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Orphan designation: 5-(Ethylsulfonyl)-2-(naphthalen-2-yl)benzo[d]oxazole (ezutromid) for: Treatment of Duchenne muscular dystrophy (updated)
Date of first decision: 04/12/2008, Withdrawn, Last updated: 10/02/2021 -
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Orphan designation: Human monoclonal antibody against Pseudomonas aeruginosa IATS-O1 for: Treatment of pneumonia caused by serotype O1 Pseudomonas aeruginosa
Date of first decision: 28/01/2010, Withdrawn, Last updated: 03/02/2021 -
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Orphan designation: RNA, [P-deoxy-P-(dimethylamino)] (2',3'-dideoxy-2',3'-imino-2',3'-seco) (2'a→5') (C-m5U-m5U-A-C-A-G-G-C-m5U-C-C-A-A-m5U-A-G-m5U-G-G-m5U-C-A-G-m5U), 5' [P-[4-[[2-[2-(2-hydroxyethoxy)ethoxy]ethoxy]carbonyl]-1-piperazinyl]-N,N-dimethylaminophosphonamidate], 3'-[2'a-[N2-acetyl-L-arginyl-6-aminohexanoyl-L-arginyl-L-arginyl-β-alanyl-L-arginyl-L-arginyl-6-aminohexanoyl-L-arginyl-L-arginyl-β-alanyl-L-arginyl-6-aminohexanoyl-β-alanyl], octahydrochloride for: Treatment of Duchenne muscular dystrophy
Date of first decision: 02/02/2010, Withdrawn, Last updated: 03/02/2021 -
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Orphan designation: Sodium thiosulfate for: Treatment of calciphylaxis
Date of first decision: 23/02/2011, Withdrawn, Last updated: 03/02/2021 -
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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Date of first decision: 18/11/2016, Withdrawn, Last updated: 03/02/2021 -
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Orphan designation: Heparin-activated recombinant human fibroblast growth factor 1 (on a biodegradable device made from alpha-calcium sulfate hemihydrate) for: Treatment of traumatic spinal cord injury
Date of first decision: 27/07/2010, Withdrawn, Last updated: 03/02/2021 -
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Orphan designation: (1S,3S)-3-amino-4-(difluoromethylene) cyclopentanecarboxylic acid hydrochloride for: Treatment of West syndrome
Date of first decision: 09/02/2012, Withdrawn, Last updated: 03/02/2021 -
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Orphan designation: Rovalpituzumab tesirine for: Treatment of small cell lung cancer
Date of first decision: 30/05/2016, Withdrawn, Last updated: 03/02/2021 -
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Orphan designation: rasagiline for: Treatment of Duchenne muscular dystrophy
Date of first decision: 28/06/2019, Withdrawn, Last updated: 12/01/2021 -
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Orphan designation: Tetrahydrobiopterin for: Treatment of hyperphenylalaninaemia
Date of first decision: 08/06/2004, Withdrawn, Last updated: 11/01/2021 -
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Orphan designation: adeno-associated viral vector serotype 2 containing the human REP1 gene (timrepigene emparvovec) for: Treatment of spinal muscular atrophy
Date of first decision: 24/08/2018, Withdrawn, Last updated: 07/01/2021 -
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Orphan designation: Antisense oligonucleotide targeting the F508delta mutation of CFTR (eluforsen) for: Treatment of cystic fibrosis
Date of first decision: 07/10/2013, Withdrawn, Last updated: 18/12/2020 -
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Orphan designation: 1-(6-benzothiazolylsulfonyl)-5-chloro-1H-indole-2-butanoic acid (lanifibranor) for: Treatment of systemic sclerosis
Date of first decision: 19/11/2014, Withdrawn, Last updated: 14/12/2020 -
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Orphan designation: 5-amino-1-(2-methyl-1H-benzo[d]imidazol-5-yl)-1H-pyrazol-4-yl 1H-indol-2-yl ketone mono[(S)-2-hydroxysuccinate] for: Treatment of biliary tract cancer
Date of first decision: 16/10/2017, Withdrawn, Last updated: 11/12/2020 -
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Orphan designation: Recombinant antibody derivative against human CD19 and CD3 (blinatumomab) for: Treatment of chronic lymphocytic leukaemia
Date of first decision: 01/12/2003, Withdrawn, Last updated: 11/12/2020 -
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Orphan designation: Recombinant antibody derivative against human CD19 and CD3 (blinatumomab) for: Treatment of mantle cell lymphoma
Date of first decision: 01/12/2003, Withdrawn, Last updated: 11/12/2020 -
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Orphan designation: pentetrazol for: Treatment of idiopathic hypersomnia
Date of first decision: 11/11/2015, Withdrawn, Last updated: 10/12/2020 -
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Orphan designation: copper meso-5,15-bis[3-[(1,2-dicarba-closo-dodecaboranyl)methoxy]phenyl]-meso-10,20-dinitroporphyrin for: Treatment of squamous-cell carcinoma of the head and neck in patients undergoing radiotherapy
Date of first decision: 27/06/2013, Withdrawn, Last updated: 10/12/2020 -
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Orphan designation: Ecothiopate iodide for: Treatment of Stargardt's disease
Date of first decision: 24/04/2015, Withdrawn, Last updated: 10/12/2020 -
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Orphan designation: Allantoin for: Treatment of epidermolysis bullosa
Date of first decision: 16/01/2014, Withdrawn, Last updated: 10/12/2020 -
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Orphan designation: Alpha-1 proteinase inhibitor for: Treatment of cystic fibrosis
Date of first decision: 14/09/2007, Withdrawn, Last updated: 08/12/2020 -
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Orphan designation: Sodium cromoglicate for: Treatment of idiopathic pulmonary fibrosis
Date of first decision: 26/06/2020, Withdrawn, Last updated: 08/12/2020 -
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Orphan designation: L-cysteine, L-leucyl-L-alpha-glutamyl-L-alpha-glutamyl-L-lysyl-L-lysylglycyl-L-asparaginyl-L-tyrosyl-L-valyl-L-valyl-L-threonyl-L-alpha-aspartyl-L-histidyl-S-[1-[(4-carboxycyclohexyl)methyl]-2,5-dioxo-3-pyrrolidinyl]-complex with keyhole limpet haemocyanin for: Treatment of glioma
Date of first decision: 27/10/2011, Withdrawn, Last updated: 04/12/2020 -
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Orphan designation: Adeno-associated viral vector serotype Anc80 containing the truncated human ATP7B gene under the control of the human alpha-1 antitrypsin promoter for: Treatment of Wilson's disease
Date of first decision: 23/08/2017, Withdrawn, Last updated: 03/12/2020 -
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Orphan designation: Sodium (2R,3S,5R)-5-(4-amino-2-oxo-1,3,5-triazin-1(2H)-yl)-2-(hydroxymethyl)tetrahydrofuran-3-yl ((2R,3S,5R)-5-(2-amino-6-oxo-1H-purin-9(6H)-yl)-3-hydroxytetrahydrofuran-2-yl)methyl phosphate (guadecitabine) for: Treatment of acute myeloid leukaemia
Date of first decision: 14/12/2015, Withdrawn, Last updated: 01/12/2020