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
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
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
Questions and answers on the outcome of assessment on use of Neuraceq to monitor treatment response
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
Orphan designation: bezuclastinib Treatment of mastocytosis, 20/06/2025 Positive
Orphan designation: Beta-lapachone Treatment of primary sclerosing cholangitis, 20/06/2025 Positive