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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
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
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
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
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
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
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: 3-(3-(3,5-bis(trifluoromethyl)phenyl)-1h-pyrazol-1-yl)propanoic acid for: Treatment of Stargardt's disease
Date of designation: 24/04/2019, Withdrawn, Last updated: 13/04/2023 -
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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 -
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Orphan designation: arimoclomol citrate for: Treatment of Niemann-Pick's disease, type C
Date of designation: 19/11/2014, Positive, Last updated: 11/04/2023 -
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Orphan designation: (R)-1-[1-(4-acetoxy-3,3-dimethyl-2-oxo-butyl)-2-oxo-5-(pyridin-2-yl)-2,3-dihydro-1H-benzo[e][1,4]diazepin-3-yl]-3-(3-methylamino-phenyl)-urea (ceclazepide) for: Treatment of gastro-entero-pancreatic neuroendocrine tumours
Date of designation: 14/12/2015, Withdrawn, Last updated: 04/04/2023 -
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Orphan designation: Streptococcus pyogenes, group A, type 3, strain Su, inactivated for: Treatment of lymphatic malformations
Date of designation: 16/05/2022, Positive, Last updated: 04/04/2023 -
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Orphan designation: Tobramycin for: Treatment of Pseudomonas aeruginosa lung infection in cystic fibrosis
Date of designation: 17/03/2003, Positive, Last updated: 03/04/2023 -
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Orphan designation: Synthetic ribonucleic acid oligonucleotide directed against superoxide dismutase 1 messenger ribonucleic acid (tofersen) for: Treatment of amyotrophic lateral sclerosis
Date of designation: 29/08/2016, Positive, Last updated: 03/04/2023 -
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Orphan designation: Humanised anti-alpha Ī½ beta 6 monoclonal antibody for: Treatment of idiopathic pulmonary fibrosis
Date of designation: 29/07/2014, Positive, Last updated: 03/04/2023 -
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Orphan designation: 2-(3-(4-(1H-Indazol-5-ylamino)quinazolin-2-yl)phenoxy)-N-isopropylacetamide-methane sulfonic acid salt for: Treatment of graft-versus-host disease
Date of designation: 17/10/2019, Positive, Last updated: 03/04/2023