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Orphan designations
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Last updated 8 days ago
Orphan designation: idebenone Treatment of Leber's hereditary optic neuropathy, 16/02/2007 Expired
8 days ago
Orphan designation: idebenone Treatment of Leber's hereditary optic neuropathy, 16/02/2007 Expired
Orphan designation: Panobinostat Treatment of multiple myeloma, 08/11/2012 Expired
8 days ago
Orphan designation: Panobinostat Treatment of multiple myeloma, 08/11/2012 Expired
Orphan designation: relacorilant Treatment of ovarian cancer, 25/07/2023 Positive
20 days ago
Orphan designation: relacorilant Treatment of ovarian cancer, 25/07/2023 Positive
Orphan designation: botaretigene sparoparvovec Treatment of inherited retinal dystrophies due to defects in the RPGR gene, 23/07/2025 Positive
20 days ago
Orphan designation: botaretigene sparoparvovec Treatment of inherited retinal dystrophies due to defects...
Orphan designation: human IgG1 (296-cysteine,301-glycine,306-cysteine) monoclonal antibody against TREM2 Treatment of CSF1R-related leukoencephalopathy, 13/10/2023 Withdrawn
20 days ago
Orphan designation: human IgG1 (296-cysteine,301-glycine,306-cysteine) monoclonal antibody against TREM2 Treatment of CSF1R-related...
Orphan designation: adeno-associated viral vector containing a modified U7 snRNA gene Treatment of Duchenne muscular dystrophy, 27/07/2005 Withdrawn
20 days ago
Orphan designation: adeno-associated viral vector containing a modified U7 snRNA gene Treatment...
Orphan designation: adeno-associated virus vector serotype 9 encoding human gigaxonin gene Treatment of giant axonal neuropathy, 13/04/2022 Withdrawn
20 days ago
Orphan designation: adeno-associated virus vector serotype 9 encoding human gigaxonin gene Treatment...
Orphan designation: hydrocortisone Treatment of adrenal insufficiency, 20/03/2007 Withdrawn
20 days ago
Orphan designation: hydrocortisone Treatment of adrenal insufficiency, 20/03/2007 Withdrawn
Orphan designation: nanatinostat,valganciclovir Treatment of peripheral T-cell lymphoma, 10/08/2022 Withdrawn
20 days ago
Orphan designation: nanatinostat,valganciclovir Treatment of peripheral T-cell lymphoma, 10/08/2022 Withdrawn
Orphan designation: valganciclovir,nanatinostat Treatment of diffuse large B-cell lymphoma, 13/01/2023 Withdrawn
20 days ago
Orphan designation: valganciclovir,nanatinostat Treatment of diffuse large B-cell lymphoma, 13/01/2023 Withdrawn
Orphan designation: 2-[6-(6,7-Dimethoxyquinolin-3-yl)pyridin-3-yl]-N-[3-(1,1,1-trifluoro-2-methylpropan-2-yl)-1,2-oxazol-5-yl]acetamide (zeteletinib) Treatment of medullary thyroid carcinoma, 19/02/2021 Withdrawn
20 days ago
Orphan designation: 2-[6-(6,7-Dimethoxyquinolin-3-yl)pyridin-3-yl]-N-[3-(1,1,1-trifluoro-2-methylpropan-2-yl)-1,2-oxazol-5-yl]acetamide (zeteletinib) Treatment of medullary thyroid carcinoma, 19/02/2021 Withdrawn
Orphan designation: Interferon gamma Treatment of Friedreich's ataxia, 09/12/2011 Withdrawn
20 days ago
Orphan designation: Interferon gamma Treatment of Friedreich's ataxia, 09/12/2011 Withdrawn