Human medicines European public assessment report (EPAR): Albrioza, sodium phenylbutyrate,Ursodoxicoltaurine, Date of refusal: 05/01/2024, Status: Refused

Orphan designation: vactosertib Treatment of osteosarcoma, 25/07/2023 Positive

Orphan designation: Ulefnersen Treatment of amyotrophic lateral sclerosis Positive

Orphan designation: Humanised IgG1 monoclonal antibody against TfR1 conjugated to exon 44 specific phosphorodiamidate morpholino oligonucleotide via a non-cleavable linker Treatment of Duchenne muscular dystrophy, 13/10/2023 Positive

Orphan designation: 2-(3-(3,5-Dimethyltriazol-4-yl)-5-((S)-oxan-4-yl(phenyl)methyl)pyrido(3,2-b)indol-7-yl)propan-2-ol Treatment of myelofibrosis, 13/10/2023 Positive

Orphan designation: zedenoleucel Treatment of acute myeloid leukaemia, 25/02/2023 Positive

Orphan designation: Autologous adipose-derived mesenchymal stem cells Treatment of oesophageal atresia, 21/04/2023 Positive

Orphan designation: Setmelanotide Treatment of acquired hypothalamic obesity, 13/10/2023 Positive

Orphan designation: (R)-(3-(2'-cyclopropyl-3-(hydroxymethyl)-[1,1'-biphenyl]-4-yl) pyrrolidin-1-yl)(5-fluoropyridin-2-yl)methanone Treatment of Olmsted syndrome, 13/10/2023 Positive

Orphan designation: sodium oxybate Treatment of narcolepsy, 13/10/2023 Positive