All resolutions concerning orphan drugs

Ivacaftor (11) Kalydeco® Vertex Pharmaceuticals Metabolic diseases METABOL ORPHAN Cystic fibrosis (CF) 100% Indication for non-quantifiable additional benefit
Gilteritinib XOSPATA® Astellas Pharma GmbH Oncological diseases ONCO ORPHAN Acute myeloid leukemia (AML) 100% Indication for considerable additional benefit
Betibeglogene autotemcel (autologe hämatopoetische Stammzellen) Zynteglo® bluebird bio (Germany) GmbH Hematopoietic diseases CV ORPHAN β-thalassemia 100% Indication for non-quantifiable additional benefit
Cannabidiol (2) Epidyolex® GW Pharmaceuticals plc Diseases of the nervous system CNS ORPHAN Dravet syndrome 100% Indication for non-quantifiable additional benefit
Niraparib (2, reassessment >€50m) Zejula® TESARO Bio Germany GmbH Oncological diseases ONCO ORPHAN Ovarian carcinoma / fallopian tube carcinoma / peritoneal carcinoma 100% no additional benefit
Cannabidiol Epidyolex® GW Pharmaceuticals plc Diseases of the nervous system CNS ORPHAN Lennox-Gastaut syndrome 100% Indication for non-quantifiable additional benefit
Asfotase alfa (2, reassessment >€50m) Strensiq® Alexion Pharma Germany GmbH Metabolic diseases METABOL ORPHAN Hypophosphatasia (HPP) 0.8% Indication for non-quantifiable additional benefit
Burosumab (2, reassessment) Crysvita® Kyowa Kirin GmbH Metabolic diseases METABOL ORPHAN Hypophosphatemia 100% Indication for non-quantifiable additional benefit
Ivacaftor (10, reassessment >€50m) Kalydeco® Vertex Pharmaceuticals (Europe) Limited Metabolic diseases METABOL ORPHAN Cystic fibrosis (CF) 100% Indication for non-quantifiable additional benefit
Ivacaftor (9, reassessment >€50m) Kalydeco® Vertex Pharmaceuticals (Europe) Limited Metabolic diseases METABOL ORPHAN Cystic fibrosis (CF) 100% Indication for minor additional benefit
Ivacaftor (8, reassessment >€50m) Kalydeco® Vertex Pharmaceuticals (Europe) Limited Metabolic diseases METABOL ORPHAN Cystic fibrosis (CF) 100% Indication for non-quantifiable additional benefit
Ivacaftor (7, reassessment >€50m) Kalydeco® Vertex Pharmaceuticals (Europe) Limited Metabolic diseases METABOL ORPHAN Cystic fibrosis (CF) 100% Indication for non-quantifiable additional benefit
Ivacaftor (6, reassessment >€50m) Kalydeco® Vertex Pharmaceuticals (Europe) Limited Metabolic diseases METABOL ORPHAN Cystic fibrosis (CF) 100% no additional benefit
Ivacaftor (5, reassessment >€50m) Kalydeco® Vertex Pharmaceuticals (Europe) Limited Metabolic diseases METABOL ORPHAN Cystic fibrosis (CF) 100% no additional benefit
Ivacaftor (4, reassessment >€50m) Kalydeco® Vertex Pharmaceuticals (Europe) Limited Metabolic diseases METABOL ORPHAN Cystic fibrosis (CF) 86% Indication for considerable additional benefit
Ramucirumab (5) Cyramza® Lilly Deutschland GmbH Oncological diseases ONCO ORPHAN Hepatocellular carcinoma (HC) 100% Proof of minor additional benefit
Ibrutinib (5) Imbruvica® Janssen-Cilag GmbH Oncological diseases ONCO ORPHAN Chronic lymphocytic leukemia (CLL) 26% Indication for minor additional benefit
Ibrutinib (6) Imbruvica® Janssen-Cilag GmbH Oncological diseases ONCO ORPHAN Waldenstroem's macroglobulinemia 100% no additional benefit
Volanesorsen Waylivra® Akcea Therapeutics Germany GmbH Metabolic diseases METABOL ORPHAN Familial chylomicronemia syndrome (FCS) 100% Indication for non-quantifiable additional benefit
Pegvaliase Palynziq® BioMarin International Limited Metabolic diseases METABOL ORPHAN Phenylketonuria (PKU) 100% Indication for non-quantifiable additional benefit
Pomalidomid (3) Imnovid® Celgene GmbH Oncological diseases ONCO ORPHAN Multiple myeloma (MM) 100% no additional benefit
Voretigen Neparvovec Luxturna® Novartis Pharma GmbH Eye diseases OTHER ORPHAN Retinal dystrophy 100% Indication for considerable additional benefit
Nintedanib (3, reassessment >€50m, Ofev®) Ofev® Boehringer Ingelheim Pharma GmbH & Co. KG Respiratory diseases METABOL ORPHAN Idiopathic pulmonary fibrosis 100% Indication for considerable additional benefit
Brentuximab Vedotin (4) Adcetris® Takeda Pharma GmbH Oncological diseases ONCO ORPHAN Hodgkin lymphoma (CD30+) 100% Hint for non-quantifiable additional benefit
Blinatumomab (3) Blincyto® Amgen GmbH Oncological diseases ONCO ORPHAN Acute lymphoblastic leukemia (ALL) 100% Hint for non-quantifiable additional benefit
Blinatumomab (4) Blincyto® Amgen GmbH Oncological diseases ONCO ORPHAN Acute lymphoblastic leukemia (ALL) 100% non-quantifiable additional benefit
Mexiletin Namuscla® Lupin Europe GmbH Diseases of the nervous system CNS ORPHAN Myotonia 100% Hint for non-quantifiable additional benefit
Lanadelumab Takhzyro® Shire Deutschland GmbH, Teil der Takeda Group Other OTHER ORPHAN Hereditary angioedema (HAE) 100% Hint for considerable additional benefit
Glycerolphenylbutyrat (2) Ravicti® Swedish Orphan Biovitrum GmbH Metabolic diseases METABOL ORPHAN Urea cycle disorders (UCD) 100% Indication for non-quantifiable additional benefit
Bedaquilin Sirturo® Janssen-Cilag GmbH Infectious diseases INFECT ORPHAN Tuberculosis 100% Hint for considerable additional benefit
Tezacaftor / Ivacaftor Symkevi® Vertex Pharmaceuticals (Europe) Limited Metabolic diseases METABOL ORPHAN Cystic fibrosis (CF) 91% Hint for considerable additional benefit
Axicabtagen-Ciloleucel (2) Yescarta® Kite, Gilead company Oncological diseases ONCO ORPHAN Diffuse large B-cell lymphoma (DLBCL) 100% Hint for non-quantifiable additional benefit
Axicabtagen-Ciloleucel Yescarta® Kite, Gilead company Oncological diseases ONCO ORPHAN Primary medial major cell B-lymphoma (PMBCL) 100% Hint for non-quantifiable additional benefit
Patisiran Onpattro® Alnylam Germany GmbH Metabolic diseases METABOL ORPHAN Amyloidosis 100% considerable additional benefit
Inotersen Tegsedi® Akcea Therapeutics Germany GmbH Metabolic diseases METABOL ORPHAN Amyloidosis 100% non-quantifiable additional benefit
Metreleptin Myalepta® Aegerion Pharmaceuticals GmbH Metabolic diseases METABOL ORPHAN Lipodystrophy 100% Hint for non-quantifiable additional benefit
Daunorubicin / Cytarabin Vyxeos® Jazz Pharmaceuticals Oncological diseases ONCO ORPHAN Acute myeloid leukemia (AML) 100% Hint for considerable additional benefit
Caplacizumab Cablivi® Sanofi-Aventis Deutschland GmbH Hematopoietic diseases CV ORPHAN Acquired thrombotic thrombocytopenic purpura (aTTP) 100% non-quantifiable additional benefit
Vestronidase alfa Mepsevii® Ultragenyx Germany GmbH Metabolic diseases METABOL ORPHAN Mucopolysaccharidosis 100% non-quantifiable additional benefit
Daratumumab (3) Darzalex® Janssen-Cilag GmbH Oncological diseases ONCO ORPHAN Multiple myeloma (MM) 100% Indication for considerable additional benefit
Tisagenlecleucel Kymriah® Novartis Pharma GmbH Oncological diseases ONCO ORPHAN Diffuse large B-cell lymphoma (DLBCL) 100% non-quantifiable additional benefit
Tisagenlecleucel (2) Kymriah® Novartis Pharma GmbH Oncological diseases ONCO ORPHAN Acute lymphoblastic leukemia (ALL) 100% Indication for non-quantifiable additional benefit
Gemtuzumab Ozogamicin Mylotarg® Pfizer Pharma GmbH Oncological diseases ONCO ORPHAN Acute myeloid leukemia (AML) 100% non-quantifiable additional benefit
Velmanase alfa Lamzede® Chiesi GmbH Metabolic diseases METABOL ORPHAN α mannosidosis 100% Indication for non-quantifiable additional benefit
Darvadstrocel Alofisel® Takeda Pharma Vertrieb GmbH & Co. KG Diseases of the digestive system OTHER ORPHAN Morbus Crohn associated perianal fistulas 100% non-quantifiable additional benefit
Carfilzomib (3, reassessment >€50m) Kyprolis® Amgen GmbH Oncological diseases ONCO ORPHAN Multiple myeloma (MM) 100% Indication for considerable additional benefit
Burosumab Crysvita® Kyowa Kirin GmbH Metabolic diseases METABOL ORPHAN Hypophosphatemic rickets (XLH) 100% Hint for non-quantifiable additional benefit
Glycerolphenylbutyrat Ravicti® Swedish Orphan Biovitrum GmbH Metabolic diseases METABOL ORPHAN Urea cycle disorders (UCD) 100% Hint for non-quantifiable additional benefit
Letermovir Prevymis® MSD Sharp & Dohme GmbH Infectious diseases INFECT ORPHAN Cytomegalovirus (CMV) prevention 100% non-quantifiable additional benefit
Brentuximab Vedotin (3) Adcetris® Takeda Pharma GmbH Oncological diseases ONCO ORPHAN Non-Hodgkin lymphoma (CD30+) 100% minor additional benefit
Allogene, genetisch modifizierte T-Zellen Zalmoxis® Dompé farmaceutici S.p.A Oncological diseases OTHER ORPHAN Hematopoietic stem cell transplantation, add-on therapy 100% Hint for non-quantifiable additional benefit
Niraparib Zejula® TESARO Bio Germany GmbH Oncological diseases ONCO ORPHAN Ovarian carcinoma 100% non-quantifiable additional benefit
Cenegermin Oxervate® Dompé farmaceutici S.p.A Eye diseases OTHER ORPHAN Neurotrophic keratitis 100% non-quantifiable additional benefit
Telotristatethyl Xermelo® Ipsen Pharma GmbH Oncological diseases OTHER ORPHAN Carcinoid diarrhea 100% non-quantifiable additional benefit
Midostaurin Rydapt® Novartis Pharma GmbH Oncological diseases ONCO ORPHAN Acute myeloid leukemia (AML), Mastocytosis (ASM) 100% non-quantifiable additional benefit
Obinutuzumab (3) Gazyvaro® Roche Pharma AG Oncological diseases ONCO ORPHAN Follicular lymphoma (FL) 100% non-quantifiable additional benefit
Elosulfase alfa (2, reassessment) Vimizim® BioMarin Deutschland GmbH Metabolic diseases METABOL ORPHAN Mucopolysaccharidosis 100% minor additional benefit
Avelumab Bavencio® Merck Serono GmbH / Pfizer Pharma GmbH Oncological diseases ONCO ORPHAN Merkel cell carcinoma (MCC) 70% Hint for non-quantifiable additional benefit
Daratumumab (2, reassessment >€50m) Darzalex® Janssen-Cilag GmbH Oncological diseases ONCO ORPHAN Multiple myeloma (MM) 72% Hint for considerable additional benefit
Inotuzumab Ozogamicin Besponsa® Pfizer Pharma GmbH Oncological diseases ONCO ORPHAN Acute lymphoblastic leukemia (ALL) 100% minor additional benefit
Nusinersen Spinraza® Biogen GmbH Diseases of the nervous system METABOL ORPHAN Spinal muscular atrophy (SMA) 10% major additional benefit
Cerliponase alfa Brineura® BioMarin Deutschland GmbH Metabolic diseases METABOL ORPHAN Neuronal ceroid lipofuscinosis (CNL2) 100% non-quantifiable additional benefit
Blinatumomab (2, reassessment) Blincyto® Amgen GmbH Oncological diseases ONCO ORPHAN Acute lymphoblastic leukemia (ALL) 100% considerable additional benefit
Obeticholsäure Ocaliva® Intercept Pharma Deutschland GmbH Diseases of the digestive system METABOL ORPHAN Hepatic cirrhosis 100% non-quantifiable additional benefit
Ixazomib Ninlaro® Takeda Pharma GmbH Oncological diseases ONCO ORPHAN Multiple myeloma (MM) 100% non-quantifiable additional benefit
Venetoclax Venclyxto® AbbVie Deutschland GmbH & Co. KG Oncological diseases ONCO ORPHAN Chronic lymphocytic leukemia (CLL) 100% non-quantifiable additional benefit
Olaratumab Lartruvo® Lilly Deutschland GmbH Oncological diseases ONCO ORPHAN Soft tissue sarcoma 100% considerable additional benefit
Macitentan (2, reassessment >€50m) Opsumit® Actelion Pharmaceuticals Deutschland GmbH Cardiovascular diseases CV ORPHAN Pulmonary hypertension (PHT) 100% no additional benefit
Ibrutinib (4) Imbruvica® Janssen-Cilag GmbH Oncological diseases ONCO ORPHAN Chronic lymphocytic leukemia (CLL) 50% Indication for considerable additional benefit
Teduglutid (2) Revestive® Shire Deutschland GmbH Diseases of the digestive system METABOL ORPHAN Short bowel syndrome 100% non-quantifiable additional benefit
Tasimelteon Hetlioz® Vanda Pharmceutical Inc Diseases of the nervous system CNS ORPHAN Non-24-hour sleep–wake disorder 100% non-quantifiable additional benefit
Pitolisant Wakix® Bioprojet Pharma SARL Diseases of the nervous system CNS ORPHAN Narcolepsy 100% non-quantifiable additional benefit
Brentuximab Vedotin (2) Adcetris® Takeda Pharma GmbH Oncological diseases ONCO ORPHAN Hodgkin lymphoma (CD30+) 100% non-quantifiable additional benefit
Carfilzomib (2) Kyprolis® Amgen GmbH Oncological diseases ONCO ORPHAN Multiple myeloma (MM) 100% minor additional benefit
Ibrutinib (3) Imbruvica® Janssen-Cilag GmbH Oncological diseases ONCO ORPHAN Chronic lymphocytic leukemia (CLL) 100% no additional benefit
Obinutuzumab (2) Gazyvaro® Roche Pharma AG Oncological diseases ONCO ORPHAN Follicular lymphoma (FL) 100% non-quantifiable additional benefit
Eftrenonacog alfa Alprolix® Swedish Orphan Biovitrum GmbH Hematopoietic diseases CV ORPHAN Hemophilia B 100% non-quantifiable additional benefit
Albutrepenonacog alfa Idelvion® CSL Behring GmbH Hematopoietic diseases CV ORPHAN Hemophilia B 100% non-quantifiable additional benefit
Migalastat Galafold® Amicus Therapeutics GmbH Metabolic diseases OTHER ORPHAN Morbus Fabry (alpha-galactosidase insuffiency) 100% non-quantifiable additional benefit
Ataluren (2, reassessment) Translarna® PTC Therapeutics International Limited Diseases of the musculoskeletal diseases OTHER ORPHAN Duchenne muscle dystrophia 100% minor additional benefit
Daratumumab Darzalex® Janssen-Cilag GmbH Oncological diseases ONCO ORPHAN Multiple myeloma (MM) 100% non-quantifiable additional benefit
Afamelanotid Scenesse® Clinuvel Ltd. Metabolic diseases METABOL ORPHAN Erythropoietic protoporphyria (EPP) 100% non-quantifiable additional benefit
Ibrutinib (2, reassessment >€50m) Imbruvica® Janssen-Cilag GmbH Oncological diseases ONCO ORPHAN Mantle cell lymphoma (MCL), Chronic lymphatic leukemia (CLL) 4% Hint for considerable additional benefit
Blinatumomab Blincyto® Amgen GmbH Oncological diseases ONCO ORPHAN Acute lymphoblastic leukemia (ALL) 100% non-quantifiable additional benefit
Carfilzomib Kyprolis® Amgen GmbH Oncological diseases ONCO ORPHAN Multiple myeloma (MM) 100% non-quantifiable additional benefit
Ivacaftor (3) Kalydeco® Vertex Pharmaceuticals (Europe) Limited Metabolic diseases METABOL ORPHAN Cystic fibrosis (CF) 75% Hint for minor additional benefit
Isavuconazol Cresemba® Basilea Pharmaceutica International Ltd. Infectious diseases INFECT ORPHAN Invasive aspergillosis and mucormycosis 100% non-quantifiable additional benefit
Sebelipase alfa Kanuma® Synageva BioPharma Limited Metabolic diseases METABOL ORPHAN Lysosomal lipase acid deficiency (LAL) 100% non-quantifiable additional benefit
Pomalidomid (2, reassessment >€50m) Imnovid® Celgene GmbH Oncological diseases ONCO ORPHAN Multiple myeloma (MM) 50% Indication for considerable additional benefit
Panobinostat Farydak® Novartis Pharma GmbH Oncological diseases ONCO ORPHAN Multiple myeloma (MM) 100% non-quantifiable additional benefit
Idebenon Raxone® Santhera Pharmaceuticals Eye diseases OTHER ORPHAN Leber hereditial optic neuropathy (LHON) 100% non-quantifiable additional benefit
Asfotase alfa Strensiq® Alexion Europe SAS Metabolic diseases METABOL ORPHAN Hypophosphatasia (HPP) 50% Hint for non-quantifiable additional benefit
Lenvatinib Lenvima® Eisai GmbH Oncological diseases ONCO ORPHAN Differentiated thyroid cancer (DTC) 100% non-quantifiable additional benefit
Olaparib Lynparza® AstraZeneca GmbH Oncological diseases ONCO ORPHAN Ovarian carcinoma 100% non-quantifiable additional benefit
Eliglustat Cerdelga® Genzyme GmbH Metabolic diseases METABOL ORPHAN Morbus Gaucher type 1 100% non-quantifiable additional benefit
Nintedanib (2, Ofev®) Ofev® Boehringer Ingelheim Pharma GmbH & Co. KG Respiratory diseases METABOL ORPHAN Idiopathic pulmonary fibrosis 100% Hint for minor additional benefit
Ramucirumab Cyramza® Lilly Deutschland GmbH Oncological diseases ONCO ORPHAN Advanced gastric cancer (adenocarcinoma) 100% Hint for minor additional benefit
Pasireotid (2) Signifor® Novartis Pharma GmbH Metabolic diseases METABOL ORPHAN Acromegalia 100% minor additional benefit
Ataluren Translarna® PTC Therapeutics International Limited Diseases of the musculoskeletal diseases METABOL ORPHAN Duchenne muscle dystrophia 100% minor additional benefit
Alipogentiparvovec Glybera® Chiesi GmbH Metabolic diseases METABOL ORPHAN Lipoprotein deficiency 100% non-quantifiable additional benefit
Ibrutinib Imbruvica® Janssen-Cilag GmbH Oncological diseases ONCO ORPHAN Mantle cell lymphoma (MCL), Chronic lymphatic leukemia (CLL) 4% Hint for non-quantifiable additional benefit
Teduglutid Revestive® NPS Pharma Germany GmbH Diseases of the digestive system METABOL ORPHAN Malabsorption syndrome 100% minor additional benefit
Ivacaftor (2) Kalydeco® Vertex Pharmaceuticals Limited Metabolic diseases METABOL ORPHAN Cystic fibrosis (CF) 100% Hint for minor additional benefit
Obinutuzumab Gazyvaro® Roche Pharma AG Oncological diseases ONCO ORPHAN Chronic lymphocytic leukemia (CLL) 100% Hint for non-quantifiable additional benefit
Cabozantinib Cometriq® Swedish Orphan Biovitrum GmbH Oncological diseases ONCO ORPHAN Medullary thyroid cancer (MTC) 100% minor additional benefit
Siltuximab Sylvant® Janssen-Cilag GmbH Oncological diseases ONCO ORPHAN Multicentric Castleman Disease (MCD) 100% non-quantifiable additional benefit
Elosulfase alfa Vimizim® BioMarin Deutschland GmbH Metabolic diseases METABOL ORPHAN Mucopolysaccharidosis / Morquio A syndrome 100% minor additional benefit
Cholsäure Orphacol® Laboratoires CTRS Metabolic diseases METABOL ORPHAN Heritage dysbalance of primary bilic acid synthesis 100% non-quantifiable additional benefit
Ruxolitinib (2, reassessment >€50m) Jakavi® Novartis Pharma GmbH Oncological diseases ONCO ORPHAN Chronic myeloproliferative disease (CML) 100% Indication for considerable additional benefit
Riociguat Adempas® Bayer Vital GmbH Cardiovascular diseases CV ORPHAN Pulmonary hypertension (PHT) 100% minor additional benefit
Macitentan Opsumit® Actelion Pharmaceuticals Deutschland GmbH Cardiovascular diseases CV ORPHAN Pulmonary hypertension (PHT) 100% minor additional benefit
Pomalidomid Imnovid® Celgene GmbH Oncological diseases ONCO ORPHAN Multiple myeloma (MM) 100% considerable additional benefit
Ponatinib Iclusig® ARIAD Pharmaceuticals (Germany) GmbH Oncological diseases ONCO ORPHAN Chronic myeloic leukemia (CML), Acute lymphoblastic leukemia (Ph+ ALL) 100% Hint for non-quantifiable additional benefit
Bosutinib Bosulif® Pfizer Pharma GmbH Oncological diseases ONCO ORPHAN Chronic myeloid leukemia (CML) 100% Hint for non-quantifiable additional benefit
Brentuximab Vedotin Adcetris® Takeda Pharma Vertrieb GmbH & Co. KG Oncological diseases ONCO ORPHAN Hodgkin lymphoma (CD30+), Anaplastic large-cell lymphoma 68% Hint for non-quantifiable additional benefit
Decitabin Dacogen® Janssen-Cilag GmbH Oncological diseases ONCO ORPHAN Acute myeloid leukemia (AML) 100% minor additional benefit
Ruxolitinib Jakavi® Novartis Pharma GmbH Oncological diseases ONCO ORPHAN Chronic myeloproliferative diseases (CML) 100% Hint for minor additional benefit
Ivacaftor Kalydeco® Vertex Pharmaceuticals GmbH Metabolic diseases METABOL ORPHAN Cystic fibrosis (CF) 84% considerable additional benefit
Pasireotid Signifor® Novartis Pharma GmbH Metabolic diseases METABOL ORPHAN Dysfunction of pituitary gland 100% minor additional benefit
Tafamidis Vyndaqel® Pfizer Pharma GmbH Other METABOL ORPHAN Amyloidosis 100% minor additional benefit
Pirfenidon Esbriet® InterMune Deutschland GmbH Respiratory diseases METABOL ORPHAN Idiopathic pulmonary fibrosis 100% non-quantifiable additional benefit