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
Orphan designation: chimeric human-murine IgG1 kappa monoclonal antibody against TNF alfa Treatment of sarcoidosis, 25/07/2023 Positive
Orphan designation: tarperprumig Treatment of sickle cell disease, 25/07/2023 Positive
Orphan designation: mitapivat sulfate Treatment of thalassaemia intermedia and major, 13/10/2023 Positive
Orphan designation: Adeno-associated viral vector serotype 1 containing the 3' portion of human OTOF gene, Adeno-associated viral vector serotype 1 containing the 5' portion of human OTOF gene Treatment of otoferlin gene-mediated hearing loss, 20/03/2023 Positive
Orphan designation: Alpelisib Treatment of lymphatic malformations, 13/10/2023 Positive
Orphan designation: paclitaxel obaluronate Treatment of malignant mesothelioma, 25/07/2023 Positive
Orphan designation: Anagrelide hydrochloride monohydrate Treatment of gastrointestinal stromal tumours, 20/03/2023 Positive
Orphan designation: 6-(4-(1-amino-3-hydroxycyclobutyl)phenyl)-1-ethyl-7-phenyl-1H-pyrido[2,3-b][1,4]oxazin-2(3H)-one, L-tartrate salt Treatment of hereditary haemorrhagic telangiectasia, 20/03/2023 Positive