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1723 results
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Orphan designation: Heterologous human adult liver-derived progenitor cells for: Treatment of argininosuccinic aciduria
Date of first decision: 17/07/2013, Positive, -
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Orphan designation: (S)-1-(4-fluorophenyl)-1-(2-(4-(6-(1-methyl-1H-pyrazol-4-yl)pyrrolo[2,1-f][1,2,4]triazin-4-yl)piperazin-yl)pyrimidin-5-yl)ethan-1-amine for: Treatment of gastrointestinal stromal tumours
Date of first decision: 17/07/2017, Positive, Last updated: 22/05/2019 -
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Orphan designation: Masitinib mesilate for: Treatment of pancreatic cancer
Date of first decision: 28/10/2009, Positive, -
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Orphan designation: Recombinant protein derived from the saliva of the Ornithodoros moubata tick (nomacopan) for: Treatment of Guillain-Barré syndrome
Date of first decision: 27/06/2016, Positive, Last updated: 26/07/2016 -
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Orphan designation: Glyceryl tri-(4-phenylbutyrate) for: Treatment of hyperargininaemia
Date of first decision: 10/06/2010, Positive, Last updated: 12/07/2019 -
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Orphan designation: 2',3',5'-Tri-O-acetyluridine for: Treatment of 5-fluorouracil overdose
Date of first decision: 15/05/2009, Positive, Last updated: 09/06/2009 -
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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: Eflornithine for: Treatment of familial adenomatous polyposis (FAP)
Date of first decision: 20/09/2010, Positive, Last updated: 29/04/2019 -
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Orphan designation: Propranolol for: Treatment of soft tissue sarcoma
Date of first decision: 12/12/2016, Positive, -
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Orphan designation: Mixture of two adeno-associated viral vectors serotype 8 containing the 5'-half sequence of human MYO7A gene and the 3'-half sequence of human MYO7A gene for: Treatment of Usher syndrome
Date of first decision: 04/07/2014, Positive, -
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Orphan designation: Polyethylene glycol-modified human recombinant truncated cystathionine beta-synthase for: Treatment of homocystinuria
Date of first decision: 30/05/2016, Positive, Last updated: 23/06/2016 -
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Orphan designation: Repertaxin L-lysine salt for: Prevention of delayed graft function in organ transplant
Date of first decision: 19/09/2001, Positive, -
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Orphan designation: Ursodeoxycholic acid for: Treatment of Niemann-Pick disease
Date of first decision: 22/05/2017, Positive, Last updated: 25/04/2019 -
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Orphan designation: Pyridoxal 5'-phosphate for: Treatment of pyridoxamine 5'-phosphate oxidase deficiency
Date of first decision: 15/10/2014, Positive, -
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Orphan designation: Recombinant human histone H1.3 and recombinant human N-bis-met-histone H1.3 for: Treatment of acute myeloid leukaemia
Date of first decision: 20/12/2007, Positive, Last updated: 13/03/2013 -
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Orphan designation: Methylthioninium for: treatment of frontotemporal dementia with parkinsonism-17
Date of first decision: 26/11/2010, Positive, Last updated: 28/03/2019 -
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Orphan designation: Poly-cyclodextrin-bis-cysteine-PEG3400-camptothecin-conjugate for: Treatment of ovarian cancer
Date of first decision: 20/03/2017, Positive, Last updated: 05/05/2017 -
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Orphan designation: Human MHC non-restricted cytotoxic T-cell line for: Treatment of ovarian cancer
Date of first decision: 30/11/2009, Positive, -
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Orphan designation: afamelanotide for: Treatment of solar urticaria
Date of first decision: 24/07/2009, Positive, Last updated: 11/04/2016 -
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Orphan designation: Adenovirus associated viral vector serotype 4 containing the human RPE65 gene for: Treatment of Leber's congenital amaurosis
Date of first decision: 22/10/2007, Positive, -
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Orphan designation: Gallium (68Ga)-edotreotide for: Diagnosis of gastro-entero-pancreatic neuroendocrine tumours
Date of first decision: 19/03/2015, Positive, Last updated: 20/02/2017 -
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Orphan designation: Nitric oxide for: Treatment of cystic fibrosis
Date of first decision: 15/10/2014, Positive, -
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Orphan designation: 68Ga-DOTA-pABzA-DIG-dPhe-Gln-Trp-Ala-Val-Gly-His-NHCH[(CH2-CH(CH3)2]2 for: Diagnosis of gastrointestinal stromal tumours
Date of first decision: 12/12/2016, Positive, -
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Orphan designation: N'-(5-chloro-2-hydroxy-3-methylbenzylidene)-2,4-dihydroxybenzhydrazide for: Treatment of partial deep dermal and full-thickness burns
Date of first decision: 22/09/2008, Positive, Last updated: 06/04/2011 -
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Orphan designation: Veliparib for: Treatment of ovarian cancer
Date of first decision: 17/12/2010, Positive, Last updated: 29/05/2018