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2361 results
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Orphan designation: dimethyl fumarate for: Adjunctive treatment in haematopoietic cell transplantation (updated)
Date of first decision: 21/03/2018, Positive, Last updated: 15/01/2021 -
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Orphan designation: rasagiline for: Treatment of Duchenne muscular dystrophy (updated)
Date of first decision: 28/06/2019, Withdrawn, Last updated: 12/01/2021 -
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Orphan designation: Tetrahydrobiopterin for: Treatment of hyperphenylalaninaemia (updated)
Date of first decision: 08/06/2004, Withdrawn, Last updated: 11/01/2021 -
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Orphan designation: Fenfluramine hydrochloride for: Treatment of Dravet syndrome (updated)
Date of first decision: 18/12/2013, Positive, Last updated: 08/01/2021 -
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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 (updated)
Date of first decision: 24/08/2018, Withdrawn, Last updated: 07/01/2021 -
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Orphan designation: Autologous CD34+ cells transfected with lentiviral vector containing the human arylsulfatase A cDNA for: treatment of metachromatic leukodystrophy (updated)
Date of first decision: 13/04/2007, Positive, Last updated: 22/12/2020 -
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Orphan designation: Miransertib for: Treatment of Proteus syndrome (updated)
Date of first decision: 21/03/2018, Positive, Last updated: 22/12/2020 -
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Orphan designation: Obeticholic acid for: Treatment of primary sclerosing cholangitis (updated)
Date of first decision: 16/03/2014, Positive, Last updated: 22/12/2020 -
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Orphan designation: Ibutamoren mesilate for: Treatment of growth hormone deficiency (updated)
Date of first decision: 20/06/2017, Positive, Last updated: 22/12/2020 -
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Orphan designation: Bardoxolone methyl for: Treatment of Alport syndrome
Date of first decision: 25/05/2018, Positive, Last updated: 21/12/2020 -
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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: Treosulfan for: Conditioning treatment prior to haematopoietic-progenitor-cell transplantation
Date of first decision: 22/02/2004, Positive, Last updated: 11/12/2020 -
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Orphan designation: Adeno-associated viral vector serotype 9 containing the human SMN gene (onasemnogene abeparvovec) for: Treatment of spinal muscular atrophy
Date of first decision: 19/06/2015, Positive, Last updated: 10/12/2020 -
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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: (S)-2-nitro-6-(4-trifluoromethoxy)benzyloxy)-6,7-dihydro-5H-imidazo[2,1-b] [1,3] oxazine (pretomanid) for: Treatment of tuberculosis
Date of first decision: 29/11/2007, Positive, Last updated: 09/12/2020 -
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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: Adeno-associated viral vector expressing acid alpha-glucosidase gene for: Treatment of glycogen storage disease type II (Pompe's disease)
Date of first decision: 27/07/2020, Positive, Last updated: 08/12/2020