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Human platelet antigen-1a immunoglobulin Prevention of fetal and neonatal alloimmune thrombocytopenia, 27/10/2011 Withdrawn
Orphan designation: sutimlimab Treatment of immune thrombocytopenia, 28/02/2020 Withdrawn
Orphan designation: Copanlisib Treatment of marginal zone lymphoma, 24/08/2018 Withdrawn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adeno-associated virus vector serotype hu37 encoding human factor VIII Treatment of haemophilia A, 22/04/2020 Withdrawn
Orphan designation: recombinant human monoclonal antibody of the IgG1 kappa class against human macrophage colony-stimulating factor (lacnotuzumab) Treatment of tenosynovial giant cell tumour, localised and diffuse type, 15/10/2014 Withdrawn
Orphan designation: Dantrolene sodium Treatment of Wolfram syndrome, 12/12/2016 Withdrawn
Orphan designation: tislelizumab Treatment of nasopharyngeal cancer, 21/06/2021 Withdrawn
Orphan designation: vincristine sulfate liposomes Treatment of acute lymphoblastic leukaemia, 08/07/2008 Withdrawn
Orphan designation: antisense NF-kBp65 oligonucleotide Treatment of active ulcerative colitis, 30/07/2002 Withdrawn
Orphan designation: acetylsalicylic acid Treatment of polycythaemia vera, 29/07/2004 Withdrawn