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Orphan designations
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Last updated 18 days ago
Orphan designation: carfilzomib Treatment of multiple myeloma, 03/06/2008 Expired
18 days ago
Orphan designation: carfilzomib Treatment of multiple myeloma, 03/06/2008 Expired
Orphan designation: Obinutuzumab Treatment of follicular lymphoma, 19/06/2015 Withdrawn
about 1 month ago
Orphan designation: Obinutuzumab Treatment of follicular lymphoma, 19/06/2015 Withdrawn
Orphan designation: recombinant human N-acetylgalactosamine-6-sulfatase (elosulfase alfa) Treatment of mucopolysaccharidosis, type IVA (Morquio A syndrome), 24/07/2009 Withdrawn
about 2 months ago
Orphan designation: recombinant human N-acetylgalactosamine-6-sulfatase (elosulfase alfa) Treatment of mucopolysaccharidosis, type IVA...
Orphan designation: 2-(3-(4-(1H-Indazol-5-ylamino)quinazolin-2-yl)phenoxy)-N-isopropylacetamide-methane sulfonic acid salt Treatment of graft-versus-host disease, 17/10/2019 Positive
about 2 months ago
Orphan designation: 2-(3-(4-(1H-Indazol-5-ylamino)quinazolin-2-yl)phenoxy)-N-isopropylacetamide-methane sulfonic acid salt Treatment of graft-versus-host disease, 17/10/2019 Positive
Orphan designation: idebenone Treatment of Leber's hereditary optic neuropathy, 16/02/2007 Expired
3 months 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
3 months ago
Orphan designation: Panobinostat Treatment of multiple myeloma, 08/11/2012 Expired
Orphan designation: relacorilant Treatment of ovarian cancer, 25/07/2023 Positive
3 months 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
3 months 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monoclonal antibody against TREM2 Treatment of CSF1R-related leukoencephalopathy, 13/10/2023 Withdrawn
3 months 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
3 months 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
3 months 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
3 months ago
Orphan designation: hydrocortisone Treatment of adrenal insufficiency, 20/03/2007 Withdrawn