Orphan designation: Dexamethasone sodium phosphate encapsulated in human erythrocytes Treatment of cystic fibrosis, 20/10/2004 Withdrawn
Orphan designation: Dexamethasone sodium phosphate encapsulated in human autologous erythrocytes Treatment of ataxia telangiectasia, 17/07/2013 Withdrawn
Orphan designation: tazemetostat Treatment of diffuse large B-cell lymphoma, 21/03/2018 Withdrawn
Orphan designation: tazemetostat Treatment of malignant mesothelioma, 21/03/2018 Withdrawn
Orphan designation: avenciguat Treatment of systemic sclerosis, 11/11/2024 Withdrawn
Orphan designation: ataluren Treatment of Becker muscular dystrophy, 04/07/2012 Withdrawn
Orphan designation: ataluren Treatment of aniridia, 09/10/2015 Withdrawn
Orphan designation: tipifarnib Treatment of peripheral T-cell lymphoma, 19/10/2020 Withdrawn
Orphan designation: tipifarnib Treatment of acute myeloid leukaemia, 10/03/2005 Withdrawn
Orphan designation: bemarituzumab Treatment of gastric cancer, 21/08/2024 Withdrawn