Orphan designation: adeno-associated viral vector containing a modified U7 snRNA gene Treatment of Duchenne muscular dystrophy, 27/07/2005 Withdrawn

Orphan designation: adeno-associated virus vector serotype 9 encoding human gigaxonin gene Treatment of giant axonal neuropathy, 13/04/2022 Withdrawn

Orphan designation: hydrocortisone Treatment of adrenal insufficiency, 20/03/2007 Withdrawn

Orphan designation: nanatinostat,valganciclovir Treatment of peripheral T-cell lymphoma, 10/08/2022 Withdrawn

Orphan designation: valganciclovir,nanatinostat Treatment of diffuse large B-cell lymphoma, 13/01/2023 Withdrawn

Orphan designation: 2-[6-(6,7-Dimethoxyquinolin-3-yl)pyridin-3-yl]-N-[3-(1,1,1-trifluoro-2-methylpropan-2-yl)-1,2-oxazol-5-yl]acetamide (zeteletinib) Treatment of medullary thyroid carcinoma, 19/02/2021 Withdrawn

Orphan designation: Interferon gamma Treatment of Friedreich's ataxia, 09/12/2011 Withdrawn

Orphan designation: humanised IgG1 kappa monoclonal antibody against tyrosine-protein phosphatase non-receptor type substrate 1, signal-regulatory protein beta-1 and signal-regulatory protein gamma Treatment of haemophagocytic lymphohistiocytosis, 18/07/2025 Positive

Orphan designation: allogeneic peripheral blood-derived T-cells, fratricide-resistant, transduced with a lentivirus vector expressing a chimeric antigen receptor against CD7 Treatment of acute lymphoblastic leukaemia, 18/07/2025 Positive

Orphan designation: aglatimagene besadenovec Treatment of pancreatic cancer, 18/07/2025 Positive