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1728 results
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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: 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: Treprostinil diethanolamine (oral use) 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: 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: 5-aminolevulinic acid for: Treatment of glioma
Date of first decision: 12/01/2017, Positive, -
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Orphan designation: Autologous CD34+ haematopoietic stem cells transduced with lentiviral vector encoding the human beta A-T87Q-globin gene for the treatment of sickle cell disease for: The treatment of sickle cell disease
Date of first decision: 29/04/2014, Positive, Last updated: 13/01/2020 -
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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: Synthetic double-stranded siRNA oligonucleotide directed against antithrombin mRNA and covalently linked to a ligand containing three N-acetylgalactosamine residues (fitusiran) for: Treatment of haemophilia A
Date of first decision: 29/07/2014, Positive, Last updated: 13/06/2018 -
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Orphan designation: Donor T lymphocytes depleted ex vivo of host alloreactive T cells using photodynamic treatment- for: Treatment in haematopoietic stem cell transplantation
Date of first decision: 27/06/2016, Positive, Last updated: 26/07/2016 -
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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: 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: 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: 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: 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: Lithium citrate tetrahydrate (in reverse-micelle formulation) for: Treatment of Huntington’s disease
Date of first decision: 28/01/2010, Positive, Last updated: 25/02/2010 -
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Orphan designation: Recombinant human placental growth factor for: Treatment of pre-eclampsia
Date of first decision: 27/06/2018, Positive, -
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Orphan designation: 3-(5-Amino-2-methyl-4-oxoquinazolin-3(4H)-yl)piperidine-2,6-dione hydrochloride- for: Treatment of diffuse large B-cell lymphoma
Date of first decision: 27/06/2016, Positive, Last updated: 23/01/2019 -
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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: 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: 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,