Orphan designation: Relacorilant Treatment of pancreatic cancer, 21/08/2019 Positive

Orphan designation: Asp-Arg-Val-Tyr-Ile-His-Pro (Angiotensin 1-7),talfirastide Treatment of Duchenne muscular dystrophy, 19/02/2014 Positive

Orphan designation: Adenovirus associated viral vector serotype 8 containing the human CNGB3 gene Treatment of achromatopsia caused by mutations in the CNGB3 gene, 11/11/2015 Positive

Orphan designation: 2-Hydroxypropyl-β-cyclodextrin Treatment of Niemann-Pick's disease, type C, 26/04/2013 Positive

Orphan designation: Donidalorsen Treatment of hereditary angioedema, 19/02/2024 Positive

Orphan designation: 5-fluoro-4-(7'-fluoro-2'-methylspiro[cyclopentane-1,3'-indol]-5'-yl)-N-(5-(1-methylpiperidin-4-yl)pyridin-2-yl)pyrimidin-2-amine,crozbaciclib Treatment of glioma, 26/03/2021 Positive

Orphan designation: Asp-Arg-Val-Tyr-Ile-His-Pro (Angiotensin 1-7),talfirastide Treatment of epidermolysis bullosa, 20/06/2017 Positive

Orphan designation: Humanised monoclonal antibody targeting interleukin-15 Treatment of eosinophilic oesophagitis, 27/06/2016 Positive

Minutes of the CAT meeting 17-19 July 2024

Orphan designation: Anti-CD123 IgG1 humanised monoclonal antibody conjugated to N1-(2-(2,5-dioxo-2,5-dihydro-1H-pyrrol-1-yl)ethyl)-N6-((S)-1-(((S)-1-((3-((((S)-8-methoxy-6-oxo-11,12,12a,13-tetrahydro-6H-benzo[5,6][1,4]diazepino[1,2-a]indol-9-yl)oxy)methyl)-5-((((S)-8-methoxy-6-oxo-12a,13-dihydro-6Hbenzo[5,6][1,4]diazepino[1,2-a]indol-9-yl)oxy)methyl)phenyl)amino)-1-oxopropan-2-yl)amino)-1-oxopropan-2-yl)adipamide,pivekimab sunirine Treatment of blastic plasmacytoid dendritic cell neoplasm, 26/06/2020 Positive