Orphan designation: Losmapimod treatment of facioscapulohumeral muscular dystrophy, 24/03/2020 Withdrawn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designation: 3-(3-(3,5-Dimethyl-1H-pyrazol-4-yl)propoxy)-4-fluorobenzoic acid Treatment of ATTR amyloidosis, 19/11/2018 Withdrawn

Orphan designation: Treatment of Huntington's disease, 15/02/2023 Withdrawn

Orphan designation: isotretinoin Treatment of recessive X-linked ichthyosis, 15/02/2023 Withdrawn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Omigapil maleate Treatment of congenital muscular dystrophy with merosin (laminin alpha 2) deficiency, 08/05/2008 Withdrawn

Orphan designation: Omigapil maleate Treatment of congenital muscular dystrophy with collagen VI deficiency, 08/05/2008 Withdrawn

Orphan designation: propagermanium Treatment of focal segmental glomerulosclerosis, 19/11/2018 Withdrawn

Orphan designation: 2'-O-4'-C-(S)-Ethyl-P-thioadenylyl-(3'-O->5'-O)-2'-O-4'-C-(S)-ethyl-P-thioguanylyl-(3'-O->5'-O)-2'-O-methyl-P-thiocytidylyl-(3'-O->5'-O)-2'-fluoro-P-thioadenylyl-(3'-O->5'-O)-2'-fluoro-P-thiocytidylyl-(3'-O->5'-O)-2'-fluoro-P-thiouridylyl-(3'-O->5'-O)-2'-O-methyl-P-thiouridylyl-(3'-O->5'-O)-2'-O-4'-C-(S)-ethyl-P-thiouridylyl-(3'-O->5'-O)-2'-O-4'-C-(S)-ethyl-adenosine Treatment of autosomal dominant polycystic kidney disease, 13/12/2025 Positive

Orphan designation: monepantel Treatment of amyotrophic lateral sclerosis, 13/12/2024 Positive

Orphan designation: allogeneic cardiosphere-derived cells Treatment of Duchenne muscular dystrophy, 13/12/2025 Positive