Orphan designation: Marzeptacog alfa (activated) Treatment of haemophilia B, 01/04/2019 Withdrawn
Orphan designation: Treatment of paroxysmal nocturnal haemoglobinuria, 19/02/2021 Withdrawn
Orphan designation: humanised fusion protein consisting of extracellular domain of CD24 linked to IgG1 Fc domain Prevention of graft-versus-host disease, 11/11/2015 Withdrawn
Orphan designation: recombinant monoclonal antibody to sialic acid-binding Ig-like lectin 8 Treatment of mastocytosis, 16/10/2017 Withdrawn
Orphan designation: anti-SIGLEC8 IgG1 humanised monoclonal antibody Treatment of eosinophilic gastroenteritis, 19/02/2021 Withdrawn
Orphan designation: sabatolimab Treatment of myelodysplastic syndromes, 20/08/2021 Withdrawn
Orphan designation: Ilginatinib maleate Treatment of myelofibrosis, 25/07/2023 Withdrawn
Orphan designation: adeno-associated viral vector serotype 8 containing the human acid alpha-glucosidase gene Treatment of glycogen storage disease type II (Pompe's disease), 16/04/2018 Withdrawn
Orphan designation: navitoclax Treatment of myelofibrosis, 16/12/2019 Withdrawn
Orphan designation: acalabrutinib Treatment of mantle cell lymphoma, 21/03/2016 Withdrawn