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668 results
Orphan designation status
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Orphan designation: Chimeric locked nucleic acid deoxynucleoside phosphorothioate-linked oligonucleotide inhibitor directed against microRNA-155-5p for: Treatment of cutaneous T-cell lymphoma (updated)
Date of designation: 22/05/2017, Withdrawn, Last updated: 22/06/2022 -
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Orphan designation: Methyl 4,6-diamino-2-[1-(2-fluorobenzyl)-1H-pyrazolo[3,4-b]pyridine-3-yl]-5-pyrimidinyl(methyl)carbamate (riociguat) for: Treatment of pulmonary arterial hypertension (updated)
Date of designation: 20/12/2007, Withdrawn, Last updated: 14/06/2022 -
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Orphan designation: (4R,5R)-1-[[4-[[4-[3,3-dibutyl-7-(dimethylamino)-2,3,4,5- tetrahydro-4-hydroxy-1,1-dioxido-1-benzothiepin-5-yl]phenoxy]methyl]phenyl]methyl]-4-aza-1-azoniabicyclo[2.2.2]octane chloride (maralixibat chloride) for: Treatment of primary sclerosing cholangitis (updated)
Date of designation: 16/01/2014, Withdrawn, Last updated: 14/06/2022 -
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Orphan designation: (4R,5R)-1-[[4-[[4-[3,3-dibutyl-7-(dimethylamino)-2,3,4,5- tetrahydro-4-hydroxy-1,1-dioxido-1-benzothiepin-5-yl]phenoxy]methyl]phenyl]methyl]-4-aza-1-azoniabicyclo[2.2.2]octane chloride (maralixibat chloride) for: Treatment of primary biliary cirrhosis (updated)
Date of designation: 16/01/2014, Withdrawn, Last updated: 14/06/2022 -
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Orphan designation: Trifarotene for: Treatment of autosomal recessive congenital ichthyosis (updated)
Date of designation: 24/03/2020, Withdrawn, Last updated: 07/06/2022 -
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Orphan designation: Inebilizumab for: Treatment of neuromyelitis optica spectrum disorders
Date of designation: 20/03/2017, Withdrawn, Last updated: 19/05/2022 -
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Orphan designation: synthetic human hepcidin for: Treatment of sickle cell disease
Date of designation: 18/11/2016, Withdrawn, Last updated: 18/05/2022 -
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Orphan designation: Synthetic hepcidin for: Treatment of beta thalassaemia intermedia and major
Date of designation: 09/10/2015, Withdrawn, Last updated: 18/05/2022 -
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Orphan designation: Obeticholic acid for: Treatment of primary sclerosing cholangitis
Date of designation: 16/03/2014, Withdrawn, Last updated: 10/05/2022 -
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Orphan designation: N-(2-amino-phenyl)-4-[(4-pyridin-3-yl-pyrimidin-2-ylamino)-methyl] benzamide (mocetinostat) for: Treatment of Hodgkin's lymphoma
Date of designation: 14/09/2007, Withdrawn, Last updated: 10/05/2022 -
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Orphan designation: N- (2-Amino-phenyl)-4-[(4-pyridin-3-yl-pyrimidin-2-ylamino)-methyl] benzamide (mocetinostat) for: Treatment of acute myeloid leukaemia
Date of designation: 31/01/2008, Withdrawn, Last updated: 04/05/2022 -
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Orphan designation: Lisocabtagene maraleucel for: Treatment of primary mediastinal large B-cell lymphoma
Date of designation: 19/11/2018, Withdrawn, Last updated: 04/05/2022 -
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Orphan designation: Autologous CD4+ and CD8+ T cells expressing a CD19-specific chimeric antigen receptor (lisocabtagene maraleucel) for: Treatment of follicular lymphoma
Date of designation: 25/05/2018, Withdrawn, Last updated: 04/05/2022 -
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Orphan designation: Autologous CD4+ and CD8+ T cells expressing a CD19-specific chimeric antigen receptor (lisocabtagene maraleucel) for: Treatment of diffuse large B-cell lymphoma
Date of designation: 17/07/2017, Withdrawn, Last updated: 04/05/2022 -
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Orphan designation: Allogeneic donor-derived ex-vivo expanded T lymphocytes transduced with a retroviral vector containing inducible caspase 9 and truncated CD19 for: Treatment in haematopoietic stem cell transplantation
Date of designation: 27/06/2016, Withdrawn, Last updated: 07/04/2022 -
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Orphan designation: Rimiducid for: Treatment of graft-versus-host disease
Date of designation: 30/05/2016, Withdrawn, Last updated: 07/04/2022 -
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Orphan designation: Multilamellar microvesicle comprising phosphatidylcholine, sphingomyelin, phosphatidylethanolamine, phosphatidylserine, phosphatidylinositol and cholesterol for: Treatment of cystic fibrosis
Date of designation: 30/08/2011, Withdrawn, Last updated: 06/04/2022 -
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Orphan designation: Autologous haematopoietic stem cells transduced with lentiviral vector Lenti-D encoding the human ABCD1 cDNA (elivaldogene autotemcel) for: Treatment of adrenoleukodystrophy
Date of designation: 06/06/2012, Withdrawn, Last updated: 04/04/2022 -
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Orphan designation: Adeno-associated virus serotype 9 expressing the cDNA for human MECP2 for: Treatment of Rett syndrome
Date of designation: 20/05/2021, Withdrawn, Last updated: 30/03/2022 -
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Orphan designation: Veledimex for: Treatment of glioma
Date of designation: 21/08/2019, Withdrawn, Last updated: 21/03/2022 -
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Orphan designation: Adenoviral vector serotype 5 encoding the human interleukin-12 p70 transgene under the control of activator ligand veledimex for: Treatment of glioma
Date of designation: 21/08/2019, Withdrawn, Last updated: 21/03/2022 -
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Orphan designation: Ponatinib hydrochloride for: Treatment of gastrointestinal stromal tumours
Date of designation: 15/01/2015, Withdrawn, Last updated: 18/03/2022 -
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Orphan designation: 3-pentylbenzeneacetic acid sodium salt for: Treatment of idiopathic pulmonary fibrosis
Date of designation: 09/10/2015, Withdrawn, Last updated: 28/02/2022 -
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Orphan designation: 3-pentylbenzeneacetic acid sodium salt for: Treatment of Alström syndrome
Date of designation: 12/01/2017, Withdrawn, Last updated: 28/02/2022 -
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Orphan designation: N-methyl-4-({4-[({3-methyl(methylsulfonyl)aminopyrazin-2-yl}methyl)amino]-5-(trifluoromethyl)pyrimidin-2-yl}amino)benzamide hydrochloride (defactinib) for: Treatment of malignant mesothelioma
Date of designation: 07/06/2013, Withdrawn, Last updated: 28/02/2022