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2394 results
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Orphan designation: genetically modified serotype 5/3 adenovirus coding for granulocyte-macrophage colony-stimulating factor for: Treatment of soft tissue sarcoma
Date of first decision: 19/06/2013, Positive, Last updated: 19/03/2021 -
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Orphan designation: Genetically modified serotype 5/3 adenovirus coding for granulocyte macrophage colony-stimulating factor for the treatment of ovarian cancer for: Treatment of ovarian cancer
Date of first decision: 29/04/2014, Positive, Last updated: 19/03/2021 -
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Orphan designation: Levosimendan for: Treatment of amyotrophic lateral sclerosis
Date of first decision: 22/02/2018, Withdrawn, Last updated: 19/03/2021 -
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Orphan designation: Humanised single-chain monoclonal antibody against CD37 (otlertuzumab) for: Treatment chronic lymphocytic leukaemia
Date of first decision: 06/12/2012, Withdrawn, Last updated: 19/03/2021 -
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Orphan designation: Adeno-associated viral vector serotype 8 containing the human UGT1A1 gene (volrubigene ralaparvovec) for: Treatment of Crigler-Najjar syndrome
Date of first decision: 22/08/2014, Withdrawn, Last updated: 19/03/2021 -
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Orphan designation: Nimorazole maleate for: Treatment of squamous-cell carcinoma of the head and neck in patients undergoing radiotherapy
Date of first decision: 09/02/2012, Withdrawn, Last updated: 19/03/2021 -
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Orphan designation: Three chimeric human/murine monoclonal antibodies against the Ebola (Zaire) surface glycoprotein (porgaviximab) for: Treatment for Ebola virus disease
Date of first decision: 09/10/2015, Withdrawn, Last updated: 19/03/2021 -
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Orphan designation: dry extract from birch bark (DER 5-10 : 1), extraction solvent n-heptane 95% (w/w) for: Treatment of epidermolysis bullosa
Date of first decision: 23/02/2011, Positive, Last updated: 09/03/2021 -
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Orphan designation: Murine anti-CD22 antibody variable region fused to truncated Pseudomonas exotoxin 38 ( Moxetumomab pasudotox) for: Treatment of hairy cell leukaemia
Date of first decision: 05/12/2008, Positive, Last updated: 09/03/2021 -
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Orphan designation: N-tert-butyl-3-[(5-methyl-2-{[4-(2-pyrrolidin-1-ylethoxy)phenyl]amino}pyrimidin-4-yl)amino] benzenesulfonamide dihydrochloride monohydrate (fedratinib) for: Treatment of post-polycythaemia vera myelofibrosis
Date of first decision: 26/11/2010, Positive, Last updated: 03/03/2021 -
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Orphan designation: N-tert-butyl-3-[(5-methyl-2-{[4-(2-pyrrolidin-1-ylethoxy)phenyl]amino}pyrimidin-4-yl)amino] benzenesulfonamide dihydrochloride monohydrate (fedratinib) for: Treatment of post-essential thrombocythaemia myelofibrosis
Date of first decision: 26/11/2010, Positive, Last updated: 03/03/2021 -
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Orphan designation: N-tert-butyl-3-[(5-methyl-2-{[4-(2-pyrrolidin-1-ylethoxy)phenyl]amino}pyrimidin-4-yl)amino] benzenesulfonamide dihydrochloride monohydrate (fedratinib) for: Treatment of primary myelofibrosis
Date of first decision: 01/10/2010, Positive, Last updated: 03/03/2021 -
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Orphan designation: (R)-1-[1-(4-acetoxy-3,3-dimethyl-2-oxo-butyl)-2-oxo-5-(pyridin-2-yl)-2,3-dihydro-1H-benzo[e][1,4]diazepin-3-yl]-3-(3-methylamino-phenyl)-urea (ceclazepide) for: Treatment of gastro-entero-pancreatic neuroendocrine tumours
Date of first decision: 14/12/2015, Positive, Last updated: 02/03/2021 -
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Orphan designation: R-1-[2,3-dihydro-2-oxo-1-pivaloylmethyl-5-(2-pyridyl)-1 H-1,4-benzodiazepin-3-yl]-3-(3-methylaminophenyl)urea (netazepide) for: Treatment of gastric carcinoid
Date of first decision: 14/06/2007, Positive, Last updated: 02/03/2021 -
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Orphan designation: N-(4-(1-cyanocyclopentyl)phenyl)-2-(4-pyridinylmethyl)amino-3-pyridinecarboxamide methanesulfonate for: Treatment of gastric cancer
Date of first decision: 27/02/2017, Positive, Last updated: 02/03/2021 -
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Orphan designation: N-acetylgalactosamine-conjugated synthetic double-stranded oligomer specific to serpin family A member 1 gene for: Treatment of congenital alpha-1 antitrypsin deficiency
Date of first decision: 31/07/2018, Positive, Last updated: 02/03/2021 -
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Orphan designation: Tadekinig alfa for: Treatment of haemophagocytic lymphohistiocytosis
Date of first decision: 14/10/2016, Positive, Last updated: 19/02/2021 -
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Orphan designation: Luspatercept for: Treatment of myelofibrosis
Date of first decision: 28/02/2020, Positive, Last updated: 18/02/2021 -
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Orphan designation: Recombinant fusion protein consisting of a modified form of the extracellular domain of human activin receptor IIB linked to the human IgG1 Fc domain (luspatercept) for: Treatment of beta thalassaemia intermedia and major
Date of first decision: 29/07/2014, Positive, Last updated: 18/02/2021 -
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Orphan designation: Recombinant fusion protein consisting of a modified form of the extracellular domain of human activin receptor IIB linked to the human IgG1 Fc domain (luspatercept) for: Treatment of myelodysplastic syndromes
Date of first decision: 22/08/2014, Positive, Last updated: 18/02/2021 -
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Orphan designation: 5-(Ethylsulfonyl)-2-(naphthalen-2-yl)benzo[d]oxazole (ezutromid) for: Treatment of Duchenne muscular dystrophy
Date of first decision: 04/12/2008, Withdrawn, Last updated: 10/02/2021 -
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Orphan designation: human anti-promyostatin monoclonal antibody (apitegromab) for: Treatment of spinal muscular atrophy
Date of first decision: 14/12/2018, Positive, Last updated: 09/02/2021 -
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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 first decision: 17/07/2017, Positive, Last updated: 09/02/2021 -
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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 first decision: 25/05/2018, Positive, Last updated: 09/02/2021 -
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Orphan designation: Lisocabtagene maraleucel for: Treatment of primary mediastinal large B-cell lymphoma
Date of first decision: 19/11/2018, Positive, Last updated: 09/02/2021