Orphan designation: cedazuridine,decitabine Treatment of myelodysplastic syndromes, 16/08/2023 Withdrawn
Orphan designation: ovine anti-colchicine polyclonal antibody fragments Treatment of colchicine poisoning, 17/12/2010 Withdrawn
Orphan designation: genetically modified serotype 5/3 adenovirus coding for granulocyte-macrophage colony-stimulating factor Treatment of soft tissue sarcoma, 19/06/2013 Withdrawn
Orphan designation: genetically modified serotype 5/3 adenovirus coding for granulocyte macrophage colony-stimulating factor Treatment of malignant mesothelioma, 16/12/2014 Withdrawn
Orphan designation: Treatment of Alagille syndrome, 09/08/2012 Withdrawn
Orphan designation: macitentan Treatment of pulmonary arterial hypertension, 27/09/2011 Withdrawn
Orphan designation: Human platelet antigen-1a immunoglobulin Prevention of fetal and neonatal alloimmune thrombocytopenia, 27/10/2011 Withdrawn
Orphan designation: sutimlimab Treatment of immune thrombocytopenia, 28/02/2020 Withdrawn
Orphan designation: Copanlisib Treatment of marginal zone lymphoma, 24/08/2018 Withdrawn
Orphan designation: adeno-associated virus vector serotype hu37 encoding human factor VIII Treatment of haemophilia A, 22/04/2020 Withdrawn