Orphan designation: Synthetic oligonucleotide selectively targeting UBE3A antisense RNA transcripts Treatment of Angelman syndrome, 09/12/2020 Positive

Orphan designation: human IgG1 monoclonal antibody against sortilin Treatment of frontotemporal dementia, 15/10/2021 Positive

Orphan designation: Entospletinib Treatment of acute myeloid leukaemia, 16/10/2017 Withdrawn

Orphan designation: 1-(4-(6-chloropyridazin-3-yl)piperazin-1-yl)-2-(4-cyclopropyl-3-fluorophenyl)ethan-1-one Treatment of propionic acidaemia, 19/07/2021 Withdrawn

Orphan designation: 1-(4-(6-chloropyridazin-3-yl)piperazin-1-yl)-2-(4-cyclopropyl-3-fluorophenyl)ethan-1-one Treatment of pantothenate kinase-associated neurodegeneration, 19/07/2021 Withdrawn

Orphan designation: nerandomilast Treatment of idiopathic pulmonary fibrosis, 28/06/2024 Withdrawn

Orphan designation: S-[5-(omega-methoxypoly(oxyethylene)-2-oxopentyl)]-L-cysteinylglycyl-L-serinylglycylgylcyl-L-isoleucyl-L-lysyl-L-glutamyl-L-phenylalanyl-L-leucyl-L-glutaminyl-L-arginyl-L-phenylalanyl-L-isoleucyl-L-histyl-L-isoleucyl-L-valyl-L-glutaminyl-L-serinyl-L-isoleucyl-L-isoleucyl-L-asparaginyl-L-threonyl-L-serinamide, acetate salt Treatment of cutaneous T-cell lymphoma, 13/04/2021 Withdrawn

Orphan designation: Adeno-associated viral vector serotype 3B encoding human multidrug resistance protein 3A Treatment of progressive familial intrahepatic cholestasis, 22/04/2020 Withdrawn

Orphan designation: Nipocalimab Prevention of fetal and neonatal alloimmune thrombocytopenia Positive

Orphan designation: Sirolimus Treatment of familial adenomatous polyposis (FAP), 16/04/2025 Positive