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
Orphan designation: adeno-associated viral vector serotype 2 containing the human CHM gene encoding human Rab escort protein 1 Treatment of choroideraemia, 04/06/2014 Positive
Orphan designation: guanabenz acetate Treatment of vanishing white matter disease, 20/03/2023 Positive
Orphan designation: milademetan tosilate monohydrate Treatment of soft tissue sarcoma, 20/03/2017 Withdrawn
Orphan designation: adeno-associated virus vector serotype 1 containing the human GRN gene Treatment of frontotemporal dementia, 19/07/2021 Withdrawn
Orphan designation: N-Hydroxy-4-(3-methyl-2-(S)-phenyl-butyrylamino) benzamide Treatment of schwannoma, 06/06/2012 Withdrawn
Orphan designation: Letermovir Treatment of cytomegalovirus disease in patients with impaired cell-mediated immunity, 06/06/2012 Withdrawn
Orphan designation: N-Hydroxy-4-(3-methyl-2-(S)-phenyl-butyrylamino) benzamide Treatment of meningioma, 06/06/2012 Withdrawn