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124 results
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Orphan designation: autologous peripheral blood T cells CD4 and CD8 selected and CD3 and CD28 activated transduced with retroviral vector expressing anti CD19 CD28/CD3-zeta chimeric antigen receptor and cultured (brexucabtagene autoleucel) for: Treatment of mantle cell lymphoma
Date of designation: 13/11/2019, Positive, Last updated: 15/09/2021 -
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Orphan designation: gemtuzumab ozogamicin for: Treatment of acute myeloid leukaemia
Date of designation: 18/10/2000, Positive, Last updated: 19/06/2013 -
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Orphan designation: Gilteritinib for: Treatment of acute myeloid leukaemia
Date of designation: 17/01/2018, Positive, Last updated: 08/11/2019 -
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Orphan designation: (3S)-3-{4-[7-(aminocarbonyl)-2H-indazol-2-yl] phenyl} piperidine tosilate monohydrate saltĀ (niraparib) for: Treatment of ovarian cancer
Date of designation: 04/08/2010, Positive, Last updated: 06/11/2020 -
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Orphan designation: carfilzomib for: Treatment of multiple myeloma
Date of designation: 03/06/2008, Positive, Last updated: 02/12/2015 -
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Orphan designation: Chimeric monoclonal antibody against GD2 for: Treatment of neuroblastoma
Date of designation: 08/11/2012, Positive, Last updated: 26/08/2021 -
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Orphan designation: polatuzumab vedotin for: Treatment of diffuse large B-cell lymphoma
Date of designation: 16/04/2018, Positive, Last updated: 27/01/2020 -
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Orphan designation: Glasdegib maleate for: Treatment of acute myeloid leukaemia
Date of designation: 16/10/2017, Positive, Last updated: 09/01/2018 -
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Orphan designation: Autologous T cells transduced with retroviral vector encoding an anti-CD19 CD28/CD3-zeta chimeric antigen receptor (axicabtagene ciloleucel) for: Treatment of primary mediastinal large B-cell lymphoma
Date of designation: 09/10/2015, Positive, Last updated: 15/09/2021 -
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Orphan designation: Autologous T cells transduced with retroviral vector encoding an anti-CD19 CD28/CD3 zeta chimeric antigen receptor (axicabtagene ciloleucel) for: Treatment of diffuse large B-cell lymphoma
Date of designation: 16/12/2014, Positive, Last updated: 15/09/2021 -
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Orphan designation: Eculizumab for: Treatment of atypical haemolytic uraemic syndrome (aHUS)
Date of designation: 24/07/2009, Positive, Last updated: 20/11/2019 -
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Orphan designation: Eculizumab for: Treatment of neuromyelitis optica spectrum disorders
Date of designation: 05/08/2013, Positive, Last updated: 20/11/2019 -
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Orphan designation: Eculizumab for: Treatment myasthenia gravis
Date of designation: 29/07/2014, Positive, Last updated: 20/11/2019 -
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Orphan designation: Fenfluramine hydrochloride for: Treatment of Dravet syndrome
Date of designation: 18/12/2013, Positive, Last updated: 08/01/2021 -
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Orphan designation: defibrotide for: Treatment of hepatic veno-occlusive disease
Date of designation: 29/07/2004, Positive, Last updated: 17/08/2016 -
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Orphan designation: Human coagulation factor X for: Treatment of hereditary factor X deficiency
Date of designation: 14/09/2007, Positive, Last updated: 26/03/2019 -
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Orphan designation: (2S)-2-{[(2R)-2-[({[3,3-dibutyl-7-(methylthio)-1,1-dioxido-5-phenyl-2,3,4,5-tetrahydro-1,2,5-benzothiadiazepin-8-yl]oxy}acetyl)amino]-2-(4-hydroxyphenyl)acetyl]amino}butanoic acid for: Treatment of progressive familial intrahepatic cholestasis
Date of designation: 17/07/2012, Positive, Last updated: 28/07/2021 -
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Orphan designation: Recombinant human tripeptidyl-peptidase 1 for: Treatment of neuronal ceroid lipofuscinosis type 2
Date of designation: 12/03/2013, Positive, Last updated: 09/02/2016 -
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Orphan designation: Humanised monoclonal antibody against P-selectin (crizanlizumab) for: Treatment of sickle cell disease
Date of designation: 09/08/2012, Positive, Last updated: 24/11/2020 -
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Orphan designation: Synthetic double-stranded siRNA oligonucleotide directed against delta-aminolevulinic acid synthase 1 mRNA, covalently linked to a ligand containing three N-acetylgalactosamine residues (givosiran) for: Treatment of acute hepatic porphyria
Date of designation: 29/08/2016, Positive, Last updated: 09/03/2020 -
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Orphan designation: Osilodrostat for: Treatment of Cushing's syndrome
Date of designation: 15/10/2014, Positive, Last updated: 07/07/2020 -
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Orphan designation: Ketoconazole for: Treatment of Cushing's syndrome
Date of designation: 23/04/2012, Positive, Last updated: 22/09/2020 -
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Orphan designation: Adenovirus-associated viral vector serotype 2 containing the human RPE65 gene (voretigene neparvovec) for: Treatment of retinitis pigmentosa
Date of designation: 28/07/2015, Positive, Last updated: 04/03/2019 -
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Orphan designation: Adenovirus-associated viral vector serotype 2 containing the human RPE65 gene (voretigene neparvovec) for: Treatment of Leber's congenital amaurosis
Date of designation: 02/04/2012, Positive, Last updated: 04/03/2019 -
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Orphan designation: cholic acid for: Treatment of inborn errors in primary bile acid synthesis
Date of designation: 18/12/2002, Positive, Last updated: 12/04/2016