Orphan designation: 68Ga-2,2'-(7-(4-((S)-1-((4S,7S,10S,13R,16S,19R)-4-((R)-1-amino-3-(4-hydroxyphenyl)-1-oxopropan-2-ylcarbamoyl)-10-(4-aminobutyl)-16-(4-((S)-2,6-dioxohexahydropyrimidine-4-carboxamido)benzyl)-7-((R)-1-hydroxyethyl)-6,9,12,15,18-pentaoxo-13-(4-ureidobenzyl)-1,2-dithia-5,8,11,14,17-pentaazacycloicosan-19-ylamino)-3-(4-chlorophenyl)-1-oxopropan-2-ylamino)-1-carboxy-4-oxobutyl)-1,4,7-triazonane-1,4-diyl)diacetic acid (satoreotide trixozetan) ,satoreotide trizoxetan gallium (Ga68) Diagnosis of gastro-entero-pancreatic neuroendocrine tumours, 19/02/2014 Positive

Orphan designation: synthetic double-stranded siRNA oligonucleotide targeted against transthyretin mRNA, with six phosphorothioate linkages in the backbone, and nine 2'-fluoro and thirty-five 2'-O-methyl nucleoside residues in the sequence, which is covalently linked via a phosphodiester group to a ligand containing three N-acetylgalactosamine residues (Vutrisiran) Treatment of transthyretin-mediated amyloidosis (ATTR amyloidosis), 25/05/2018 Positive

Orphan designation: lepunafusp alfa Treatment of mucopolysaccharidosis type I, 26/03/2021 Positive

Orphan designation: (2S,4R)-1-(2-(3-acetyl-5-(2-methylpyrimidine-5-yl)-1H-indazol-1-yl)acetyl)-N-(6-bromopyridine-2-yl)-4-fluoropyrrolidine-2-carboxamide Treatment of C3 glomerulopathy, 21/03/2018 Withdrawn

Orphan designation: utreloxastat Treatment of amyotrophic lateral sclerosis, 09/12/2022 Withdrawn

Orphan designation: Obecabtagene autoleucel Treatment of acute lymphoblastic leukaemia, 13/04/2022 Withdrawn

Orphan designation: diflunisal Treatment of ATTR amyloidosis, 21/06/2022 Withdrawn

Orphan designation: iloprost Treatment of systemic sclerosis, 20/06/2025 Positive

Orphan designation: Humanised monoclonal antibody targeting B-cell maturation antigen conjugated with maleimidocaproyl monomethyl auristatin F Treatment of multiple myeloma, 16/10/2017 Withdrawn

Orphan designation: (R)-3-(5-dimethylcarbamoyl-pent-1-enyl)-N-(2-hydroxy-1methylethyl)benzamide Treatment of fragile X syndrome Positive