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4190 results
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Orphan designation: Thalidomide for: Treatment of graft-versus-host disease
Date of first decision: 09/07/2001, Withdrawn, Last updated: 15/10/2009 -
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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: ciclosporin for: Prevention of rejection for corneal transplant
Date of first decision: 03/11/2006, Withdrawn, Last updated: 15/03/2010 -
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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 -
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Orphan designation: Cladribine for: Treatment of indolent non-Hodgkin's lymphoma
Date of first decision: 18/09/2001, Expired, Last updated: 18/06/2014 -
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Orphan designation: Treprostinil diethanolamine for: Treatment of pulmonary arterial hypertension and chronic thromboembolic pulmonary hypertension
Date of first decision: 26/08/2005, Positive, Last updated: 14/03/2019 -
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Orphan designation: (Z)-N-[2-(Diethylamino)ethyl]-5-[(5-fluoro-2-oxo-1,2-dihydro-3H-indol-3-ylidene)methyl]-2,4-dimethyl-1H-pyrrole-3-carboxamide (S)-2-hydroxysyccinate for: Treatment of malignant gastrointestinal stromal tumours
Date of first decision: 10/03/2005, Withdrawn, Last updated: 15/10/2009 -
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Orphan designation: Volasertib for: Treatment of acute myeloid leukaemia
Date of first decision: 26/03/2014, Positive, -
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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
Date of first decision: 11/11/2015, Positive, Last updated: 09/11/2020 -
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Orphan designation: 5,6,7,8-Tetrahydrobiopterin for: Treatment of hyperphenylalaninaemia
Date of first decision: 02/10/2003, Positive, Last updated: 23/02/2007 -
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Orphan designation: Tralokinumab for: Treatment of idiopathic pulmonary fibrosis
Date of first decision: 08/11/2012, Withdrawn, -
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Orphan designation: Caffeine citrate for: Prevention of bronchopulmonary dysplasia
Date of first decision: 11/04/2014, 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: Recombinant human serum amyloid P for: Prevention of scarring post glaucoma filtration surgery
Date of first decision: 09/10/2009, Withdrawn, -
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Orphan designation: chlorproguanil hydrochloride, dapsone for: Treatment of acute uncomplicated plasmodium falciparum malaria
Date of first decision: 26/07/2002, Negative, Last updated: 08/01/2003 -
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Orphan designation: Methotrexate for: Treatment of alkaptonuria
Date of first decision: 29/08/2016, Positive, -
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Orphan designation: Vatreptacog alfa (activated) for: Treatment of haemophilia B
Date of first decision: 09/08/2012, Withdrawn, -
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Orphan designation: 1, 1'-[1,4-Phenylenebis (methylene)]-bis-1,4,8,11-tetraazacyclotetradecane for: Treatment to mobilize progenitor cells prior to stem cell transplantation
Date of first decision: 20/10/2004, Positive, Last updated: 18/08/2008