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Medicine
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Orphan designation: Viltolarsen for: Treatment of Duchenne muscular dystrophy (updated)
Date of designation: 04/06/2020, Positive, Last updated: 01/06/2023 -
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Orphan designation: Ivosidenib for: Treatment of biliary tract cancer (updated)
Date of designation: 21/03/2018, Positive, Last updated: 12/05/2023 -
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Orphan designation: Ivosidenib for: Treatment of acute myeloid leukaemia (updated)
Date of designation: 12/12/2016, Positive, Last updated: 12/05/2023 -
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Orphan designation: Govorestat for: Treatment of galactosaemia (updated)
Date of designation: 21/06/2022, Positive, Last updated: 10/05/2023 -
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Orphan designation: N-[(2S)-5-{[(1R, 2S)-2-(4-fluorophenyl)cyclopropyl]amino}-1-(4-methylpiperazin-1-yl)-1-oxopentan-2-yl]-4-(1H-1,2,3-triazol-1-yl)benzamide, bis-tosilate salt (Bomedemstat ditosilate) for: Treatment of myelofibrosis (updated)
Date of designation: 14/10/2016, Positive, Last updated: 08/05/2023 -
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Orphan designation: 1-[(3S)-3-{4-amino-3-[(3,5-dimethoxyphenyl)ethynyl]-1H-pyrazolo[3,4-d]pyrimidin-1-yl}pyrrolidin-1-yl]-2-propen-1-one (futibatinib) for: Treatment of biliary tract cancer (updated)
Date of designation: 01/04/2019, Positive, Last updated: 08/05/2023 -
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Orphan designation: Nimorazole for: Treatment of squamous-cell carcinoma of the head and neck in patients undergoing radiotherapy (updated)
Date of designation: 23/02/2011, Positive, Last updated: 08/05/2023 -
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Orphan designation: Troriluzole hydrochloride for: Treatment of spinocerebellar ataxia (updated)
Date of designation: 10/12/2021, Positive, Last updated: 08/05/2023 -
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Orphan designation: 2-hydroxy-6-((2-(1-isopropyl-1H-pyrazol-5-yl)pyridin-3-yl)methoxy)benzaldehyde (voxelotor) for: Treatment of sickle cell disease (updated)
Date of designation: 18/11/2016, Positive, Last updated: 04/05/2023 -
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Orphan designation: Gallium (68Ga)-edotreotide for: Diagnosis of gastro-entero-pancreatic neuroendocrine tumours (updated)
Date of designation: 19/03/2015, Positive, Last updated: 03/05/2023 -
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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 (updated)
Date of designation: 12/12/2016, Positive, Last updated: 03/05/2023 -
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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 (updated)
Date of designation: 19/02/2014, Positive, Last updated: 03/05/2023 -
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Orphan designation: Lutetium (177Lu)-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-L-cysteinyl-L-threonine-cyclic(2-7)disulfideĀ (lutetium (177Lu) oxodotreotide) for: Treatment of gastro-entero-pancreatic neuroendocrine tumours (updated)
Date of designation: 31/01/2008, Positive, Last updated: 03/05/2023 -
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Orphan designation: Bomedemstat ditosilate for: Treatment of essential thrombocythaemia (updated)
Date of designation: 21/06/2021, Positive, Last updated: 02/05/2023 -
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Orphan designation: Ex-vivo fused autologous human bone marrow-derived mesenchymal stem cell with allogenic human myoblast for: Treatment of Duchenne muscular dystrophy
Date of designation: 31/07/2018, Positive, Last updated: 28/04/2023 -
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Orphan designation: Ex vivo fused normal allogeneic human myoblast with another normal allogeneic human myoblast for: Treatment of Duchenne muscular dystrophy
Date of designation: 19/11/2018, Positive, Last updated: 28/04/2023 -
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Orphan designation: Ile-Ser-Ile-Thr-Glu-Ile-Lys-Gly-Val-Ile-Val-His-Arg-Ile-Glu-Thr-Ile-Leu-Phe-Lys-Lys-Lys-Lys-Glu-Met-Pro-Ser-Glu-Glu-Gly-Tyr-Gln-Asp for: Treatment of multiple system atrophy
Date of designation: 19/11/2018, Positive, Last updated: 28/04/2023 -
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Orphan designation: Ex vivo fused normal allogeneic human myoblast with autologous human myoblast derived from Duchenne muscular dystrophy affected donor for: Treatment of Duchenne muscular dystrophy
Date of designation: 19/11/2019, Positive, Last updated: 28/04/2023 -
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Orphan designation: Tazemetostat for: Treatment of follicular lymphoma
Date of designation: 21/03/2018, Positive, Last updated: 28/04/2023 -
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Orphan designation: Tazemetostat for: Treatment of diffuse large B-cell lymphoma
Date of designation: 21/03/2018, Positive, Last updated: 28/04/2023 -
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Orphan designation: Tazemetostat for: Treatment of malignant mesothelioma
Date of designation: 21/03/2018, Positive, Last updated: 28/04/2023 -
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Orphan designation: thymidine, deoxycytidine for: Treatment of mitochondrial DNA depletion syndrome, myopathic form
Date of designation: 20/04/2017, Positive, Last updated: 25/04/2023 -
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Orphan designation: Fenfluramine hydrochloride for: Treatment of Lennox-Gastaut syndrome
Date of designation: 27/02/2017, Positive, Last updated: 25/04/2023 -
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Orphan designation: Fenfluramine hydrochloride for: Treatment of Dravet syndrome
Date of designation: 18/12/2013, Positive, Last updated: 25/04/2023 -
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Orphan designation: tripotassium citrate monohydrate, Potassium hydrogen carbonate for: Treatment of cystinuria
Date of designation: 09/01/2020, Positive, Last updated: 13/04/2023