Orphan designation: lepunafusp alfa Treatment of mucopolysaccharidosis type I, 26/03/2021 Positive
Orphan designation: (2S,4R)-1-(2-(3-acetyl-5-(2-methylpyrimidine-5-yl)-1H-indazol-1-yl)acetyl)-N-(6-bromopyridine-2-yl)-4-fluoropyrrolidine-2-carboxamide Treatment of C3 glomerulopathy, 21/03/2018 Withdrawn
Orphan designation: utreloxastat Treatment of amyotrophic lateral sclerosis, 09/12/2022 Withdrawn
Orphan designation: Obecabtagene autoleucel Treatment of acute lymphoblastic leukaemia, 13/04/2022 Withdrawn
Orphan designation: diflunisal Treatment of ATTR amyloidosis, 21/06/2022 Withdrawn
Orphan designation: iloprost Treatment of systemic sclerosis, 20/06/2025 Positive
Orphan designation: Humanised monoclonal antibody targeting B-cell maturation antigen conjugated with maleimidocaproyl monomethyl auristatin F Treatment of multiple myeloma, 16/10/2017 Withdrawn
Orphan designation: (R)-3-(5-dimethylcarbamoyl-pent-1-enyl)-N-(2-hydroxy-1methylethyl)benzamide Treatment of fragile X syndrome Positive
Orphan designation: 2-(2-(2-[2-(4-Benzothiazol-2-yl-phenoxy)-ethoxy]-ethoxy)-ethoxy)-ethanol Treatment of amyotrophic lateral sclerosis, 20/06/2025 Positive
Orphan designation: sodium 2-(3'(-3-(1-(4-(tert-butyl)benzyl)-4-ethyl-5-oxo-4,5-dihydro-1H-1,2,4-triazol-3-yl)propyl)-4-ethoxy-[1,1'-biphenyl]-3-yl)acetate Treatment of hepatocellular carcinoma, 20/06/2025 Positive