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adeno-associated virus vector serotype hu37 encoding human factor VIII Treatment of haemophilia A, 22/04/2020 Withdrawn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factor (lacnotuzumab) Treatment of tenosynovial giant cell tumour, localised and diffuse type, 15/10/2014 Withdrawn
Orphan designation: Dantrolene sodium Treatment of Wolfram syndrome, 12/12/2016 Withdrawn
Orphan designation: tislelizumab Treatment of nasopharyngeal cancer, 21/06/2021 Withdrawn
Orphan designation: vincristine sulfate liposomes Treatment of acute lymphoblastic leukaemia, 08/07/2008 Withdrawn
Orphan designation: antisense NF-kBp65 oligonucleotide Treatment of active ulcerative colitis, 30/07/2002 Withdrawn
Orphan designation: acetylsalicylic acid Treatment of polycythaemia vera, 29/07/2004 Withdrawn
Orphan designation: recombinant human factor IX protein modified with three point mutations (dalcinonacog alfa) Treatment of haemophilia B, 20/06/2017 Withdrawn
Orphan designation: Marzeptacog alfa (activated) Treatment of haemophilia B, 01/04/2019 Withdrawn
Orphan designation: Treatment of paroxysmal nocturnal haemoglobinuria, 19/02/2021 Withdrawn