Orphan designation: Measles virus Schwarz strain (live, attenuated) Treatment of mesothelioma, 23/02/2026 Positive
Orphan designation: tobevibart Treatment of hepatitis delta virus infection, 13/12/2024 Positive
Orphan designation: elebsiran Treatment of hepatitis delta virus infection, 13/12/2024 Positive
Orphan designation: Infigratinib Treatment of achondroplasia, 19/07/2021 Positive
Orphan designation: Gallium (68Ga)-edotreotide Diagnosis of gastro-entero-pancreatic neuroendocrine tumours, 19/03/2015 Positive
Orphan designation: Lutetium (177Lu)-N-[(4,7,10-Tricarboxymethyl-1,4,7,10-tetraazacyclododec-1-yl)acetyl]-D-phenylalanyl-L-cysteinyl-L-tyrosyl-D-tryptophanyl-L-lysyl-L-threoninyl-L-cysteinyl-L-threonine-cyclic(2-7)disulfide (lutetium (177Lu) oxodotreotide) Treatment of gastro-entero-pancreatic neuroendocrine tumours, 31/01/2008 Positive
Orphan designation: mRNA encoding Cas12HF endonuclease, Single guide RNA against the human HAO1 gene Treatment of primary hyperoxaluria, 22/08/2025 Positive
Orphan designation: (R)-(3-(2'-cyclopropyl-3-(hydroxymethyl)-[1,1'-biphenyl]-4-yl)pyrrolidin-1-yl)(5-hydroxy-6-methylpyridin-2-yl)methanone Treatment of Olmsted syndrome, 26/02/2025 Positive
Orphan designation: autologous CD4+ and CD8+ T cells transduced with a lentiviral vector encoding an affinity enhanced T cell receptor specific to MAGE-A4 Treatment of soft tissue sarcoma, 04/06/2020 Positive
United for health at WorldPride 2026