Orphan drugs

Orphan drugs are considered to provide additional benefit by law, as long as sales within 12 months stay below a turnover limit of €30 mio (until 2022: €50 mio) xx different products comprising xx resolutions fall into this category. The G-BA only decides on the extent of this additional benefit. Therefore, orphan drugs received the most positive assessment so far from the G-BA: xx% of the patient subpopulations were granted a major additional benefit, xx% a considerable, xx% a minor and xx% a not quantifiable additional benefit. No additional benefit was identified in xx% of the patient population.

All resolutions concerning orphan drugs

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)
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)
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
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
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
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
Fosdenopterin Nulibry® Sentynl Therapeutics Nervous system diseases Molybdenum cofactor deficiency type A 2 100% Hint for non-quantifiable additional benefit Orphan
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
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
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
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)
Fedratinib (2, reassessment) Inrebic® Bristol-Myers Squibb GmbH Oncological diseases Myelofibrosis 640–1,710 100% Hint for non-quantifiable additional benefit Orphan
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
Exagamglogen autotemcel (2) Casgevy® Vertex Pharmaceuticals (Germany) GmbH Hematopoietic diseases Sickle cell disease with recurrent vaso-occlusive crises; ≥ 12 years; no HLA-matched related stem cell donation available 130–1,230 100% Hint for non-quantifiable additional benefit Orphan
Exagamglogen autotemcel Casgevy® Vertex Pharmaceuticals (Germany) GmbH Hematopoietic diseases Β-thalassaemia, transfusion-dependent, ≥ 12 years, no HLA-compatible related stem cell donation available 20–150 100% Indication of non-quantifiable additional benefit Orphan
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
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
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)
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)
Artesunat Artesunate Amivas® Amivas Ireland Ltd Infectious diseases Severe malaria, from birth 87 100% non-quantifiable additional benefit Orphan
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
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
Polihexanid Akantior® SIFI S.p.A. Eye diseases Acanthamoebic keratitis; ≥ 12 years of age 70–410 100% Hint for non-quantifiable additional benefit Orphan
Isavuconazol (2) Cresemba® Pfizer Pharma GmbH Infectious diseases Mucormycosis, ≥ 1 to ≤ 17 years 8 100% Hint for non-quantifiable additional benefit Orphan
Isavuconazol (3) Cresemba® Pfizer Pharma GmbH Infectious diseases Aspergillosis, ≥ 1 to ≤ 17 years 40–215 100% Hint for non-quantifiable additional benefit Orphan
Sotatercept Winrevair® MSD Sharp & Dohme GmbH Cardiovascular diseases Pulmonary arterial hypertension 580–7,850 100% Hint for minor additional benefit Orphan
rADAMTS13 Adzynma® Takeda GmbH Hematopoietic diseases ADAMTS13 deficiency in congenital thrombotic thrombocytopenic purpura (cTTP) 120–180 100% Hint for non-quantifiable additional benefit Orphan
Sparsentan Filspari® Vifor Pharma Deutschland GmbH Genitourinary system diseases Immunoglobulin A - Nephropathy, primary 900–13,000 100% Hint for minor additional benefit Orphan
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
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)
Iptacopan Fabhalta® Novartis Pharma GmbH Hematopoietic diseases Paroxysmal nocturnal hemoglobinuria 290–945 100% Hint for non-quantifiable additional benefit Orphan
Tofersen Qalsody® Biogen GmbH Nervous system diseases Amyotrophic lateral sclerosis (ALS) 90–170 100% 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
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
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
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
Luspatercept (6) Reblozyl® Bristol-Myers Squibb GmbH & Co. KGaA Hematopoietic diseases Myelodysplastic syndromes with transfusion-dependent anemia, not pretreated, and without ring sideroblasts, pretreated 4,960–7,080 80% Hint for minor additional benefit Orphan (turnover limit)
Efanesoctocog alfa Altuvoct® Swedish Orphan Biovitrum GmbH Hematopoietic diseases Hemophilia A 0 100% no additional benefit Orphan
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)
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)
Omaveloxolon Skyclarys® Biogen GmbH Nervous system diseases Friedreich's ataxia, ≥ 16 years 970 100% Hint for non-quantifiable additional benefit Orphan
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
Momelotinib Omjjara® GlaxoSmithKline GmbH & Co. KG Oncological diseases Myelofibrosis 210–1,160 50% Hint for minor additional benefit Orphan
Rezafungin Rezzayo® Mundipharma Infectious diseases Invasive candida infections 31,800–34,600 100% Hint for non-quantifiable additional benefit Orphan
Pegzilarginase Loargys® Immedica Pharma Germany GmbH Metabolic diseases Hyperargininemia (ARG1-D), ≥ 2 years 50 100% Hint for non-quantifiable additional benefit Orphan
Vamorolon Agamree® Santhera Pharmaceuticals GmbH Musculoskeletal system diseases Duchenne muscular dystrophy, ≥ 4 years 740–3,670 100% Hint for non-quantifiable additional benefit Orphan
Palopegteriparatid Yorvipath® Ascendis Pharma Endocrinology GmbH Metabolic diseases Chronic hypoparathyroidism, parathyroid hormone (PTH) substitution therapy 18,300–20,800 100% no additional benefit Orphan
Avapritinib (3) Ayvakyt® Blueprint Medicines GmbH Oncological diseases Indolent systemic mastocytosis (ISM) 715–1,000 100% Indication of minor additional benefit Orphan
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)
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)
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)
Letermovir (2) Prevymis® MSD Sharp & Dohme GmbH Infectious diseases CMV disease, prophylaxis after kidney transplantation 320 100% no additional benefit Orphan (turnover limit)
Lanadelumab (3) Takhzyro® Takeda GmbH Other diseases Hereditary angioedema, prophylaxis, 2 to < 12 years 1–30 100% no additional benefit Orphan (turnover limit)
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)
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)
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)
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)
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)
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)
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)
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)
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)
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)
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)
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)
Midostaurin (3, reassessment >€30m) Rydapt® Novartis Pharma AG Oncological diseases Systemic Mastocytosis 300–400 100% no additional benefit Orphan (turnover limit)
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)
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
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
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
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
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
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)
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
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
Migalastat (3, reassessment >€30m) Galafold® Amicus Therapeutics GmbH Metabolic diseases Fabry disease, ≥ 12 years 20–460 100% no additional benefit Orphan (turnover limit)
Bedaquilin (4, reassessment) Sirturo® Janssen-Cilag GmbH Infectious diseases Multidrug-resistant pulmonary tuberculosis 70–100 100% Hint for considerable additional benefit Orphan
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
Eftrenonacog alfa (2, reassessment >€30m) Alprolix® Swedish Orphan Biovitrum GmbH Hematopoietic diseases Hemophilia B 560–720 100% no additional benefit Orphan (turnover limit)
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
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
Axicabtagen-Ciloleucel (6) Yescarta® Gilead Sciences GmbH Oncological diseases Follicular lymphoma, after ≥ 3 prior therapies 60–270 100% no additional benefit Orphan (turnover limit)
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)
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)
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
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
Luspatercept (5, reassessment >€30m) Reblozyl® Bristol-Myers Squibb GmbH & Co. KGaA Hematopoietic diseases Β-thalassaemia, transfusion-dependent anaemia 250–330 100% no additional benefit Orphan (turnover limit)
Luspatercept (4, reassessment >€30m) Reblozyl® Bristol-Myers Squibb GmbH & Co. KGaA Hematopoietic diseases Myelodysplastic syndrome with transfusion-dependent anaemia, pre-treated 790–1,860 100% no additional benefit Orphan (turnover limit)
Etranacogen Dezaparvovec Hemgenix® CSL Behring GmbH Hematopoietic diseases Hemophilia B 231–358 100% Hint for non-quantifiable additional benefit Orphan
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
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
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
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)
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
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
Maralixibat Livmarli® Mirum Pharmaceuticals Germany GmbH Digestive system diseases Alagille-Syndrome, ≥ 2 months 139–377 100% Hint for non-quantifiable additional benefit Orphan
Sutimlimab Enjaymo® Sanofi-Aventis Deutschland GmbH Hematopoietic diseases Cold agglutinin disease (CAD) 370–1,510 100% Hint for minor additional benefit Orphan
Maribavir Livtencity® Takeda GmbH Infectious diseases Cytomegalovirus infection (CMI), refractory to therapy 90–130 100% Hint for minor additional benefit Orphan
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
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
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
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
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
Valoctocogene roxaparvovec Roctavian® BioMarin International Ltd. Hematopoietic diseases Hemophilia A 690–800 100% Hint for non-quantifiable additional benefit Orphan
Burosumab (6) Crysvita® Kyowa Kirin GmbH Metabolic diseases FGF23-related hypophosphatemia in tumour-induced osteomalacia 60–140 100% no additional benefit Orphan (turnover limit)
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
Birkenrindenextrakt Filsuvez® Amryt Pharma GmbH Other diseases Wound treatment in epidermolysis bullosa (from 6 months) 270–860 100% Hint for minor additional benefit Orphan
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
Mosunetuzumab Lunsumio® Roche Pharma AG Oncological diseases Follicular lymphoma (FL), after ≥ 2 prior therapies 650–690 100% Hint for non-quantifiable additional benefit Orphan
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
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
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
Tisagenlecleucel (5) Kymriah® Novartis Pharma GmbH Oncological diseases Follicular lymphoma (FL), pre-treated patients 650–690 100% Hint for non-quantifiable additional benefit Orphan
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
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
Tebentafusp Kimmtrak® Immunocore Ireland Ltd. Oncological diseases Uveal melanoma, HLA-A*02:01-positive 0
110
100% Hint for considerable additional benefit Orphan
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
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
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
Voretigen Neparvovec (2, reassessment) Luxturna® Novartis Pharma GmbH Eye diseases Hereditary retinal dystrophy 100–530 100% Hint for considerable additional benefit Orphan
Pegcetacoplan Aspaveli® Swedish Orphan Biovitrum GmbH Hematopoietic diseases Paroxysmal nocturnal haemoglobinuria (PNH), pre-treated patients 190–520 100% Hint for non-quantifiable additional benefit Orphan
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
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)
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)
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)
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)
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)
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)
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)
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)
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)
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)
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)
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
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)
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)
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
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
Delamanid Deltyba® Otsuka Novel Products Infectious diseases Multidrug-resistant pulmonary tuberculosis (TBC), ≥ 10 kg 80–114 100% Hint for non-quantifiable additional benefit Orphan
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
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)
Vosoritid Voxzogo® BioMarin International Limited Other diseases Achondroplasia, ≥ 2 years 0
340–480
100% Hint for non-quantifiable additional benefit Orphan
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)
Odevixibat Bylvay® Albireo Pharma AB Digestive system diseases Cholestasis, ≥ 6 months 40–110 100% Hint for minor additional benefit Orphan
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
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
Selumetinib Koselugo® AstraZeneca GmbH Oncological diseases Neurofibromatosis (type 1), ≥ 3 to < 18 years 0
510–740
100% Hint for non-quantifiable additional benefit Orphan
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)
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
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)
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
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
Cabozantinib (7, reassessment) Cometriq® Ipsen Pharma GmbH Oncological diseases Thyroid carcinoma (MTC) 50–670 100% Hint for non-quantifiable additional benefit Orphan
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
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)
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)
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)
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)
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)
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)
Lanadelumab (2, reassessment >€50m) Takhzyro® Takeda GmbH Other diseases Hereditary angioedema (HAE), prophylaxis, ≥ 12 years 140–430 100% no additional benefit Orphan (turnover limit)
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)
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)
Obinutuzumab (5, reassessment >€50m) Gazyvaro® Roche Pharma AG Oncological diseases Chronic lymphocytic leukemia (CLL) 820–1,480 100% no additional benefit Orphan (turnover limit)
Obinutuzumab (4, reassessment >€50m) Gazyvaro® Roche Pharma AG Oncological diseases Follicular lymphoma (FL) 790–940 100% no additional benefit Orphan (turnover limit)
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
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
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
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
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
Imlifidase Idefirix® Hansa Biopharma AB Other diseases Desensitisation in kidney transplantation 3–69 100% Hint for non-quantifiable additional benefit Orphan
Fedratinib Inrebic® Celgene GmbH Oncological diseases Myelofibrosis (MF) 740–3,590
1,370–5,280
100% Hint for non-quantifiable additional benefit Orphan
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
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
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)
Fenfluramin Fintepla® Zogenix GmbH Nervous system diseases Dravet syndrome, ≥ 2 years 450–2,450 100% Hint for considerable additional benefit Orphan
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)
Afamelanotid (2, reassessment) Scenesse® Clinuvel (UK) Limited Metabolic diseases Erythropoietic protoporphyria 540–1,090 100% Hint for non-quantifiable additional benefit Orphan
Lumasiran Oxlumo® Alnylam Germany GmbH Metabolic diseases Hyperoxaluria 50–880 100% Hint for non-quantifiable additional benefit Orphan
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
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)
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)
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)
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)
Tafamidis (3, reassessment >€50m) Vyndaqel® Pfizer Pharma GmbH Metabolic diseases Amyloid Neuropathy 230 100% no additional benefit Orphan (turnover limit)
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)
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)
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)
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)
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)
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
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
Avapritinib Ayvakyt® Blueprint Medicines (Germany) Oncological diseases Gastrointestinal stromal tumor (GIST) 1–90 100% Hint for non-quantifiable additional benefit Orphan
Burosumab (3) Crysvita® Kyowa Kirin GmbH Metabolic diseases X-linked hypophosphatemia (XLH), ≥ 18 years 0
410–810
100% Hint for minor additional benefit Orphan
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
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
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)
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
Bulevirtid Hepcludex® MYR GmbH Infectious diseases Chronic hepatitis Delta 300–4,800 100% Hint for non-quantifiable additional benefit Orphan
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)
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)
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)
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)
Glasdegib Daurismo® Pfizer Pharma GmbH Oncological diseases Acute myeloid leukaemia (AML), combination with cytarabine (LDAC) 780–840 100% Hint for considerable additional benefit Orphan
Luspatercept (2) Reblozyl® Celgene GmbH Hematopoietic diseases Beta thalassemia 0
170–300
100% Hint for non-quantifiable additional benefit Orphan
Luspatercept Reblozyl® Celgene GmbH Hematopoietic diseases Myelodysplastic syndrome (MDS) 0
840–1,870
100% Hint for non-quantifiable additional benefit Orphan
Osilodrostat Isturisa® Recordati Rare Diseases Germany GmbH Metabolic diseases Endogenous Cushing syndrome 1,130–1,550 100% Hint for non-quantifiable additional benefit Orphan
Caplacizumab (2) Cablivi® Sanofi-Aventis Deutschland GmbH Hematopoietic diseases Thrombotic thrombocytopenic purpura (aTTP)Thrombotic thrombocytopenic purpura (TTP), 12 to < 18 years 2–3 100% Hint for non-quantifiable additional benefit Orphan
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)
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)
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)
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
Mogamulizumab Poteligeo® Kyowa Kirin GmbH Oncological diseases Mycosis Fungoides; Sézary Syndrome 310–460 100% Hint for non-quantifiable additional benefit Orphan
Ponatinib (3, reassessment) Iclusig® Incyte Biosciences Germany GmbH Oncological diseases Chronic myeloid leukaemia (CML) 500–940 100% Hint for non-quantifiable additional benefit Orphan
Ponatinib (2, reassessment) Iclusig® Incyte Biosciences Germany GmbH Oncological diseases Acute myeloid leukaemia (AML) 25–195 100% Hint for non-quantifiable additional benefit Orphan
Givosiran Givlaari® Alnylam Germany GmbH Metabolic diseases Acute hepatic porphyria, ≥ 12 years 1,000–1,700 100% Indication of considerable additional benefit Orphan
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
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
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)
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)
Tafamidis (2) Vyndaqel® Pfizer Pharma GmbH Metabolic diseases Amyloid cardiomyopathy 0
1,630–1,730
100% Hint for considerable additional benefit Orphan
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
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
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)
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)
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)
Gilteritinib XOSPATA® Astellas Pharma GmbH Oncological diseases Acute myeloid leukaemia (AML), FLT3 mutation 220–580 100% Hint for considerable additional benefit Orphan
Betibeglogene autotemcel Zynteglo® bluebird bio (Germany) GmbH Hematopoietic diseases Beta thalassemia 50 100% Hint for non-quantifiable additional benefit Orphan
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
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)
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
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)
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
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)
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)
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)
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)
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)
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)
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)
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)
Ibrutinib (6) Imbruvica® Janssen-Cilag GmbH Oncological diseases Waldenström's disease, combination with rituximab 590–1,180 100% no additional benefit Orphan (turnover limit)
Volanesorsen Waylivra® Akcea Therapeutics Germany GmbH Metabolic diseases Chylomicronaemia syndrome 60–120 100% Hint for non-quantifiable additional benefit Orphan
Pegvaliase Palynziq® BioMarin International Limited Metabolic diseases Phenylketonuria 435 100% Hint for non-quantifiable additional benefit Orphan
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)
Voretigen Neparvovec Luxturna® Novartis Pharma GmbH Eye diseases Hereditary retinal dystrophy 0
100–530
100% Hint for considerable additional benefit Orphan
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)
Brentuximab Vedotin (4) Adcetris® Takeda Pharma GmbH Oncological diseases Hodgkin lymphoma (HL), CD30+, first-line 220–380 100% non-quantifiable additional benefit Orphan
Blinatumomab (3) Blincyto® Amgen GmbH Oncological diseases B-cell acute lymphoblastic leukaemia (ALL), MRD-positive patients 40–110 100% non-quantifiable additional benefit Orphan
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
Mexiletin Namuscla® Lupin Europe GmbH Nervous system diseases Myotonia 530–650 100% non-quantifiable additional benefit Orphan
Lanadelumab Takhzyro® Shire Deutschland GmbH, Teil der Takeda Group Other diseases Hereditary angioedema (HAE) 0
140–430
100% considerable additional benefit Orphan
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
Bedaquilin Sirturo® Janssen-Cilag GmbH Infectious diseases Multidrug-resistant pulmonary tuberculosis 0
70–100
100% considerable additional benefit Orphan
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
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
Axicabtagen-Ciloleucel Yescarta® Kite, Gilead company Oncological diseases Diffuse large B-cell lymphoma (DLBCL) 0
440–700
100% non-quantifiable additional benefit Orphan
Patisiran Onpattro® Alnylam Germany GmbH Metabolic diseases Amyloidosis 0
350
100% considerable additional benefit Orphan
Inotersen Tegsedi® Akcea Therapeutics Germany GmbH Metabolic diseases Amyloidosis 350 100% non-quantifiable additional benefit Orphan
Metreleptin Myalepta® Aegerion Pharmaceuticals GmbH Metabolic diseases Lipodystrophy 90–180 100% non-quantifiable additional benefit Orphan
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
Caplacizumab Cablivi® Sanofi-Aventis Deutschland GmbH Hematopoietic diseases Thrombotic thrombocytopenic purpura (TTP) 150 100% non-quantifiable additional benefit Orphan
Vestronidase alfa Mepsevii® Ultragenyx Germany GmbH Metabolic diseases Mucopolysaccharidosis (MPS VII; Sly syndrome) 2–7 100% non-quantifiable additional benefit Orphan
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)
Tisagenlecleucel Kymriah® Novartis Pharma GmbH Oncological diseases Diffuse large B-cell lymphoma (DLBCL) 0
440–700
100% non-quantifiable additional benefit Orphan
Tisagenlecleucel (2) Kymriah® Novartis Pharma GmbH Oncological diseases B-cell acute lymphoblastic leukaemia (ALL) 0
50–65
100% non-quantifiable additional benefit Orphan
Gemtuzumab Ozogamicin Mylotarg® Pfizer Pharma GmbH Oncological diseases Acute myeloid leukaemia (AML) 560–1,150 100% non-quantifiable additional benefit Orphan
Velmanase alfa Lamzede® Chiesi GmbH Metabolic diseases Alpha mannosidosis 70–140 100% non-quantifiable additional benefit Orphan
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
Burosumab Crysvita® Kyowa Kirin GmbH Metabolic diseases X-linked hypophosphatemia (XLH), ≥ 1 to < 18 years 0
200–500
100% non-quantifiable additional benefit Orphan
Glycerolphenylbutyrat Ravicti® Swedish Orphan Biovitrum GmbH Metabolic diseases Urea cycle disorders (UCD), ≥ 2 months 100–250 100% non-quantifiable additional benefit Orphan
Letermovir Prevymis® MSD Sharp & Dohme GmbH Infectious diseases Cytomegalovirus infection 0
1,000–1,800
100% non-quantifiable additional benefit Orphan
Brentuximab Vedotin (3) Adcetris® Takeda Pharma GmbH Oncological diseases Cutaneous T-cell lymphoma, CD30+ 40–130 100% minor additional benefit Orphan
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
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
Cenegermin Oxervate® Dompé farmaceutici S.p.A Eye diseases Keratitis 1,730–3,170 100% non-quantifiable additional benefit Orphan
Telotristatethyl Xermelo® Ipsen Pharma GmbH Oncological diseases Carcinoid tumor, neuroendocrine tumors, combination with SAA therapy 300–1,000 100% non-quantifiable additional benefit Orphan
Midostaurin Rydapt® Novartis Pharma GmbH Oncological diseases Acute myeloid leukaemia (AML); Systemic mastocytosis (ASM) 0
400–710
80% considerable additional benefit Orphan
Obinutuzumab (3) Gazyvaro® Roche Pharma AG Oncological diseases Follicular lymphoma (FL), first-line 0
1,300–1,500
100% non-quantifiable additional benefit Orphan
Elosulfase alfa (2, reassessment) Vimizim® BioMarin Deutschland GmbH Metabolic diseases Mucopolysaccharidosis (type IVA) 20–100 100% minor additional benefit Orphan
Avelumab Bavencio® Merck Serono GmbH / Pfizer Pharma GmbH Oncological diseases Merkel-cell carcinoma (MCC) 0
160–410
100% non-quantifiable additional benefit Orphan
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)
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)
Inotuzumab Ozogamicin Besponsa® Pfizer Pharma GmbH Oncological diseases B-cell acute lymphoblastic leukaemia (ALL) 120–200 100% minor additional benefit Orphan
Nusinersen Spinraza® Biogen GmbH Nervous system diseases Spinal muscular atrophy (SMA) 0
840–1,060
10% major additional benefit Orphan
Cerliponase alfa Brineura® BioMarin Deutschland GmbH Metabolic diseases Neuronal ceroid lipofuscinosis type 2 0
20–40
100% non-quantifiable additional benefit Orphan
Blinatumomab (2, reassessment) Blincyto® Amgen GmbH Oncological diseases B-cell acute lymphoblastic leukaemia (ALL) 60–170 100% considerable additional benefit Orphan
Obeticholsäure Ocaliva® Intercept Pharma Deutschland GmbH Digestive system diseases Biliary cirrhosis 0
1,050–7,350
100% non-quantifiable additional benefit Orphan
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
Venetoclax Venclyxto® AbbVie Deutschland GmbH & Co. KG Oncological diseases Chronic lymphocytic leukaemia (CLL), monotherapy 0
300–700
100% non-quantifiable additional benefit Orphan
Olaratumab Lartruvo® Lilly Deutschland GmbH Oncological diseases Soft tissue sarcoma 0
1,200–1,400
100% considerable additional benefit Orphan
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)
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)
Teduglutid (2) Revestive® Shire Deutschland GmbH Digestive system diseases Short bowel syndrome, 1 to < 18 years 230–500 100% non-quantifiable additional benefit Orphan
Tasimelteon Hetlioz® Vanda Pharmceutical Inc Nervous system diseases Sleep disorders (sleep-wake rhythm), blind adults 7,000 100% non-quantifiable additional benefit Orphan
Pitolisant Wakix® Bioprojet Pharma SARL Nervous system diseases Narcolepsy 15,000–30,000 100% non-quantifiable additional benefit Orphan
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
Carfilzomib (2) Kyprolis® Amgen GmbH Oncological diseases Multiple myeloma (MM), combination with dexamethasone 0
4,700–7,000
100% minor additional benefit Orphan
Ibrutinib (3) Imbruvica® Janssen-Cilag GmbH Oncological diseases Chronic lymphocytic leukemia (CLL), first-line 2,840 100% no additional benefit Orphan (turnover limit)
Obinutuzumab (2) Gazyvaro® Roche Pharma AG Oncological diseases Follicular lymphoma (FL) 0
790–940
100% non-quantifiable additional benefit Orphan
Eftrenonacog alfa Alprolix® Swedish Orphan Biovitrum GmbH Hematopoietic diseases Hemophilia B 0
580–660
100% non-quantifiable additional benefit Orphan
Albutrepenonacog alfa Idelvion® CSL Behring GmbH Hematopoietic diseases Hemophilia B 0
580–660
100% non-quantifiable additional benefit Orphan
Migalastat Galafold® Amicus Therapeutics GmbH Metabolic diseases Fabry disease 0
20–490
100% non-quantifiable additional benefit Orphan
Ataluren (2, reassessment) Translarna® PTC Therapeutics International Limited Musculoskeletal system diseases Duchenne muscular dystrophy (DMD), ≥ 5 years 30–40 100% minor additional benefit Orphan
Daratumumab Darzalex® Janssen-Cilag GmbH Oncological diseases Multiple myeloma (MM), monotherapy, pre-treated patients 0
2,300
100% non-quantifiable additional benefit Orphan
Afamelanotid Scenesse® Clinuvel Ltd. Metabolic diseases Erythropoietic protoporphyria 0
540–1,090
100% non-quantifiable additional benefit Orphan
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)
Blinatumomab Blincyto® Amgen GmbH Oncological diseases B-cell acute lymphoblastic leukaemia (ALL) 0
60–170
100% non-quantifiable additional benefit Orphan
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
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
Isavuconazol Cresemba® Basilea Pharmaceutica International Ltd. Infectious diseases Aspergillosis, mucormycosis 1,293–5,342 100% non-quantifiable additional benefit Orphan
Sebelipase alfa Kanuma® Synageva BioPharma Limited Metabolic diseases Lysosomal acid lipase deficiency 0
31–843
100% non-quantifiable additional benefit Orphan
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)
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
Idebenon Raxone® Santhera Pharmaceuticals Eye diseases Leber hereditary optic neuropathy 0
1,500–3,000
100% non-quantifiable additional benefit Orphan
Asfotase alfa Strensiq® Alexion Europe SAS Metabolic diseases Hypophosphatasia (HPP) 0
1,000
100% non-quantifiable additional benefit Orphan
Lenvatinib Lenvima® Eisai GmbH Oncological diseases Thyroid carcinoma (DTC) 0
600–815
100% non-quantifiable additional benefit Orphan
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
Ruxolitinib (3) Jakavi® Novartis Pharma GmbH Oncological diseases Polycythaemia vera 240–1,470 100% Hint for considerable additional benefit Orphan (turnover limit)
Eliglustat Cerdelga® Genzyme GmbH Metabolic diseases Sphingolipidoses (Gaucher disease type 1) 150–500 100% non-quantifiable additional benefit Orphan
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
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
Pasireotid (2) Signifor® Novartis Pharma GmbH Metabolic diseases Acromegaly 265–1,133 100% minor additional benefit Orphan
Ataluren Translarna® PTC Therapeutics International Limited Musculoskeletal system diseases Duchenne muscular dystrophy (DMD), ≥ 5 years 0
82–110
100% minor additional benefit Orphan
Alipogentiparvovec Glybera® Chiesi GmbH Metabolic diseases Familial lipoprotein lipase deficiency 17–35 100% non-quantifiable additional benefit Orphan
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
Teduglutid Revestive® NPS Pharma Germany GmbH Digestive system diseases Short bowel syndrome 1,100–2,400 100% minor additional benefit Orphan
Ivacaftor (2) Kalydeco® Vertex Pharmaceuticals Limited Metabolic diseases Cystic fibrosis (CF), various mutations, ≥ 6 years 0
10–11
100% minor additional benefit Orphan
Obinutuzumab Gazyvaro® Roche Pharma AG Oncological diseases Chronic lymphocytic leukemia (CLL) 0
818–1,477
100% non-quantifiable additional benefit Orphan
Cabozantinib Cometriq® Swedish Orphan Biovitrum GmbH Oncological diseases Thyroid carcinoma (MTC) 0
60–500
100% minor additional benefit Orphan
Siltuximab Sylvant® Janssen-Cilag GmbH Oncological diseases Multicentric Castleman Disease (MCD) 130–1,460 100% non-quantifiable additional benefit Orphan
Elosulfase alfa Vimizim® BioMarin Deutschland GmbH Metabolic diseases Mucopolysaccharidosis (type IVA) 0
15–95
100% minor additional benefit Orphan
Cholsäure Orphacol® Laboratoires CTRS Metabolic diseases Bile acid synthesis disorder 10–25 100% non-quantifiable additional benefit Orphan
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)
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
Macitentan Opsumit® Actelion Pharmaceuticals Deutschland GmbH Cardiovascular diseases Pulmonary arterial hypertension (PAH) 0
580–7,850
100% minor additional benefit Orphan
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
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
Bosutinib Bosulif® Pfizer Pharma GmbH Oncological diseases Chronic myeloid leukaemia (CML) 0
380–500
100% non-quantifiable additional benefit Orphan
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
Decitabin Dacogen® Janssen-Cilag GmbH Oncological diseases Acute myeloid leukaemia (AML) 300–780 100% minor additional benefit Orphan
Ruxolitinib Jakavi® Novartis Pharma GmbH Oncological diseases Myelofibrosis (MF) 0
1,600
100% minor additional benefit Orphan
Ivacaftor Kalydeco® Vertex Pharmaceuticals GmbH Metabolic diseases Cystic fibrosis (CF), G551D mutation, ≥ 6 years 0
170
84% considerable additional benefit Orphan
Pasireotid Signifor® Novartis Pharma GmbH Metabolic diseases Pituitary dysfunction 160–360 100% minor additional benefit Orphan
Tafamidis Vyndaqel® Pfizer Pharma GmbH Metabolic diseases Amyloid Neuropathy 0
40–104
100% minor additional benefit Orphan
Pirfenidon Esbriet® InterMune Deutschland GmbH Respiratory system diseases Idiopathic pulmonary fibrosis 6,000 100% non-quantifiable additional benefit Orphan