Everything you care about in one place
Follow feeds: blogs, news, RSS and more. An effortless way to read and digest content of your choice.
Get Feederema.europa.eu
Orphan designations
Get the latest updates from Orphan designations directly as they happen.
Follow now 192 followers
Latest posts
Last updated 5 days ago
Orphan designation: recombinant human N-acetylgalactosamine-6-sulfatase (elosulfase alfa) Treatment of mucopolysaccharidosis, type IVA (Morquio A syndrome), 24/07/2009 Withdrawn
5 days 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
11 days 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
about 1 month 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
about 1 month ago
Orphan designation: Panobinostat Treatment of multiple myeloma, 08/11/2012 Expired
Orphan designation: relacorilant Treatment of ovarian cancer, 25/07/2023 Positive
about 2 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
about 2 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monoclonal antibody against TREM2 Treatment of CSF1R-related leukoencephalopathy, 13/10/2023 Withdrawn
about 2 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adeno-associated viral vector containing a modified U7 snRNA gene Treatment of Duchenne muscular dystrophy, 27/07/2005 Withdrawn
about 2 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
about 2 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
about 2 months 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
about 2 months 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
about 2 months ago
Orphan designation: valganciclovir,nanatinostat Treatment of diffuse large B-cell lymphoma, 13/01/2023 Withdrawn