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
Orphan designation: messenger ribonucleic acid coding for coiled-coil domain-containing protein 40 Treatment of primary ciliary dyskinesia, 25/07/2023 Positive
Concept paper for the development of a reflection paper on a tailored clinical approach in biosimilar development
Orphan designation: Nomacopan Treatment in haematopoietic stem cell transplantation, 25/07/2023 Positive
Orphan designation: bezuclastinib Treatment of gastrointestinal stromal tumours, 20/03/2023 Positive