Orphan designation: setanaxib Treatment of Alport syndrome, 08/11/2023 Positive

Orphan designation: 2-(2-chlorophenyl)-4-[3-(dimethylamino)phenyl]-5-methyl-1H-pyrazolo[4,3-C]pyridine-3,6(2H,5H)-dione Treatment of systemic sclerosis, 09/10/2015 Positive

Orphan designation: setanaxib Treatment of primary biliary cholangitis, 06/01/2021 Positive

Orphan designation: ziftomenib Treatment of acute myeloid leukaemia, 12/01/2024 Positive

Orphan designation: daraxonrasib Treatment of pancreatic cancer, 20/04/2026 Positive

Orphan designation: edaravone Treatment of amyotrophic lateral sclerosis, 19/06/2015 Positive

Orphan designation: Treatment of Leber’s hereditary optic neuropathy, 14/01/2022 Positive

Orphan designation: 2-(2-chlorophenyl)-4-[3-(dimethylamino)phenyl]-5-methyl-1H-pyrazolo[4,3-C]pyridine-3,6(2H,5H)-dione Treatment of idiopathic pulmonary fibrosis, 26/11/2010 Positive

Orphan designation: 7-ethyl-10-hydroxy-camptothecin,irinotecan hydrochloride trihydrate Treatment of small cell lung cancer, 24/05/2024 Positive

Orphan designation: human IgG1 monoclonal antibody against hepatitis B virus, surface antigen Treatment of hepatitis D virus infection, 28/06/2024 Positive