Orphan designation: tobevibart Treatment of hepatitis delta virus infection, 13/12/2024 Positive

Orphan designation: mitapivat sulfate Treatment of sickle cell disease, 13/12/2024 Positive

Orphan designation: 4-{[(2R,3S,4R,5S)-4-(4-chloro-2-fluoro-phenyl)-3-(3-chloro-2-fluoro-phenyl)-4-cyano-5-(2,2-dimethyl-propyl)-pyrrolidine-2-carbonyl]-amino}-3-methoxy-benzoic acid  Treatment of acute myeloid leukaemia, 22/08/2014 Withdrawn

Orphan designation: Losmapimod treatment of facioscapulohumeral muscular dystrophy, 24/03/2020 Withdrawn

Orphan designation: 3-(3-(3,5-Dimethyl-1H-pyrazol-4-yl)propoxy)-4-fluorobenzoic acid Treatment of ATTR amyloidosis, 19/11/2018 Withdrawn

Orphan designation: Treatment of Huntington's disease, 15/02/2023 Withdrawn

Orphan designation: isotretinoin Treatment of recessive X-linked ichthyosis, 15/02/2023 Withdrawn

Orphan designation: Omigapil maleate Treatment of congenital muscular dystrophy with merosin (laminin alpha 2) deficiency, 08/05/2008 Withdrawn

Orphan designation: Omigapil maleate Treatment of congenital muscular dystrophy with collagen VI deficiency, 08/05/2008 Withdrawn

Orphan designation: propagermanium Treatment of focal segmental glomerulosclerosis, 19/11/2018 Withdrawn