Table 2 Drugs with EMA orphan designation approved from Jan 2015 to Mar 2020
Generic name | Brand name | Indication | Estimated incidence of indication1 |
|---|---|---|---|
Asfotase Alfa | Strensiq | Hypophosphatasia | 1–5/10,000 |
Avelumab | Bavencio | Merkel Cell Carcinoma | 1–2/500,000 |
Axicabtagene Ciloleucel | Yescarta | Primary Mediastinal Large B-Cell Lymphoma, Large B-Cell Lymphoma | 1–5/10,000 |
Budesonide | Jorveza | Eosinophilic Esophagitis | 1–5/10,000 |
Burosumab | Crysvita | Hypophosphatemia (X-Linked) | 1–9/1,000,000 |
Cannabidiol | Epidyolex | Seizures (Lennox-Gastaut Syndrome), Dravet Syndrome | 1–5/10,000 |
Caplacizumab | Cablivi | Thrombotic Thrombocytopenic Purpura | 1/77,000 (France, less prevalent globally) |
cenegermin-bkbj | Oxervate | Neurotrophic keratitis | 1–5/10,000 |
Cerliponase Alfa | Brineura | Neuronal Ceroid Lipofuscinosis Type 2 | 1/ > 50,000 |
Chlormethine | Ledaga | Cutaneous T-Cell Lymphoma | 1–5/10,000 |
Coagulation Factor Ix | Idelvion | Haemophilia B | 1–9/100,000 |
Coagulation Factor Ix | Alprolix | Haemophilia B | 1–9/100,000 |
Coagulation Factor X | Coagadex | Factor X Deficiency | 1–9/1,000,000 |
Cytarabine & Daunorubicin | Vyxeos | Acute Myeloid Leukemia | 1–5/10,000 |
Daratumumab | Darzalex | Multiple Myeloma | 1–5/10,000 |
Darvadstrocel | Alofisel | Crohn's Disease (Fistulising) | 1–5/10,000 |
Dinutuximab Beta | Unituxin | Neuroblastoma | 1–5/10,000 |
Eliglustat | Cerdelga | Gaucher Disease | 1–9/100,000 |
Gallium (68 Ga) Edotreotide | Somakit Toc | Diagnostic Use ((Gastroenteropancreatic Neuroendocrine Tumours) | 1–5/10,000 |
Gemtuzumab Ozogamicin | Mylotarg | Acute Myeloid Leukemia | 1–5/10,000 |
Gilteritinib | Xospata | Acute Myeloid Leukemia (Flt3 +) | 1–5/10,000 |
Givosiran | Givlaari | Acute hepatic porphyria | 1–5/10,000 |
Glibenclamide | Amglidia | Neonatal Diabetes | 1/300,000 |
Glycerol Phenylbutyrate | Ravicti | Urea Cycle Disorder | 1–5/10,000 |
Idebenone | Raxone | Leber's Hereditary Optic Neuropathy | 1–9/100,000 |
Inotersen | Tegsedi | Transthyretin Amyloidosis | 1–5/10,000 |
Inotuzumab ozogamicin | Besponsa | Acute lymphoblastic leukemia | 1–5/10,000 |
Isavuconazole | Cresemba | Invasive Aspergillosis and Mucormycosis | 1–9/100,000 |
Ivacaftor & Lumacaftor | Orkambi | Cystic Fibrosis | 1–9/100,000 |
Ivacaftor & Tezacaftor | Symdeko | Cystic Fibrosis | 1–9/100,000 |
Ixazomib | Ninlaro | Multiple Myeloma | 1–5/10,000 |
Lanadelumab | Takhzyro | Hereditary Angioedema | 1–9/100,000 |
larotrectinib | Vitrakvi | cancers with NTRK fusion | 1–9/100,000 |
Letermovir | Prevymis | Cytomegalovirus Infection | 1–5/10,000 |
Limbal Stems Cells, Autologous | Holoclar | Limbal Stem Cell Deficiency | 1–5/10,000 |
Lutetium (177Lu) Oxodotreotide | Lutathera | Neuroendocrine Tumors (Gastroenteropancreatic) | 1–5/10,000 |
Mercaptamine | Cystadrops | Cystinosis | 1–9/100,000 |
Metreleptin | Myalepta | Lipodystrophy | 1–9/1,000,000 |
Mexiletine | Namuscla | Myotonia | 1–9/100,000 |
Midostaurin | Rydapt | Acute Myeloid Leukemia | 1–5/10,000 |
Midostaurin | Rydapt | Aggressive Systemic Mastocytosis | 1–9/1,000,000 |
Migalastat | Galafold | Fabry Disease | 1–5/10,000 |
Nintedanib | Ofev | Idiopathic Pulmonary Fibrosis | 1–5/10,000 |
Niraparib | Zejula | Ovarian Cancer | 1–5/10,000 |
Nusinersen | Spinraza | Spinal Muscular Atrophy |  < 1/1,000,000 |
Obeticholic Acid | Ocaliva | Primary Biliary Cirrhosis | 1–5/10,000 |
Other Antineoplastic Agents | Zalmoxis | Haematological Malignancy (Haploidentical HSCT) | 1–5/10,000 |
Panobinostat | Farydak | Multiple Myeloma | 1–5/10,000 |
Parathyroid Hormone | Natpara | Hypoparathyroidism |  < 1/1,000,000 |
Patisiran | Onpattro | Transthyretin Amyloidosis | 1–5/10,000 |
Pegvaliase | Palynziq | Phenylketonuria | 1/15,000 |
Pitolisant | Wakix | Narcolepsy | 1–5/10,000 |
Polatuzumab Vedotin | Polivy | Diffuse Large B-Cell Lymphoma | 1–5/10,000 |
Sebelipase Alfa | Kanuma | Lysosomal Acid Lipase Deficiency | 1–9/100,000 |
Tagraxofusp | Elzonris | Blastic Plasmacytoid Dendritic Cell Neoplasm | 1–5/10,000 |
Tasimelteon | Hetlioz | Non-24 h sleep–wake syndrome |  < 1/1,000,000 |
Telotristat | Xermelo | Diarrhea (Carcinoid Syndrome) | 1–9/100,000 |
Tisagenlecleucel | Kymriah | Large B-Cell Lymphoma | 1–5/10,000 |
Transduced Cd34 + Cell | Strimvelis | Severe Combined Immunodeficiency (Adenosine Deaminase Deficiency) | 1–9/1,000,000 |
Velmanase Alfa | Lamzede | Alpha Mannosidosis | 1–9/1,000,000 |
Vestronidase Alfa | Mepsevii | Mucopolysaccharidosis VII |  < 1/1,000,000 |
Volanesorsen | Waylivra | Familial Chylomicronemia Syndrome | 1–9/1,000,000 |
Voretigene Neparvovec | Luxturna | Leber's Congenital Amaurosis (Rpe65) | 1–9/100,000 |