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1701 results
Medicine
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Orphan designation: hydrocortisone for: Treatment of adrenal insufficiency (updated)
Date of first decision: 20/03/2007, Positive, Last updated: 20/10/2020 -
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Orphan designation: Magrolimab for: Treatment of myelodysplastic syndromes (updated)
Date of first decision: 26/06/2020, Positive, Last updated: 16/10/2020 -
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Orphan designation: Enzastaurin hydrochloride for: Treatment of diffuse large B-cell lymphoma (updated)
Date of first decision: 20/03/2007, Positive, Last updated: 16/10/2020 -
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Orphan designation: Enzastaurin hydrochloride for: Treatment of glioma (updated)
Date of first decision: 23/12/2005, Positive, Last updated: 16/10/2020 -
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Orphan designation: (2R,3S)-2-(4-cyclopentylaminophenyl)-1-(2-fluoro-6-methylbenzoyl)piperidine-3-carboxylic acid(4-methyl-3-trifluoromethylphenyl)amNide for: Treatment of microscopic polyangiitis (avacopan) (updated)
Date of first decision: 19/11/2014, Positive, Last updated: 15/10/2020 -
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Orphan designation: (2R,3S)-2-(4-cyclopentylaminophenyl)-1-(2-fluoro-6-methylbenzoyl)piperidine-3-carboxylic acid(4-methyl-3-trifluoromethylphenyl)amide (avacopan) for: Treatment of granulomatosis with polyangiitis (updated)
Date of first decision: 19/11/2014, Positive, Last updated: 15/10/2020 -
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Orphan designation: 1-[(3S)-3-{4-amino-3-[(3,5-dimethoxyphenyl)ethynyl]-1H-pyrazolo[3,4-d]pyrimidin-1-yl}pyrrolidin-1-yl]-2-propen-1-one (futibatinib) for: Treatment of biliary tract cancer (updated)
Date of first decision: 01/04/2019, Positive, Last updated: 15/10/2020 -
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Orphan designation: 2-hydroxymethyl-2-methoxymethyl-1-azabicyclo[2,2,2]octan-3-one for: Treatment of ovarian cancer (updated)
Date of first decision: 16/12/2014, Positive, Last updated: 13/10/2020 -
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Orphan designation: 2-(hydroxymethyl)-2-(methoxymethyl)-1-azabicyclo[2.2.2]octan-3-one for: Treatment of myelodysplastic syndromes (updated)
Date of first decision: 25/07/2019, Positive, Last updated: 13/10/2020 -
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Orphan designation: 2-Methoxymethyl-2-hydroxymethyl-1-azabicyclo[2,2,2]octan-3-one for: Treatment of acute myeloid leukaemia (updated)
Date of first decision: 10/06/2010, Positive, Last updated: 13/10/2020 -
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Orphan designation: 2ʹ-O-(2-methoxyethyl) antisense oligonucleotide targeting microtubule-associated protein tau pre-mRNA for: treatment of behavioural variant frontotemporal dementia (updated)
Date of first decision: 31/07/2018, Positive, Last updated: 13/10/2020 -
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Orphan designation: Encapsulated human retinal pigment epithelial cell line transfected with plasmid vector expressing human ciliary neurotrophic factor for: Treatment of retinitis pigmentosa (updated)
Date of first decision: 24/01/2013, Positive, Last updated: 09/10/2020 -
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Orphan designation: humanised fusion protein consisting of extracellular domain of CD24 linked to IgG1 Fc domain for: Prevention of graft-versus-host disease (updated)
Date of first decision: 11/11/2015, Positive, Last updated: 09/10/2020 -
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Orphan designation: DNA plasmid encoding a recombinant fusion protein consisting of the extracellular domain of human TNFα p55 receptor linked to the human IgG1 Fc domain for: Treatment of non-infectious uveitis (updated)
Date of first decision: 17/02/2016, Positive, Last updated: 29/09/2020 -
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Orphan designation: Adeno-associated viral vector serotype rh10 containing the human factor IX gene for the treatment of haemophilia B- for: Treatment of haemophilia B (updated)
Date of first decision: 14/12/2015, Positive, Last updated: 29/09/2020 -
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Orphan designation: Synthetic glucagon analogue modified to contain 7 amino acid substitutions for: Treatment of congenital hyperinsulinism (updated)
Date of first decision: 20/06/2017, Positive, Last updated: 25/09/2020 -
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Orphan designation: adeno-associated viral vector serotype 8 containing the human glucose-6-phosphatase gene (pariglasgene brecaparvovec) for: treatment of glycogen storage disease type Ia (updated)
Date of first decision: 18/11/2016, Positive, Last updated: 25/09/2020 -
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Orphan designation: Adeno-associated viral vector serotype 8 encoding human ornithine transcarbamylase (avalotcagene ontaparvovec) for: Treatment of ornithine transcarbamylase deficiency (updated)
Date of first decision: 21/03/2016, Positive, Last updated: 25/09/2020 -
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Orphan designation: Ketoconazole for: Treatment of Cushing's syndrome (updated)
Date of first decision: 23/04/2012, Positive, Last updated: 22/09/2020 -
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Orphan designation: Adeno-associated viral vector serotype 8 containing the human MTM1 gene (resamirigene bilparvovec) for: Treatment of X-linked myotubular myopathy
Date of first decision: 10/08/2015, Positive, Last updated: 18/09/2020 -
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Orphan designation: Obiltoxaximab for: Treatment of anthrax
Date of first decision: 24/08/2018, Positive, Last updated: 18/09/2020 -
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Orphan designation: Polyphenyl(disodium 3-O-sulfo-beta-D-glucopyranuronate)-(1->3)-beta-D-galactopyranoside for: Treatment of anti-MAG neuropathy
Date of first decision: 17/07/2017, Positive, Last updated: 18/09/2020 -
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Orphan designation: 2-[4-Methoxy-3-(2-m-tolyl-ethoxy)-benzoylamino]-indan-2-carboxylic acid for: Treatment of systemic sclerosis
Date of first decision: 12/03/2013, Positive, Last updated: 18/09/2020 -
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Orphan designation: Flucytosine for: Treatment of glioma
Date of first decision: 22/02/2018, Positive, Last updated: 18/09/2020 -
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Orphan designation: Vocimagene amiretrorepvec for: Treatment of glioma
Date of first decision: 22/02/2018, Positive, Last updated: 18/09/2020