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2394 results
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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: Amikacin sulfate for: Treatment of Pseudomonas aeruginosa lung infection in cystic fibrosis
Date of first decision: 16/12/2014, Positive, Last updated: 26/02/2015 -
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Orphan designation: Histamine dihydrochloride for: Treatment of malignant melanoma
Date of first decision: 24/08/2004, Negative, -
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Orphan designation: Deoxyribose phosphorothioate (5'-tct-ccc-agc-gtg-cgc-cat-3') for: Treatment of chronic lymphocytic leukaemia
Date of first decision: 21/11/2001, Withdrawn, -
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Orphan designation: Synthetic double-stranded oligomer specific to the SERPINA1 gene and containing a cholesterol-conjugated, acyclic nucleobase analogue for: Treatment of congenital alpha-1 antitrypsin deficiency
Date of first decision: 11/01/2016, Withdrawn, Last updated: 17/05/2017 -
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Orphan designation: Sodium (1R,3R,4R,5S)-3-({2-N-acetylamino-2-deoxy-3-O-[(1S)-1-carboxylato-2-cyclohexylethyl]-beta-D-galactopyranosyl}oxy)-4-({6-deoxy-alpha-L-galactopyranosyl}oxy)-5-ethyl-cyclohexan-1-yl-(38-oxo-2,5,8,11,14,17,20,23,26,29,32,35-dodecaoxa-39-azahentetracontan-41-yl)carboxamide for: Treatment of acute myeloid leukaemia
Date of first decision: 22/05/2017, Positive, Last updated: 03/04/2018 -
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Orphan designation: Suberolylanilide hydroxamic acid for: Treatment of cutaneous T-cell lymphoma
Date of first decision: 21/06/2004, Withdrawn, Last updated: 12/03/2013 -
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Orphan designation: Argon for: Treatment of perinatal asphyxia
Date of first decision: 27/06/2018, Positive, -
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Orphan designation: Autologous adipose tissue-derived stromal vascular fraction cells for: Treatment of systemic sclerosis
Date of first decision: 19/03/2015, Positive, Last updated: 11/05/2015 -
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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: 2-((2-ethyl-6-(4-(2-(3-hydroxyazetidin-1-yl)-2-oxoethyl)-piperazin-1-yl)-8-methylimidazo[1,2-alpha]pyridin-3-yl)-(methyl)amino)-4-(4-fluorophenyl)-thiazole-5-carbonitrile for: Treatment of idiopathic pulmonary fibrosis
Date of first decision: 29/08/2016, 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, -
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Orphan designation: Gallium [Ga-68]-N-[(4,7,10-Tricarboxymethyl-1,4,7,10-tetraazacyclododec-1-yl)acetyl]-D-phenylalanyl-L-cysteinyl-L-tyrosyl-D-tryptophanyl-L-lysyl-L-threoninyl-Lcysteinyl-L-threonine-cyclic(2-7)disulfide for: Diagnosis of gastro-entero-pancreatic neuroendocrine tumours
Date of first decision: 19/02/2014, Positive, -
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Orphan designation: Trebananib for: Treatment of ovarian cancer
Date of first decision: 13/11/2013, Positive, -
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Orphan designation: Decitabine for: Treatment of acute myeloid leukaemia
Date of first decision: 08/06/2006, Positive, Last updated: 19/09/2013 -
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Orphan designation: Human monoclonal IgG1 antibody against tissue factor pathway inhibitor for: Treatment of haemophilia A
Date of first decision: 14/10/2016, Positive, Last updated: 14/11/2016 -
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Orphan designation: Asunercept for: Treatment of myelodysplastic syndromes
Date of first decision: 23/08/2017, Positive, -
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Orphan designation: Nilotinib hydrochloride monohydrate for: Treatment of gastrointestinal stromal tumours
Date of first decision: 13/04/2007, Withdrawn, -
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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: Volanesorsen sodium for: Treatment of familial partial lipodystrophy
Date of first decision: 14/07/2016, Positive, Last updated: 23/05/2019 -
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Orphan designation: milciclib maleate for: Treatment of malignant thymoma
Date of first decision: 08/11/2012, Positive, -
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Orphan designation: 2-(1,5-dimethyl-3-phenyl-1H-pyrrol-2-yl)-N-{4-[4-(5-fluoro-pyrimidin-2-yl)piperazin-1-yl]-phenyl}-2-oxo-acetamide (also known as F901318) (olorofim) for: Treatment of scedosporiosis
Date of first decision: 29/08/2016, Positive, Last updated: 03/10/2016 -
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Orphan designation: Metformin for: Treatment of progressive myoclonic epilepsy type 2 (Lafora disease)
Date of first decision: 12/12/2016, Positive, -
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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,