–50
100%
Hint for
major additional benefit
Orphan
01.06.2022
01.12.2022
Setmelanotid
Imcivree®
Rhythm Pharmaceuticals Inc.
Metabolic diseases
Obesity and control of hunger, POMC-, PCSK1- or LEPR-deficient obesity, ≥ 6 years
140–280
100%
Hint for
non-quantifiable additional benefit
Orphan
01.06.2022
01.12.2022
Polatuzumab Vedotin (2)
Polivy®
Roche Pharma AG
Oncological diseases
Diffuse large B-cell lymphoma (DLBCL), combination with rituximab, cyclophosphamide, doxorubicin and prednisone (R-CHP)
0
5,510–6,130
100%
Hint for
non-quantifiable additional benefit
Orphan
01.06.2022
01.12.2022
Tisagenlecleucel (5)
Kymriah®
Novartis Pharma GmbH
Oncological diseases
Follicular lymphoma (FL), pre-treated patients
650–690
100%
Hint for
non-quantifiable additional benefit
Orphan
15.05.2022
03.11.2022
Axicabtagen-Ciloleucel (3, reassessment)
Yescarta®
Gilead Sciences GmbH
Oncological diseases
Diffuse large B-cell lymphoma (DLBCL) and primary mediastinal large B-cell lymphoma, after at least 2 prior therapies
0
455–729
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
01.05.2022
20.10.2022
Tebentafusp
Kimmtrak®
Immunocore Ireland Ltd.
Oncological diseases
Uveal melanoma, HLA-A*02:01-positive
0
110
100%
Hint for
considerable additional benefit
Orphan
15.04.2022
06.10.2022
Glucarpidase
Voraxaze®
SERB GmbH
Other diseases
Reduction of toxic MTX plasma concentrations; children ≥ 28 days and adults.
90–440
100%
Hint for
non-quantifiable additional benefit
Orphan
01.04.2022
15.09.2022
Avapritinib (2)
Ayvakyt®
Blueprint Medicines (Germany) GmbH
Oncological diseases
Systemic mastocytosis, after at least 1 prior therapy
270–680
100%
Hint for
non-quantifiable additional benefit
Orphan
01.04.2022
15.09.2022
Somatrogon
Ngenla®
Pfizer Pharma GmbH
Metabolic diseases
Growth disturbance due to growth hormone deficiency, ≥ 3 to < 18 years of age
5,710–6,550
100%
Hint for
non-quantifiable additional benefit
Orphan
01.04.2022
15.09.2022
Voretigen Neparvovec (2, reassessment)
Luxturna®
Novartis Pharma GmbH
Eye diseases
Hereditary retinal dystrophy
100–530
100%
Hint for
considerable 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
01.04.2022
15.09.2022
Idebenon (2, reassessment)
Raxone®
Chiesi GmbH
Eye diseases
Leber's hereditary optic neuropathy (LHOP)
1,400–3,000
100%
Hint for
non-quantifiable additional benefit
Orphan
01.04.2022
15.09.2022
Daratumumab (9, reassessment)
Darzalex®
Janssen-Cilag
Oncological diseases
Multiple myeloma (MM), after at least 1 previous therapy, combination with lenalidomide and dexamethasone or bortezomib and dexamethasone
4,700–7,000
100%
Proof of
considerable additional benefit
Orphan (turnover limit)
15.02.2022
04.08.2022
Ivacaftor / Tezacaftor / Elexacaftor (10)
Kaftrio®
Vertex Pharmaceuticals (Germany) GmbH
Metabolic diseases
Cystic fibrosis (CF), F508del mutation, heterozygous and other or unknown mutation, ≥ 6 to ≤ 11 years, combination with ivacaftor
56
100%
no additional benefit
Orphan (turnover limit)
15.02.2022
04.08.2022
Ivacaftor / Tezacaftor / Elexacaftor (9)
Kaftrio®
Vertex Pharmaceuticals (Germany) GmbH
Metabolic diseases
Cystic fibrosis (CF), F508del mutation, heterozygous and RF mutation, ≥ 6 to ≤ 11 years, combination with ivacaftor
21
100%
no additional benefit
Orphan (turnover limit)
15.02.2022
04.08.2022
Ivacaftor / Tezacaftor / Elexacaftor (8)
Kaftrio®
Vertex Pharmaceuticals (Germany) GmbH
Metabolic diseases
Cystic fibrosis, F508del mutation, heterozygous and gating mutation, ≥ 6 to ≤ 11 years, combination with ivacaftor)
28
100%
no additional benefit
Orphan (turnover limit)
15.02.2022
04.08.2022
Ivacaftor / Tezacaftor / Elexacaftor (7)
Kaftrio®
Vertex Pharmaceuticals (Germany) GmbH
Metabolic diseases
Cystic fibrosis (CF), combination treatment with ivacaftor, 6 to ≤ 11 years (homozygous for F508del mutation)
470
100%
Hint for
non-quantifiable additional benefit
Orphan (turnover limit)
15.02.2022
04.08.2022
Ivacaftor / Tezacaftor / Elexacaftor (6)
Kaftrio®
Vertex Pharmaceuticals (Germany) GmbH
Metabolic diseases
Cystic fibrosis (CF), combination treatment with ivacaftor, from 6 to ≤ 11 years (heterozygous for F508del and MF mutation)
233
100%
Indication of
considerable additional benefit
Orphan (turnover limit)
15.02.2022
04.08.2022
Ivacaftor (26)
Kalydeco®
Vertex Pharmaceuticals (Germany) GmbH
Metabolic diseases
Cystic fibrosis (CF), F508del mutation, heterozygous and other or unknown mutation, ≥ 6 to ≤ 11 years, combination with ivacaftor / tezacaftor / elexacaftor
56
100%
no additional benefit
Orphan (turnover limit)
15.02.2022
04.08.2022
Ivacaftor (25)
Kalydeco®
Vertex Pharmaceuticals (Germany) GmbH
Metabolic diseases
Cystic fibrosis (CF), F508del mutation, heterozygous and RF mutation, ≥ 6 to ≤ 11 years, combination with ivacaftor / tezacaftor / elexacaftor
21
100%
no additional benefit
Orphan (turnover limit)
15.02.2022
04.08.2022
Ivacaftor (24)
Kalydeco®
Vertex Pharmaceuticals (Germany) GmbH
Metabolic diseases
Cystic fibrosis (CF), F508del mutation, heterozygous and gating mutation, ≥ 6 to ≤ 11 years, combination with ivacaftor / tezacaftor / elexacaftor).
28
100%
no additional benefit
Orphan (turnover limit)
15.02.2022
04.08.2022
Ivacaftor (23)
Kalydeco®
Vertex Pharmaceuticals (Germany) GmbH
Metabolic diseases
Cystic fibrosis (CF), combination treatment with ivacaftor/tezacaftor/elexacaftor, 6 to ≤ 11 years (homozygous for F508del mutation)
470
100%
Hint for
non-quantifiable additional benefit
Orphan (turnover limit)
15.02.2022
04.08.2022
Ivacaftor (22)
Kalydeco®
Vertex Pharmaceuticals (Germany) GmbH
Metabolic diseases
Cystic fibrosis (CF), combination treatment with ivacaftor/tezacaftor/elexacaftor, 6 to ≤ 11 years (heterozygous for F508del and MF mutation)
230
100%
Indication of
considerable additional benefit
Orphan (turnover limit)
15.02.2022
04.08.2022
Avacopan
Tavneos®
Vifor Pharma Deutschland GmbH
Musculoskeletal system diseases
Granulomatosis with polyangiitis or microscopic polyangiitis, combination with rituximab or cyclophosphamide
2,180–2,280
100%
Hint for
minor additional benefit
Orphan
01.02.2022
21.07.2022
Burosumab (5, reassessment >€50m)
Crysvita®
Kyowa Kirin GmbH
Metabolic diseases
X-linked hypophosphatemia, ≥ 18 years of age
410–810
100%
no additional benefit
Orphan (turnover limit)
01.02.2022
21.07.2022
Burosumab (4, reassessment >€50m)
Crysvita®
Kyowa Kirin GmbH
Metabolic diseases
X-linked hypophosphatemia, ≥ 1 to < 18 years of age
200–550
100%
Hint for
non-quantifiable additional benefit
Orphan (turnover limit)
01.01.2022
16.06.2022
Idecabtagen vicleucel
Abecma®
Bristol-Myers Squibb GmbH & Co KGaA
Oncological diseases
Multiple myeloma (MM), at least 3 previous therapies
1,200–1,300
100%
Hint for
non-quantifiable additional benefit
Orphan
01.01.2022
16.06.2022
Ripretinib
Qinlock®
Deciphera Pharmaceuticals (Netherlands) B.V.
Oncological diseases
Gastrointestinal stromal tumours (GIST), ≥ 3 prior therapies
220–300
100%
Hint for
major additional benefit
Orphan
15.11.2021
05.05.2022
Delamanid
Deltyba®
Otsuka Novel Products
Infectious diseases
Multidrug-resistant pulmonary tuberculosis (TBC), ≥ 10 kg
80–114
100%
Hint for
non-quantifiable additional benefit
Orphan
01.11.2021
21.04.2022
Ixazomib (2, reassessment)
Ninlaro®
Takeda GmbH
Oncological diseases
Multiple myeloma (MM), at least 1 prior therapy, combination with lenalidomide and dexamethasone
4,700–7,000
100%
Hint for
non-quantifiable additional benefit
Orphan
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
Vosoritid
Voxzogo®
BioMarin International Limited
Other diseases
Achondroplasia, ≥ 2 years
0
340–480
100%
Hint for
non-quantifiable additional benefit
Orphan
01.10.2021
18.03.2022
Daratumumab (8, reassessment)
Darzalex®
Janssen-Cilag GmbH
Oncological diseases
Multiple myeloma (MM), newly diagnosed, patients not suitable for autologous stem cell transplantation, combination with lenalidomide and dexamethasone
3,470–3,670
100%
Hint for
considerable additional benefit
Orphan (turnover limit)
15.09.2021
03.03.2022
Odevixibat
Bylvay®
Albireo Pharma AB
Digestive system diseases
Cholestasis, ≥ 6 months
40–110
100%
Hint for
minor additional benefit
Orphan
15.09.2021
03.03.2022
Tafasitamab
Minjuvi®
Incyte Biosciences Germany
Oncological diseases
Diffuse large B-cell lymphoma (DLBCL), combination with lenalidomide
730–1,560
100%
Hint for
non-quantifiable additional benefit
Orphan
01.09.2021
17.02.2022
Migalastat (2)
Galafold®
Amicus Therapeutics GmbH
Metabolic diseases
Fabry disease, 12 to < 16 years
0
1–19
100%
Hint for
non-quantifiable additional benefit
Orphan
15.08.2021
03.02.2022
Selumetinib
Koselugo®
AstraZeneca GmbH
Oncological diseases
Neurofibromatosis (type 1), ≥ 3 to < 18 years
0
510–740
100%
Hint for
non-quantifiable additional benefit
Orphan
01.08.2021
03.02.2022
Daratumumab (7)
Darzalex®
Janssen-Cilag GmbH
Oncological diseases
Multiple Myeloma (MM), at least 1 pretreatment, combination with Pomalidomid and Dexamethason
3,190–3,600
38%
Hint for
minor additional benefit
Orphan (turnover limit)
01.08.2021
20.01.2022
Blinatumomab (6)
Blincyto®
Amgen GmbH
Oncological diseases
B-cell acute lymphoblastic leukaemia (ALL), high-risk first relapse, Ph-, CD19+, 1 to ≤ 18 years
7–30
100%
Indication of
major additional benefit
Orphan
01.08.2021
20.01.2022
Daratumumab (6)
Darzalex®
Janssen-Cilag GmbH
Oncological diseases
Systemic light chain amyloidosis, first-line, combination with cyclophosphamide, bortezomib and dexamethasone
440–1,030
50%
Hint for
minor additional benefit
Orphan (turnover limit)
15.07.2021
06.01.2022
Satralizumab
Enspryng®
Roche Pharma AG
Nervous system diseases
Neuromyelitis optica spectrum disorders (NMOSD), anti-aquaporin-4 IgG seropositive, ≥ 12 years
460–5,050
100%
Hint for
minor additional benefit
Orphan
01.07.2021
16.12.2021
Brentuximab Vedotin (6, reassessment)
Adcetris®
Takeda GmbH
Oncological diseases
Systemic anaplastic large cell lymphoma; sALCL; first-line; combination with cyclophosphamide, doxorubicin and prednisone
125–127
100%
Hint for
minor additional benefit
Orphan
01.07.2021
16.12.2021
Cabozantinib (7, reassessment)
Cometriq®
Ipsen Pharma GmbH
Oncological diseases
Thyroid carcinoma (MTC)
50–670
100%
Hint for
non-quantifiable additional benefit
Orphan
15.06.2021
02.12.2021
Tagraxofusp
Elzonris®
Stemline Therapeutics B.V.
Oncological diseases
Blastic plasmacytoid dendritic cell neoplasm (BPDCN), first-line
30–90
100%
Hint for
non-quantifiable additional benefit
Orphan
01.06.2021
19.11.2021
Ivacaftor / Tezacaftor / Elexacaftor (5)
Kaftrio®
Vertex Pharmaceuticals (Germany) GmbH
Metabolic diseases
Cystic fibrosis (CF), combination treatment with ivacaftor in patients ≥ 12 years (heterozygous for F508del and others or unknown mutation).
310
100%
no additional benefit
Orphan (turnover limit)
01.06.2021
19.11.2021
Ivacaftor / Tezacaftor / Elexacaftor (4)
Kaftrio®
Vertex Pharmaceuticals (Germany) GmbH
Metabolic diseases
Cystic fibrosis (CF), combination treatment with ivacaftor in patients ≥ 12 years (heterozygous for F508del and RF mutation)
173
100%
no additional benefit
Orphan (turnover limit)
01.06.2021
19.11.2021
Ivacaftor / Tezacaftor / Elexacaftor (3)
Kaftrio®
Vertex Pharmaceuticals (Germany) GmbH
Metabolic diseases
Cystic fibrosis (CF), combination treatment with ivacaftor in patients > 12 years (heterozygous for F508del and gating mutation (incl. R117H)).
133
100%
no additional benefit
Orphan (turnover limit)
01.06.2021
19.11.2021
Ivacaftor (21)
Kalydeco®
Vertex Pharmaceuticals (Germany) GmbH
Metabolic diseases
Cystic fibrosis (CF), combination treatment with ivacaftor/tezacaftor/elexacaftor in patients ≥ 12 years (heterozygous for F508del and others or unknown mutation)
310
100%
no additional benefit
Orphan (turnover limit)
01.06.2021
19.11.2021
Ivacaftor (20)
Kalydeco®
Vertex Pharmaceuticals (Germany) GmbH
Metabolic diseases
Cystic fibrosis (CF), combination treatment with ivacaftor/tezacaftor/elexacaftor in patients ≥ 12 years (heterozygous for F508del and RF mutation)
173
100%
no additional benefit
Orphan (turnover limit)
01.06.2021
19.11.2021
Ivacaftor (19)
Kalydeco®
Vertex Pharmaceuticals (Germany) GmbH
Metabolic diseases
Cystic fibrosis (CF), combination treatment with ivacaftor/tezacaftor/elexacaftor in patients ≥ 12 years (heterozygous for F508del and gating mutation (incl. R117H))
133
100%
no additional benefit
Orphan (turnover limit)
15.05.2021
04.11.2021
Lanadelumab (2, reassessment >€50m)
Takhzyro®
Takeda GmbH
Other diseases
Hereditary angioedema (HAE), prophylaxis, ≥ 12 years
140–430
100%
no additional benefit
Orphan (turnover limit)
15.05.2021
04.11.2021
Onasemnogen-Abeparvovec
Zolgensma®
Novartis Gene Therapies EU Limited
Nervous system diseases
5q-associated spinal muscular atrophy (SMA)
46–67
100%
no additional benefit
Orphan (turnover limit)
15.05.2021
04.11.2021
Obinutuzumab (6, reassessment >€50m)
Gazyvaro®
Roche Pharma AG
Oncological diseases
Follicular lymphoma (FL), first-line
1,300–1,500
100%
no additional benefit
Orphan (turnover limit)
15.05.2021
04.11.2021
Obinutuzumab (5, reassessment >€50m)
Gazyvaro®
Roche Pharma AG
Oncological diseases
Chronic lymphocytic leukemia (CLL)
820–1,480
100%
no additional benefit
Orphan (turnover limit)
15.05.2021
04.11.2021
Obinutuzumab (4, reassessment >€50m)
Gazyvaro®
Roche Pharma AG
Oncological diseases
Follicular lymphoma (FL)
790–940
100%
no additional benefit
Orphan (turnover limit)
15.05.2021
04.11.2021
Cannabidiol (5)
Epidyolex®
GW Pharmaceuticals plc
Other diseases
Seizures in Tuberous Sclerosis, ≥ 2 years
0
500–2,700
100%
Hint for
non-quantifiable additional benefit
Orphan
01.05.2021
04.11.2021
Atidarsagen autotemcel OTL-200
Libmeldy®
Orchard Therapeutics GmbH
Metabolic diseases
Metachromatic leukodystrophy with biallelic mutation in the ARSA gene
2–4
67%
Hint for
major additional benefit
Orphan
01.05.2021
21.10.2021
Risdiplam
Evrysdi®
Roche Pharma AG
Nervous system diseases
5q-associated spinal muscular atrophy (SMA)
992–2,142
42%
Hint for
non-quantifiable additional benefit
Orphan
15.04.2021
07.10.2021
Pemigatinib
Pemazyre®
Incyte Biosciences Germany GmbH
Oncological diseases
Cholangiocarcinoma with FGFR2 fusion or FGFR2 rearrangement, at least 1 prior therapy
35–300
100%
Hint for
non-quantifiable additional benefit
Orphan
01.04.2021
16.09.2021
Bedaquilin (3)
Sirturo®
Janssen-Cilag GmbH
Infectious diseases
Multidrug-resistant pulmonary tuberculosis, 5 to 11 years
1
100%
Hint for
non-quantifiable additional benefit
Orphan
15.03.2021
02.09.2021
Imlifidase
Idefirix®
Hansa Biopharma AB
Other diseases
Desensitisation in kidney transplantation
3–69
100%
Hint for
non-quantifiable additional benefit
Orphan
15.03.2021
02.09.2021
Fedratinib
Inrebic®
Celgene GmbH
Oncological diseases
Myelofibrosis (MF)
740–3,590
1,370–5,280
100%
Hint for
non-quantifiable additional benefit
Orphan
15.02.2021
05.08.2021
Autologe Anti-CD19-transduzierte CD3-positive Zellen
Tecartus®
Gilead Sciences GmbH
Oncological diseases
Mantle cell lymphoma (MCL), pretreated
105–150
100%
Hint for
non-quantifiable additional benefit
Orphan
01.02.2021
15.07.2021
Blinatumomab (5)
Blincyto®
Amgen GmbH
Oncological diseases
B-cell acute lymphoblastic leukaemia, relapsed or refractory, Ph+ CD19+
5–10
100%
Hint for
non-quantifiable additional benefit
Orphan
01.02.2021
15.07.2021
Niraparib (4, reassessment)
Zejula®
GlaxoSmithKline GmbH & Co. KG
Oncological diseases
Ovarian carcinoma, fallopian tube carcinoma or primary peritoneal carcinoma
700–1,000
100%
no additional benefit
Orphan (turnover limit)
01.02.2021
15.07.2021
Fenfluramin
Fintepla®
Zogenix GmbH
Nervous system diseases
Dravet syndrome, ≥ 2 years
450–2,450
100%
Hint for
considerable additional benefit
Orphan
15.01.2021
15.07.2021
Carfilzomib (4)
Kyprolis®
Amgen GmbH
Oncological diseases
Multiple myeloma (MM), at least 1 prior therapy, combination with daratumumab and dexamethasone
4,700–7,000
100%
no additional benefit
Orphan (turnover limit)
01.01.2021
01.07.2021
Afamelanotid (2, reassessment)
Scenesse®
Clinuvel (UK) Limited
Metabolic diseases
Erythropoietic protoporphyria
540–1,090
100%
Hint for
non-quantifiable additional benefit
Orphan
01.01.2021
01.07.2021
Lumasiran
Oxlumo®
Alnylam Germany GmbH
Metabolic diseases
Hyperoxaluria
50–880
100%
Hint for
non-quantifiable additional benefit
Orphan
01.12.2020
03.06.2021
Sebelipase alfa (2, reassessment)
Kanuma®
Alexion Pharma Germany GmbH
Metabolic diseases
Lysosomal acid lipase deficiency
35–70
100%
Hint for
non-quantifiable additional benefit
Orphan
01.12.2020
20.05.2021
Tezacaftor / Ivacaftor (5)
Symkevi®
Vertex Pharmaceuticals (Ireland) Limited
Metabolic diseases
Cystic fibrosis (CF), combination therapy with ivacaftor in patients 6 to < 12 years (heterozygous for F508del and RF mutation)
50
100%
no additional benefit
Orphan (turnover limit)
01.12.2020
20.05.2021
Nusinersen (2, reassessment >€50m)
Spinraza®
Biogen GmbH
Nervous system diseases
Spinal muscular atrophy (SMA)
1,013–1,203
19%
Indication of
major additional benefit
Orphan (turnover limit)
01.12.2020
20.05.2021
Tezacaftor / Ivacaftor (4)
Symkevi®
Vertex Pharmaceuticals (Ireland) Limited
Metabolic diseases
Cystic fibrosis (CF), combination therapy with ivacaftor in patients 6 to < 12 years (homozygous for F508del)
470
100%
no additional benefit
Orphan (turnover limit)
01.12.2020
20.05.2021
Tafamidis (4, reassessment >€50m)
Vyndaqel®
Pfizer Pharma GmbH
Metabolic diseases
Amyloid cardiomyopathy
1,760–1,810
100%
Indication of
considerable additional benefit
Orphan (turnover limit)
01.12.2020
20.05.2021
Tafamidis (3, reassessment >€50m)
Vyndaqel®
Pfizer Pharma GmbH
Metabolic diseases
Amyloid Neuropathy
230
100%
no additional benefit
Orphan (turnover limit)
01.12.2020
20.05.2021
Niraparib (3)
Zejula®
GlaxoSmithKline GmbH & Co. KG
Oncological diseases
Ovarian carcinoma, fallopian tube carcinoma or primary peritoneal carcinoma, FIGO stages III and IV, maintenance therapy
2,010–2,810
100%
no additional benefit
Orphan (turnover limit)
01.12.2020
20.05.2021
Ivacaftor (18)
Kalydeco®
Vertex Pharmaceuticals (Ireland) Limited
Metabolic diseases
Cystic fibrosis (CF), combination therapy with tezacaftor/ivacaftor in patients 6 to < 12 years (heterozygous for F508del and MF mutation)
50
100%
no additional benefit
Orphan (turnover limit)
01.12.2020
20.05.2021
Ivacaftor (17)
Kalydeco®
Vertex Pharmaceuticals (Ireland) Limited
Metabolic diseases
Cystic fibrosis (CF), combination therapy with tezacaftor/ivacaftor in patients 6 to < 12 years (homozygous for F508del mutation)
470
100%
no additional benefit
Orphan (turnover limit)
01.12.2020
20.05.2021
Ivacaftor (16)
Kalydeco®
Vertex Pharmaceuticals (Ireland) Limited
Metabolic diseases
Cystic fibrosis (CF), patients from 4 to < 6 months, R117H mutation
1
100%
Hint for
non-quantifiable additional benefit
Orphan (turnover limit)
01.12.2020
20.05.2021
Ivacaftor (15)
Kalydeco®
Vertex Pharmaceuticals (Ireland) Limited
Metabolic diseases
Cystic fibrosis (CF), patients from 4 to < 6 months, gating mutations
1
100%
Hint for
non-quantifiable additional benefit
Orphan (turnover limit)
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
01.12.2020
20.05.2021
Amikacin (liposomal)
Arikayce® liposomal
Insmed Germany GmbH
Infectious diseases
Mycobacterium avium complex (MAC) lung infections
350–760
100%
Hint for
non-quantifiable additional benefit
Orphan
01.11.2020
15.04.2021
Avapritinib
Ayvakyt®
Blueprint Medicines (Germany)
Oncological diseases
Gastrointestinal stromal tumor (GIST)
1–90
100%
Hint for
non-quantifiable additional benefit
Orphan
01.11.2020
15.04.2021
Burosumab (3)
Crysvita®
Kyowa Kirin GmbH
Metabolic diseases
X-linked hypophosphatemia (XLH), ≥ 18 years
0
410–810
100%
Hint for
minor additional benefit
Orphan
15.10.2020
15.04.2021
Cannabidiol (4, reassessment)
Epidyolex®
GW Pharmaceuticals plc
Nervous system diseases
Lennox-Gastaut syndrome, ≥ 2 years, combination with clobazam
0
2,600–22,700
100%
Hint for
considerable additional benefit
Orphan
15.10.2020
15.04.2021
Cannabidiol (3, reassessment)
Epidyolex®
GW Pharmaceuticals plc
Nervous system diseases
Dravet syndrome, ≥ 2 years, combination with clobazam
0
1,100–3,100
100%
Hint for
considerable additional benefit
Orphan
01.10.2020
01.04.2021
Ibrutinib (7)
Imbruvica®
Janssen-Cilag GmbH
Oncological diseases
Chronic lymphocytic leukaemia (CLL), first-line, combination with rituximab
3,090
59%
Hint for
considerable additional benefit
Orphan (turnover limit)
15.09.2020
04.03.2021
Belantamab-Mafodotin
Blenrep®
GlaxoSmithKline GmbH & Co. KG
Oncological diseases
Multiple myeloma (MM), at least 4 prior therapies, monotherapy
0
570–1,130
100%
Hint for
non-quantifiable additional benefit
Orphan
01.09.2020
18.02.2021
Bulevirtid
Hepcludex®
MYR GmbH
Infectious diseases
Chronic hepatitis Delta
300–4,800
100%
Hint for
non-quantifiable additional benefit
Orphan
01.09.2020
18.02.2021
Ivacaftor / Tezacaftor / Elexacaftor
Kaftrio®
Vertex Pharmaceuticals Ltd
Metabolic diseases
Cystic fibrosis (CF), combination treatment with ivacaftor in patients ≥ 12 years (homozygous for F508del mutation)
2,400
100%
Indication of
major additional benefit
Orphan (turnover limit)
01.09.2020
18.02.2021
Ivacaftor / Tezacaftor / Elexacaftor (2)
Kaftrio®
Vertex Pharmaceuticals Ltd
Metabolic diseases
Cystic fibrosis (CF), combination treatment with ivacaftor in patients ≥12 years (heterozygous for F508del and MF mutation)
1,000
100%
Hint for
major additional benefit
Orphan (turnover limit)
01.09.2020
18.02.2021
Ivacaftor (14)
Kalydeco®
Vertex Pharmaceuticals Ltd
Metabolic diseases
Cystic fibrosis (CF), combination therapy with ivacaftor/tezacaftor/elexacaftor in patients ≥ 12 years (heterozygous for F508del and MF mutation)
1,000
100%
Hint for
major additional benefit
Orphan (turnover limit)
01.09.2020
18.02.2021
Ivacaftor (13)
Kalydeco®
Vertex Pharmaceuticals Limited
Metabolic diseases
Cystic fibrosis (CF), combination therapy with ivacaftor/tezacaftor/elexacaftor in patients ≥ 12 years (homozygous for F508del mutation)
2,400
100%
Indication of
major additional benefit
Orphan (turnover limit)
15.08.2020
18.02.2021
Glasdegib
Daurismo®
Pfizer Pharma GmbH
Oncological diseases
Acute myeloid leukaemia (AML), combination with cytarabine (LDAC)
780–840
100%
Hint for
considerable additional benefit
Orphan
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
Osilodrostat
Isturisa®
Recordati Rare Diseases Germany GmbH
Metabolic diseases
Endogenous Cushing syndrome
1,130–1,550
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
Ivacaftor (12)
Kalydeco®
Vertex Pharmaceuticals
Metabolic diseases
Cystic fibrosis (CF), patients ≥ 6 months to < 18 years
26
100%
Hint for
non-quantifiable additional benefit
Orphan (turnover limit)
01.07.2020
17.12.2020
Tezacaftor / Ivacaftor (3, reassessment >€50m)
Symkevi®
Vertex Pharmaceuticals
Metabolic diseases
Cystic fibrosis (CF), combination treatment with ivacaftor in patients ≥ 12 years (homozygous for F508del)
2,400
100%
no additional benefit
Orphan (turnover limit)
01.07.2020
17.12.2020
Tezacaftor / Ivacaftor (2, reassessment >€50m)
Symkevi®
Vertex Pharmaceuticals
Metabolic diseases
Cystic fibrosis (CF), combination treatment with ivacaftor in patients ≥ 12 years (heterozygous for F508del)
200–300
100%
no additional benefit
Orphan (turnover limit)
15.06.2020
03.12.2020
Brentuximab Vedotin (5)
Adcetris®
Takeda GmbH
Oncological diseases
Systemic anaplastic large cell lymphoma; first-line; combination with cyclophosphamide, doxorubicin and prednisone
0
125–127
100%
Hint for
minor additional benefit
Orphan
15.06.2020
03.12.2020
Mogamulizumab
Poteligeo®
Kyowa Kirin GmbH
Oncological diseases
Mycosis Fungoides; Sézary Syndrome
310–460
100%
Hint for
non-quantifiable additional benefit
Orphan
01.06.2020
20.11.2020
Ponatinib (3, reassessment)
Iclusig®
Incyte Biosciences Germany GmbH
Oncological diseases
Chronic myeloid leukaemia (CML)
500–940
100%
Hint for
non-quantifiable additional benefit
Orphan
01.06.2020
20.11.2020
Ponatinib (2, reassessment)
Iclusig®
Incyte Biosciences Germany GmbH
Oncological diseases
Acute myeloid leukaemia (AML)
25–195
100%
Hint for
non-quantifiable additional benefit
Orphan
15.04.2020
15.10.2020
Givosiran
Givlaari®
Alnylam Germany GmbH
Metabolic diseases
Acute hepatic porphyria, ≥ 12 years
1,000–1,700
100%
Indication of
considerable additional benefit
Orphan
15.03.2020
17.09.2020
Tisagenlecleucel (3, reassessment)
Kymriah®
Novartis Pharma GmbH
Oncological diseases
B-cell acute lymphoblastic leukaemia (ALL)
0
50–65
100%
Hint for
non-quantifiable additional benefit
Orphan
15.03.2020
17.09.2020
Tisagenlecleucel (4, reassessment)
Kymriah®
Novartis Pharma GmbH
Oncological diseases
Diffuse large B-cell lymphoma (DLBCL)
0
450–720
100%
Hint for
non-quantifiable additional benefit
Orphan
15.03.2020
03.09.2020
Riociguat (2, reassessment >€50m)
Adempas®
MSD Sharp & Dohme GmbH
Cardiovascular diseases
Chronic thromboembolic pulmonary hypertension
920–5,460
100%
no additional benefit
Orphan (turnover limit)
15.03.2020
03.09.2020
Riociguat (3, reassessment >€50m)
Adempas®
MSD Sharp & Dohme GmbH
Cardiovascular diseases
Pulmonary arterial hypertension (PAH)
580–7,850
100%
no additional benefit
Orphan (turnover limit)
01.03.2020
20.08.2020
Tafamidis (2)
Vyndaqel®
Pfizer Pharma GmbH
Metabolic diseases
Amyloid cardiomyopathy
0
1,630–1,730
100%
Hint for
considerable additional benefit
Orphan
15.02.2020
20.08.2020
Polatuzumab Vedotin
Polivy®
Roche Pharma AG
Oncological diseases
Diffuse large B-cell lymphoma (DLBCL), combination with bendamustine and rituximab
0
730–1,560
100%
Hint for
non-quantifiable additional benefit
Orphan
15.02.2020
20.08.2020
Bedaquilin (2)
Sirturo®
Janssen-Cilag GmbH
Infectious diseases
Multidrug-resistant pulmonary tuberculosis, 12 to < 18 years
9–13
100%
Hint for
non-quantifiable additional benefit
Orphan
15.02.2020
20.08.2020
Daratumumab (5)
Darzalex®
Janssen-Cilag GmbH
Oncological diseases
Multiple myeloma (MM), newly diagnosed, patients not suitable for autologous stem cell transplantation, combination with lenalidomide and dexamethasone
0
3,479–3,670
100%
Hint for
minor additional benefit
Orphan (turnover limit)
15.02.2020
20.08.2020
Daratumumab (4)
Darzalex®
Janssen-Cilag GmbH
Oncological diseases
Multiple myeloma (MM), newly diagnosed, patients suitable for autologous stem cell transplantation, combination with bortezomib, thalidomide and dexamethasone
1,800–1,900
100%
Hint for
non-quantifiable additional benefit
Orphan (turnover limit)
15.12.2019
04.06.2020
Ivacaftor (11)
Kalydeco®
Vertex Pharmaceuticals
Metabolic diseases
Cystic fibrosis (CF), ≥ 6 to < 12 months
2
100%
Hint for
non-quantifiable additional benefit
Orphan (turnover limit)
01.12.2019
14.05.2020
Gilteritinib
XOSPATA®
Astellas Pharma GmbH
Oncological diseases
Acute myeloid leukaemia (AML), FLT3 mutation
220–580
100%
Hint for
considerable additional benefit
Orphan
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
15.10.2019
02.04.2020
Cannabidiol (2)
Epidyolex®
GW Pharmaceuticals plc
Nervous system diseases
Dravet syndrome, ≥ 2 years, combination with clobazam
0
1,100–3,100
100%
Hint for
non-quantifiable additional benefit
Orphan
15.10.2019
02.04.2020
Niraparib (2, reassessment >€50m)
Zejula®
TESARO Bio Germany GmbH
Oncological diseases
Ovarian carcinoma, fallopian tube carcinoma or primary peritoneal carcinoma
0
1,900–2,400
100%
no additional benefit
Orphan (turnover limit)
15.10.2019
02.04.2020
Cannabidiol
Epidyolex®
GW Pharmaceuticals plc
Nervous system diseases
Lennox-Gastaut syndrome, ≥ 2 years, combination with clobazam
0
2,600–22,700
100%
Hint for
non-quantifiable additional benefit
Orphan
15.10.2019
02.04.2020
Asfotase alfa (2, reassessment >€50m)
Strensiq®
Alexion Pharma Germany GmbH
Metabolic diseases
Hypophosphatasia (HPP)
1,074
0.8%
Hint for
non-quantifiable additional benefit
Orphan (turnover limit)
01.10.2019
02.04.2020
Burosumab (2, reassessment)
Crysvita®
Kyowa Kirin GmbH
Metabolic diseases
X-linked hypophosphatemia (XLH), ≥ 1 to < 18 years
0
200–500
100%
Hint for
non-quantifiable additional benefit
Orphan
01.09.2019
20.02.2020
Ivacaftor (10)
Kalydeco®
Vertex Pharmaceuticals (Europe) Limited
Metabolic diseases
Cystic fibrosis (CF), 12 ro < 24 months
5
100%
Hint for
non-quantifiable additional benefit
Orphan (turnover limit)
01.09.2019
20.02.2020
Ivacaftor (9, reassessment >€50m)
Kalydeco®
Vertex Pharmaceuticals (Europe) Limited
Metabolic diseases
Cystic fibrosis (CF), patients ≥ 18 years with R117H mutation
35–44
100%
Hint for
minor additional benefit
Orphan (turnover limit)
01.09.2019
20.02.2020
Ivacaftor (8, reassessment >€50m)
Kalydeco®
Vertex Pharmaceuticals (Europe) Limited
Metabolic diseases
Cystic fibrosis (CF), patients from 2 to 5 years
15
100%
Hint for
non-quantifiable additional benefit
Orphan (turnover limit)
01.09.2019
20.02.2020
Ivacaftor (7, reassessment >€50m)
Kalydeco®
Vertex Pharmaceuticals (Europe) Limited
Metabolic diseases
Cystic fibrosis (CF), various gating mutations, ≥ 6 years
10–11
100%
Hint for
non-quantifiable additional benefit
Orphan (turnover limit)
01.09.2019
20.02.2020
Ivacaftor (6, reassessment >€50m)
Kalydeco®
Vertex Pharmaceuticals (Europe) Limited
Metabolic diseases
Cystic fibrosis (CF), heterozygous for F508del, ≥ 12 years, combination with tezacaftor
200–300
100%
no additional benefit
Orphan (turnover limit)
01.09.2019
20.02.2020
Ivacaftor (5, reassessment >€50m)
Kalydeco®
Vertex Pharmaceuticals (Europe) Limited
Metabolic diseases
Cystic fibrosis (CF), homozygous for F508del, ≥ 12 years, combination with tezacaftor
2,400
100%
no additional benefit
Orphan (turnover limit)
01.09.2019
20.02.2020
Ivacaftor (4, reassessment >€50m)
Kalydeco®
Vertex Pharmaceuticals (Europe) Limited
Metabolic diseases
Cystic fibrosis (CF), G551D mutation, ≥ 6 years
210
86%
Hint for
considerable additional benefit
Orphan (turnover limit)
01.09.2019
20.02.2020
Ibrutinib (5)
Imbruvica®
Janssen-Cilag GmbH
Oncological diseases
Chronische lymphatische Leukämie (CLL), Erstlinie, Kombination mit Obinutuzumab
3,090
26%
Hint for
minor additional benefit
Orphan (turnover limit)
01.09.2019
20.02.2020
Ibrutinib (6)
Imbruvica®
Janssen-Cilag GmbH
Oncological diseases
Waldenström's disease, combination with rituximab
590–1,180
100%
no additional benefit
Orphan (turnover limit)
15.08.2019
20.02.2020
Volanesorsen
Waylivra®
Akcea Therapeutics Germany GmbH
Metabolic diseases
Chylomicronaemia syndrome
60–120
100%
Hint for
non-quantifiable additional benefit
Orphan
01.07.2019
19.12.2019
Pegvaliase
Palynziq®
BioMarin International Limited
Metabolic diseases
Phenylketonuria
435
100%
Hint for
non-quantifiable additional benefit
Orphan
15.06.2019
05.12.2019
Pomalidomid (3)
Imnovid®
Celgene GmbH
Oncological diseases
Multiple myeloma (MM), at least 1 prior therapy, combination with bortezomib and dexamethasone
3,060–3,450
100%
no additional benefit
Orphan (turnover limit)
15.04.2019
17.10.2019
Voretigen Neparvovec
Luxturna®
Novartis Pharma GmbH
Eye diseases
Hereditary retinal dystrophy
0
100–530
100%
Hint for
considerable additional benefit
Orphan
15.04.2019
17.10.2019
Nintedanib (3, reassessment >€50m, Ofev®)
Ofev®
Boehringer Ingelheim Pharma GmbH & Co. KG
Respiratory system diseases
Idiopathic pulmonary fibrosis
1,800–18,900
100%
Hint for
considerable additional benefit
Orphan (turnover limit)
15.03.2019
05.09.2019
Brentuximab Vedotin (4)
Adcetris®
Takeda Pharma GmbH
Oncological diseases
Hodgkin lymphoma (HL), CD30+, first-line
220–380
100%
non-quantifiable additional benefit
Orphan
15.02.2019
15.08.2019
Blinatumomab (3)
Blincyto®
Amgen GmbH
Oncological diseases
B-cell acute lymphoblastic leukaemia (ALL), MRD-positive patients
40–110
100%
non-quantifiable additional benefit
Orphan
15.02.2019
15.08.2019
Blinatumomab (4)
Blincyto®
Amgen GmbH
Oncological diseases
B-cell acute lymphoblastic leukaemia (ALL), ≥ 1 to <18 years
30–80
100%
non-quantifiable additional benefit
Orphan
01.02.2019
01.08.2019
Mexiletin
Namuscla®
Lupin Europe GmbH
Nervous system diseases
Myotonia
530–650
100%
non-quantifiable additional benefit
Orphan
01.02.2019
01.08.2019
Lanadelumab
Takhzyro®
Shire Deutschland GmbH, Teil der Takeda Group
Other diseases
Hereditary angioedema (HAE)
0
140–430
100%
considerable additional benefit
Orphan
15.01.2019
04.07.2019
Glycerolphenylbutyrat (2)
Ravicti®
Swedish Orphan Biovitrum GmbH
Metabolic diseases
Urea cycle disorders (UCD), 0 to < 2 months
10–18
100%
non-quantifiable additional benefit
Orphan
15.01.2019
04.07.2019
Bedaquilin
Sirturo®
Janssen-Cilag GmbH
Infectious diseases
Multidrug-resistant pulmonary tuberculosis
0
70–100
100%
considerable additional benefit
Orphan
01.12.2018
16.05.2019
Tezacaftor / Ivacaftor
Symkevi®
Vertex Pharmaceuticals (Europe) Limited
Metabolic diseases
Cystic fibrosis (CF), F508del mutation, ≥ 12 years
0
2,600–2,700
91%
considerable additional benefit
Orphan
01.11.2018
02.05.2019
Axicabtagen-Ciloleucel (2)
Yescarta®
Kite, Gilead company
Oncological diseases
Primary mediastinal large B-cell lymphoma (PMBCL)
0
5–9
100%
non-quantifiable additional benefit
Orphan
01.11.2018
02.05.2019
Axicabtagen-Ciloleucel
Yescarta®
Kite, Gilead company
Oncological diseases
Diffuse large B-cell lymphoma (DLBCL)
0
440–700
100%
non-quantifiable additional benefit
Orphan
01.10.2018
22.03.2019
Patisiran
Onpattro®
Alnylam Germany GmbH
Metabolic diseases
Amyloidosis
0
350
100%
considerable additional benefit
Orphan
01.10.2018
22.03.2019
Inotersen
Tegsedi®
Akcea Therapeutics Germany GmbH
Metabolic diseases
Amyloidosis
350
100%
non-quantifiable additional benefit
Orphan
01.10.2018
22.03.2019
Metreleptin
Myalepta®
Aegerion Pharmaceuticals GmbH
Metabolic diseases
Lipodystrophy
90–180
100%
non-quantifiable additional benefit
Orphan
01.10.2018
22.03.2019
Daunorubicin / Cytarabin (liposomale Formulierung)
Vyxeos® liposomal
Jazz Pharmaceuticals
Oncological diseases
Therapy-related acute myeloid leukaemia (AML); acute myeloid leukaemia (AML) with multilinear dysplasia
310–510
100%
considerable additional benefit
Orphan
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
01.10.2018
22.03.2019
Vestronidase alfa
Mepsevii®
Ultragenyx Germany GmbH
Metabolic diseases
Mucopolysaccharidosis (MPS VII; Sly syndrome)
2–7
100%
non-quantifiable additional benefit
Orphan
01.10.2018
22.03.2019
Daratumumab (3)
Darzalex®
Janssen-Cilag GmbH
Oncological diseases
Multiple myeloma (MM), first-line, unsuitable for stem cell transplantation, combination with bortezomib, melphalan and prednisone
0
3,380–3,900
100%
Hint for
considerable additional benefit
Orphan (turnover limit)
15.09.2018
07.03.2019
Tisagenlecleucel
Kymriah®
Novartis Pharma GmbH
Oncological diseases
Diffuse large B-cell lymphoma (DLBCL)
0
440–700
100%
non-quantifiable additional benefit
Orphan
15.09.2018
07.03.2019
Tisagenlecleucel (2)
Kymriah®
Novartis Pharma GmbH
Oncological diseases
B-cell acute lymphoblastic leukaemia (ALL)
0
50–65
100%
non-quantifiable additional benefit
Orphan
01.09.2018
21.02.2019
Gemtuzumab Ozogamicin
Mylotarg®
Pfizer Pharma GmbH
Oncological diseases
Acute myeloid leukaemia (AML)
560–1,150
100%
non-quantifiable additional benefit
Orphan
01.07.2018
20.12.2018
Velmanase alfa
Lamzede®
Chiesi GmbH
Metabolic diseases
Alpha mannosidosis
70–140
100%
non-quantifiable additional benefit
Orphan
01.06.2018
22.11.2018
Darvadstrocel
Alofisel®
Takeda Pharma Vertrieb GmbH & Co. KG
Digestive system diseases
Anal fistulas in Crohn's disease
90–230
100%
non-quantifiable additional benefit
Orphan
15.04.2018
04.10.2018
Burosumab
Crysvita®
Kyowa Kirin GmbH
Metabolic diseases
X-linked hypophosphatemia (XLH), ≥ 1 to < 18 years
0
200–500
100%
non-quantifiable additional benefit
Orphan
01.03.2018
16.08.2018
Glycerolphenylbutyrat
Ravicti®
Swedish Orphan Biovitrum GmbH
Metabolic diseases
Urea cycle disorders (UCD), ≥ 2 months
100–250
100%
non-quantifiable additional benefit
Orphan
15.02.2018
02.08.2018
Letermovir
Prevymis®
MSD Sharp & Dohme GmbH
Infectious diseases
Cytomegalovirus infection
0
1,000–1,800
100%
non-quantifiable additional benefit
Orphan
15.01.2018
05.07.2018
Brentuximab Vedotin (3)
Adcetris®
Takeda Pharma GmbH
Oncological diseases
Cutaneous T-cell lymphoma, CD30+
40–130
100%
minor additional benefit
Orphan
15.01.2018
05.07.2018
Allogene, genetisch modifizierte T-Zellen
Zalmoxis®
Dompé farmaceutici S.p.A
Oncological diseases
Haematological malignancies, concomitant therapy in haploidentical haematopoietic stem cell transplantation
100–140
100%
non-quantifiable additional benefit
Orphan
15.12.2017
07.06.2018
Niraparib
Zejula®
TESARO Bio Germany GmbH
Oncological diseases
Ovarian carcinoma, fallopian tube carcinoma or primary peritoneal carcinoma
0
1,900–2,400
100%
non-quantifiable additional benefit
Orphan
15.11.2017
03.05.2018
Cenegermin
Oxervate®
Dompé farmaceutici S.p.A
Eye diseases
Keratitis
1,730–3,170
100%
non-quantifiable additional benefit
Orphan
15.10.2017
05.04.2018
Telotristatethyl
Xermelo®
Ipsen Pharma GmbH
Oncological diseases
Carcinoid tumor, neuroendocrine tumors, combination with SAA therapy
300–1,000
100%
non-quantifiable additional benefit
Orphan
15.10.2017
05.04.2018
Midostaurin
Rydapt®
Novartis Pharma GmbH
Oncological diseases
Acute myeloid leukaemia (AML); Systemic mastocytosis (ASM)
0
400–710
80%
considerable additional benefit
Orphan
15.10.2017
05.04.2018
Obinutuzumab (3)
Gazyvaro®
Roche Pharma AG
Oncological diseases
Follicular lymphoma (FL), first-line
0
1,300–1,500
100%
non-quantifiable additional benefit
Orphan
15.09.2017
16.03.2018
Elosulfase alfa (2, reassessment)
Vimizim®
BioMarin Deutschland GmbH
Metabolic diseases
Mucopolysaccharidosis (type IVA)
20–100
100%
minor additional benefit
Orphan
01.10.2017
16.03.2018
Avelumab
Bavencio®
Merck Serono GmbH / Pfizer Pharma GmbH
Oncological diseases
Merkel-cell carcinoma (MCC)
0
160–410
100%
non-quantifiable additional benefit
Orphan
15.08.2017
15.02.2018
Carfilzomib (3, reassessment >€50m)
Kyprolis®
Amgen GmbH
Oncological diseases
Multiple myeloma (MM), at least 1 prior therapy, combination with dexamethasone or lenalidomide and dexamethasone
4,700–7,000
100%
Hint for
considerable additional benefit
Orphan (turnover limit)
15.08.2017
15.02.2018
Daratumumab (2, reassessment >€50m)
Darzalex®
Janssen-Cilag GmbH
Oncological diseases
Multiple myeloma (MM), monotherapy; multiple myeloma, at least 1 prior therapy, combination with lenalidomide and dexamethasone or bortezomib and dexamethasone
2,300
7,000–9,300
72%
Indication of
considerable additional benefit
Orphan (turnover limit)
15.07.2017
18.01.2018
Inotuzumab Ozogamicin
Besponsa®
Pfizer Pharma GmbH
Oncological diseases
B-cell acute lymphoblastic leukaemia (ALL)
120–200
100%
minor additional benefit
Orphan
01.07.2017
21.12.2017
Nusinersen
Spinraza®
Biogen GmbH
Nervous system diseases
Spinal muscular atrophy (SMA)
0
840–1,060
10%
major additional benefit
Orphan
01.07.2017
21.12.2017
Cerliponase alfa
Brineura®
BioMarin Deutschland GmbH
Metabolic diseases
Neuronal ceroid lipofuscinosis type 2
0
20–40
100%
non-quantifiable additional benefit
Orphan
15.06.2017
07.12.2017
Blinatumomab (2, reassessment)
Blincyto®
Amgen GmbH
Oncological diseases
B-cell acute lymphoblastic leukaemia (ALL)
60–170
100%
considerable additional benefit
Orphan
15.01.2017
06.07.2017
Obeticholsäure
Ocaliva®
Intercept Pharma Deutschland GmbH
Digestive system diseases
Biliary cirrhosis
0
1,050–7,350
100%
non-quantifiable additional benefit
Orphan
15.01.2017
06.07.2017
Ixazomib
Ninlaro®
Takeda Pharma GmbH
Oncological diseases
Multiple myeloma (MM), at least 1 prior therapy, combination with lenalidomide and dexamethasone
0
4,700–7,000
100%
non-quantifiable additional benefit
Orphan
01.01.2017
15.06.2017
Venetoclax
Venclyxto®
AbbVie Deutschland GmbH & Co. KG
Oncological diseases
Chronic lymphocytic leukaemia (CLL), monotherapy
0
300–700
100%
non-quantifiable additional benefit
Orphan
01.12.2016
18.05.2017
Olaratumab
Lartruvo®
Lilly Deutschland GmbH
Oncological diseases
Soft tissue sarcoma
0
1,200–1,400
100%
considerable additional benefit
Orphan
15.10.2016
06.04.2017
Macitentan (2, reassessment >€50m)
Opsumit®
Actelion Pharmaceuticals Deutschland GmbH
Cardiovascular diseases
Pulmonary arterial hypertension (PAH)
580–7,850
100%
no additional benefit
Orphan (turnover limit)
01.10.2016
16.03.2017
Ibrutinib (4)
Imbruvica®
Janssen-Cilag GmbH
Oncological diseases
Chronic lymphocytic leukaemia (CLL), at least 1 prior therapy, combination with bendamustine and rituximab
1,500–5,600
50%
Hint for
considerable additional benefit
Orphan (turnover limit)
01.08.2016
19.01.2017
Teduglutid (2)
Revestive®
Shire Deutschland GmbH
Digestive system diseases
Short bowel syndrome, 1 to < 18 years
230–500
100%
non-quantifiable additional benefit
Orphan
01.08.2016
19.01.2017
Tasimelteon
Hetlioz®
Vanda Pharmceutical Inc
Nervous system diseases
Sleep disorders (sleep-wake rhythm), blind adults
7,000
100%
non-quantifiable additional benefit
Orphan
01.08.2016
19.01.2017
Pitolisant
Wakix®
Bioprojet Pharma SARL
Nervous system diseases
Narcolepsy
15,000–30,000
100%
non-quantifiable additional benefit
Orphan
01.08.2016
19.01.2017
Brentuximab Vedotin (2)
Adcetris®
Takeda Pharma GmbH
Oncological diseases
Hodgkin lymphoma (HL), CD30+, increased risk of recurrence or progression after transplantation
40–60
100%
non-quantifiable additional benefit
Orphan
01.08.2016
19.01.2017
Carfilzomib (2)
Kyprolis®
Amgen GmbH
Oncological diseases
Multiple myeloma (MM), combination with dexamethasone
0
4,700–7,000
100%
minor additional benefit
Orphan
01.07.2016
15.12.2016
Ibrutinib (3)
Imbruvica®
Janssen-Cilag GmbH
Oncological diseases
Chronic lymphocytic leukemia (CLL), first-line
2,840
100%
no additional benefit
Orphan (turnover limit)
01.07.2016
15.12.2016
Obinutuzumab (2)
Gazyvaro®
Roche Pharma AG
Oncological diseases
Follicular lymphoma (FL)
0
790–940
100%
non-quantifiable additional benefit
Orphan
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
01.06.2016
01.12.2016
Migalastat
Galafold®
Amicus Therapeutics GmbH
Metabolic diseases
Fabry disease
0
20–490
100%
non-quantifiable additional benefit
Orphan
01.06.2016
01.12.2016
Ataluren (2, reassessment)
Translarna®
PTC Therapeutics International Limited
Musculoskeletal system diseases
Duchenne muscular dystrophy (DMD), ≥ 5 years
30–40
100%
minor additional benefit
Orphan
01.06.2016
01.12.2016
Daratumumab
Darzalex®
Janssen-Cilag GmbH
Oncological diseases
Multiple myeloma (MM), monotherapy, pre-treated patients
0
2,300
100%
non-quantifiable additional benefit
Orphan
15.02.2016
04.08.2016
Afamelanotid
Scenesse®
Clinuvel Ltd.
Metabolic diseases
Erythropoietic protoporphyria
0
540–1,090
100%
non-quantifiable additional benefit
Orphan
01.02.2016
21.07.2016
Ibrutinib (2, reassessment >€50m)
Imbruvica®
Janssen-Cilag GmbH
Oncological diseases
Mantle cell lymphoma (MCL), chronic lymphocytic leukaemia (CLL), Waldenström's disease
3,780–11,100
12%
Indication of
considerable additional benefit
Orphan (turnover limit)
15.12.2015
02.06.2016
Blinatumomab
Blincyto®
Amgen GmbH
Oncological diseases
B-cell acute lymphoblastic leukaemia (ALL)
0
60–170
100%
non-quantifiable additional benefit
Orphan
15.12.2015
02.06.2016
Carfilzomib
Kyprolis®
Amgen GmbH
Oncological diseases
Multiple myeloma (MM), at least 1 prior therapy, combination with dexamethasone or lenalidomide and dexamethasone
0
4,700–7,000
100%
non-quantifiable additional benefit
Orphan
15.12.2015
02.06.2016
Ivacaftor (3)
Kalydeco®
Vertex Pharmaceuticals (Europe) Limited
Metabolic diseases
Cystic fibrosis (CF), R117H mutation, 2 to 5 years, > 18 years
0
59
75%
minor additional benefit
Orphan
15.11.2015
04.05.2016
Isavuconazol
Cresemba®
Basilea Pharmaceutica International Ltd.
Infectious diseases
Aspergillosis, mucormycosis
1,293–5,342
100%
non-quantifiable additional benefit
Orphan
01.10.2015
17.03.2016
Sebelipase alfa
Kanuma®
Synageva BioPharma Limited
Metabolic diseases
Lysosomal acid lipase deficiency
0
31–843
100%
non-quantifiable additional benefit
Orphan
01.10.2015
17.03.2016
Pomalidomid (2, reassessment >€50m)
Imnovid®
Celgene GmbH
Oncological diseases
Multiple myeloma (MM), at least 2 prior therapies, combination with dexamethasone
2,300
50%
Hint for
considerable additional benefit
Orphan (turnover limit)
01.10.2015
17.03.2016
Panobinostat
Farydak®
Novartis Pharma GmbH
Oncological diseases
Multiple myeloma (MM), at least 1 prior therapy, combination with pomalidomide and dexamethasone
2,300
100%
non-quantifiable additional benefit
Orphan
01.10.2015
17.03.2016
Idebenon
Raxone®
Santhera Pharmaceuticals
Eye diseases
Leber hereditary optic neuropathy
0
1,500–3,000
100%
non-quantifiable additional benefit
Orphan
01.10.2015
17.03.2016
Asfotase alfa
Strensiq®
Alexion Europe SAS
Metabolic diseases
Hypophosphatasia (HPP)
0
1,000
100%
non-quantifiable additional benefit
Orphan
01.07.2015
17.12.2015
Lenvatinib
Lenvima®
Eisai GmbH
Oncological diseases
Thyroid carcinoma (DTC)
0
600–815
100%
non-quantifiable additional benefit
Orphan
01.06.2015
27.11.2015
Olaparib
Lynparza®
AstraZeneca GmbH
Oncological diseases
Ovarian carcinoma, fallopian tube carcinoma or primary peritoneal carcinoma, platinum-sensitive, maintenance therapy
0
200–600
100%
non-quantifiable additional benefit
Orphan
15.04.2015
15.10.2015
Ruxolitinib (3)
Jakavi®
Novartis Pharma GmbH
Oncological diseases
Polycythaemia vera
240–1,470
100%
Hint for
considerable additional benefit
Orphan (turnover limit)
01.04.2015
01.10.2015
Eliglustat
Cerdelga®
Genzyme GmbH
Metabolic diseases
Sphingolipidoses (Gaucher disease type 1)
150–500
100%
non-quantifiable additional benefit
Orphan
15.03.2015
03.09.2015
Nintedanib (2, Ofev®)
Ofev®
Boehringer Ingelheim Pharma GmbH & Co. KG
Respiratory system diseases
Idiopathic pulmonary fibrosis
0
2,080–19,700
100%
minor additional benefit
Orphan
01.02.2015
16.07.2015
Ramucirumab
Cyramza®
Lilly Deutschland GmbH
Oncological diseases
Gastric or gastro-oesophageal junction adenocarcinoma; combination with paclitaxel
0
5,900–7,900
100%
minor additional benefit
Orphan
01.01.2015
18.06.2015
Pasireotid (2)
Signifor®
Novartis Pharma GmbH
Metabolic diseases
Acromegaly
265–1,133
100%
minor additional benefit
Orphan
01.12.2014
21.05.2015
Ataluren
Translarna®
PTC Therapeutics International Limited
Musculoskeletal system diseases
Duchenne muscular dystrophy (DMD), ≥ 5 years
0
82–110
100%
minor additional benefit
Orphan
01.11.2014
21.05.2015
Alipogentiparvovec
Glybera®
Chiesi GmbH
Metabolic diseases
Familial lipoprotein lipase deficiency
17–35
100%
non-quantifiable additional benefit
Orphan
01.11.2014
16.04.2015
Ibrutinib
Imbruvica®
Janssen-Cilag GmbH
Oncological diseases
Mantle cell lymphoma (MCL), Chronic lymphocytic leukemia (CLL)
0
2,900–8,750
100%
non-quantifiable additional benefit
Orphan
01.09.2014
19.02.2015
Teduglutid
Revestive®
NPS Pharma Germany GmbH
Digestive system diseases
Short bowel syndrome
1,100–2,400
100%
minor additional benefit
Orphan
01.09.2014
19.02.2015
Ivacaftor (2)
Kalydeco®
Vertex Pharmaceuticals Limited
Metabolic diseases
Cystic fibrosis (CF), various mutations, ≥ 6 years
0
10–11
100%
minor additional benefit
Orphan
15.08.2014
05.02.2015
Obinutuzumab
Gazyvaro®
Roche Pharma AG
Oncological diseases
Chronic lymphocytic leukemia (CLL)
0
818–1,477
100%
non-quantifiable additional benefit
Orphan
01.08.2014
22.01.2015
Cabozantinib
Cometriq®
Swedish Orphan Biovitrum GmbH
Oncological diseases
Thyroid carcinoma (MTC)
0
60–500
100%
minor additional benefit
Orphan
15.06.2014
04.12.2014
Siltuximab
Sylvant®
Janssen-Cilag GmbH
Oncological diseases
Multicentric Castleman Disease (MCD)
130–1,460
100%
non-quantifiable additional benefit
Orphan
01.06.2014
20.11.2014
Elosulfase alfa
Vimizim®
BioMarin Deutschland GmbH
Metabolic diseases
Mucopolysaccharidosis (type IVA)
0
15–95
100%
minor additional benefit
Orphan
15.05.2014
06.11.2014
Cholsäure
Orphacol®
Laboratoires CTRS
Metabolic diseases
Bile acid synthesis disorder
10–25
100%
non-quantifiable additional benefit
Orphan
15.05.2014
06.11.2014
Ruxolitinib (2, reassessment >€50m)
Jakavi®
Novartis Pharma GmbH
Oncological diseases
Myelofibrosis (MF)
1,600–5,000
100%
Hint for
considerable additional benefit
Orphan (turnover limit)
01.05.2014
16.10.2014
Riociguat
Adempas®
Bayer Vital GmbH
Cardiovascular diseases
Pulmonary arterial hypertension (PAH), Chronic thromboembolic pulmonary hypertension (CTPH)
0
1,500–13,310
100%
minor additional benefit
Orphan
01.02.2014
17.07.2014
Macitentan
Opsumit®
Actelion Pharmaceuticals Deutschland GmbH
Cardiovascular diseases
Pulmonary arterial hypertension (PAH)
0
580–7,850
100%
minor additional benefit
Orphan
01.09.2013
20.02.2014
Pomalidomid
Imnovid®
Celgene GmbH
Oncological diseases
Multiple myeloma (MM), at least 2 prior therapies, combination with dexamethasone
0
1,900
100%
considerable additional benefit
Orphan
01.08.2013
23.01.2014
Ponatinib
Iclusig®
ARIAD Pharmaceuticals (Germany) GmbH
Oncological diseases
Chronic myeloid leukaemia (CML); acute lymphoblastic leukaemia (ALL), Ph+ or T315I mutation
0
525–1,135
100%
non-quantifiable additional benefit
Orphan
01.05.2013
17.10.2013
Bosutinib
Bosulif®
Pfizer Pharma GmbH
Oncological diseases
Chronic myeloid leukaemia (CML)
0
380–500
100%
non-quantifiable additional benefit
Orphan
01.12.2012
16.05.2013
Brentuximab Vedotin
Adcetris®
Takeda Pharma Vertrieb GmbH & Co. KG
Oncological diseases
Hodgkin lymphoma (HL), CD30+; Systemic anaplastic large cell lymphoma
75–420
100%
non-quantifiable additional benefit
Orphan
01.11.2012
02.05.2013
Decitabin
Dacogen®
Janssen-Cilag GmbH
Oncological diseases
Acute myeloid leukaemia (AML)
300–780
100%
minor additional benefit
Orphan
15.09.2012
07.03.2013
Ruxolitinib
Jakavi®
Novartis Pharma GmbH
Oncological diseases
Myelofibrosis (MF)
0
1,600
100%
minor additional benefit
Orphan
15.08.2012
07.02.2013
Ivacaftor
Kalydeco®
Vertex Pharmaceuticals GmbH
Metabolic diseases
Cystic fibrosis (CF), G551D mutation, ≥ 6 years
0
170
84%
considerable additional benefit
Orphan
15.06.2012
06.12.2012
Pasireotid
Signifor®
Novartis Pharma GmbH
Metabolic diseases
Pituitary dysfunction
160–360
100%
minor additional benefit
Orphan
15.12.2011
07.06.2012
Tafamidis
Vyndaqel®
Pfizer Pharma GmbH
Metabolic diseases
Amyloid Neuropathy
0
40–104
100%
minor additional benefit
Orphan
15.09.2011
15.03.2012
Pirfenidon
Esbriet®
InterMune Deutschland GmbH
Respiratory system diseases
Idiopathic pulmonary fibrosis
6,000
100%
non-quantifiable additional benefit
Orphan