Orphan designation: Allogeneic umbilical cord-derived mesenchymal stromal cells, pooled Treatment of perinatal asphyxia, 13/12/2023 Positive
Orphan designation: Humanised IgG1 kappa antibody against serum amyloid A and AL amyloid Treatment of amyloid light-chain amyloidosis, 08/02/2013 Positive
Orphan designation: Treatment of progressive familial intrahepatic cholestasis, 17/07/2012 Positive
Orphan designation: 2S, 4R ketoconazole Treatment of Cushing's syndrome, 04/07/2012 Positive
Orphan designation: Flucytosine Treatment of glioma, 22/02/2018 Positive
Orphan designation: Vocimagene amiretrorepvec Treatment of glioma, 22/02/2018 Positive
Orphan designation: Reldesemtiv Treatment of spinal muscular atrophy, 28/06/2019 Positive
Orphan designation: Reldesemtiv Treatment of amyotrophic lateral sclerosis, 28/02/2020 Positive
Orphan designation: Enzastaurin hydrochloride Treatment of Ehlers-Danlos syndrome, 24/02/2022 Positive
Orphan designation: Cyclo(-gamma-aminobutyryl-L-phenylalanyl-L-tryptophanyl-D-tryptophanyl-L-lysyl-L-threonyl-L phenylalanyl-N-3-carboxypropyl)-glycine amide, acetate salt Treatment of acromegaly, 06/12/2012 Positive