Orphan designation: Adenovirus associated viral vector serotype 8 containing the human AIPL1 gene Treatment of Leber's congenital amaurosis, 12/12/2017 Positive
Orphan designation: Relacorilant Treatment of Cushing's syndrome, 29/05/2019 Positive
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