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: 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

Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 21-24 July 2025