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Orphan designations
Get the latest updates from Orphan designations directly as they happen.
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Last updated about 1 month ago
Orphan designation: Humanised IgG1 monoclonal antibody against adrenocorticotropic hormone Treatment of congenital adrenal hyperplasia, 20/06/2025 Positive
about 1 month ago
Orphan designation: Humanised IgG1 monoclonal antibody against adrenocorticotropic hormone Treatment of congenital...
Orphan designation: 4-Amino-1-[(2R,4S,5S)-5-(1,2-dihydroxyethyl)-4-hydroxyoxolan-2-yl]pyrimidin-2-one Treatment of glioma, 20/06/2025 Positive
about 1 month ago
Orphan designation: 4-Amino-1-[(2R,4S,5S)-5-(1,2-dihydroxyethyl)-4-hydroxyoxolan-2-yl]pyrimidin-2-one Treatment of glioma, 20/06/2025 Positive
Orphan designation: empasiprubart Treatment of multifocal motor neuropathy, 20/06/2025 Positive
about 1 month ago
Orphan designation: empasiprubart Treatment of multifocal motor neuropathy, 20/06/2025 Positive
Orphan designation: Exidavnemab Treatment of multiple system atrophy, 20/06/2025 Positive
about 1 month ago
Orphan designation: Exidavnemab Treatment of multiple system atrophy, 20/06/2025 Positive
Orphan designation: Alpha-N-acetyl glucosaminidase fused to a humanised monoclonal antibody against transferrin receptor Treatment of mucopolysaccharidosis type IIIB (Sanfilippo B syndrome), 20/06/2025 Positive
about 1 month ago
Orphan designation: Alpha-N-acetyl glucosaminidase fused to a humanised monoclonal antibody against transferrin...
Orphan designation: 2-[[Hydroxy[(R)-2-[((5Z,8Z,11Z,14Z)-eicosa-5,8,11,14-tetraenoyl)oxy]-3-(octadecyloxy)propoxy]phosphoryl]oxy]ethan-1-amine Treatment of Rett syndrome, 20/06/2025 Positive
about 1 month ago
Orphan designation: 2-[[Hydroxy[(R)-2-[((5Z,8Z,11Z,14Z)-eicosa-5,8,11,14-tetraenoyl)oxy]-3-(octadecyloxy)propoxy]phosphoryl]oxy]ethan-1-amine Treatment of Rett syndrome, 20/06/2025 Positive
Orphan designation: Dipalmitoyl hydroxyproline Treatment of Netherton syndrome, 20/06/2025 Positive
about 1 month ago
Orphan designation: Dipalmitoyl hydroxyproline Treatment of Netherton syndrome, 20/06/2025 Positive
Orphan designation: Humanised IgG4 bispecific monoclonal antibody against sclerostin and dickkopf-related protein 1 Treatment of osteogenesis imperfecta, 20/06/2025 Positive
about 1 month ago
Orphan designation: Humanised IgG4 bispecific monoclonal antibody against sclerostin and dickkopf-related protein...
Orphan designation: bezuclastinib Treatment of mastocytosis, 20/06/2025 Positive
about 1 month ago
Orphan designation: bezuclastinib Treatment of mastocytosis, 20/06/2025 Positive
Orphan designation: Beta-lapachone Treatment of primary sclerosing cholangitis, 20/06/2025 Positive
about 1 month ago
Orphan designation: Beta-lapachone Treatment of primary sclerosing cholangitis, 20/06/2025 Positive
Orphan designation: 1-(3-Bromobenzyl)-N3-(3,4-dichlorophenyl)-1H-1,2,4-triazole-3,5-diamine Treatment of radiation induced maculopathy, 20/06/2025 Positive
about 1 month ago
Orphan designation: 1-(3-Bromobenzyl)-N3-(3,4-dichlorophenyl)-1H-1,2,4-triazole-3,5-diamine Treatment of radiation induced maculopathy, 20/06/2025 Positive
Orphan designation: 3-(Carbamoylamino)-5-[2-(3-fluorophenyl)ethynyl]-N-[(3S)-piperidin-3-yl]thiophene-2-carboxamide hydrochloric acid Treatment of acute myeloid leukaemia, 20/06/2025 Positive
about 1 month ago
Orphan designation: 3-(Carbamoylamino)-5-[2-(3-fluorophenyl)ethynyl]-N-[(3S)-piperidin-3-yl]thiophene-2-carboxamide hydrochloric acid Treatment of acute myeloid leukaemia, 20/06/2025 Positive