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16 results
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Orphan designation: 2'-O-methyl phosphorothioate RNA oligonucleotide, 5'-m5CUGm5CUGm5CUGm5CUGm5CUGm5CUGm5CUG-3' for: Treatment of spinocerebellar ataxia
Date of designation: 19/02/2021, Positive, Last updated: 22/02/2022 -
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Orphan designation: 18-(p-[131I]-iodophenyl)octadecyl phosphocholine for: Treatment of lymphoplasmacytic lymphoma
Date of designation: 19/02/2021, Positive, Last updated: 22/02/2022 -
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Orphan designation: Anti-SIGLEC8 IgG1 humanised monoclonal antibody for: Treatment of eosinophilic gastroenteritis
Date of designation: 19/02/2021, Positive, Last updated: 22/02/2022 -
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Orphan designation: (1R,3S,5R)-2-(2-(3-acetyl-5-(2-methylpyrimidin-5-yl)-1H-indazol-1-yl)acetyl)-N-(6-bromo-3-methylpyridin-2-yl)-5-methyl-2-azabicyclo[3.1.0]hexane-3-carboxamide for: Treatment of paroxysmal nocturnal haemoglobinuria
Date of designation: 19/02/2021, Positive, Last updated: 22/02/2022 -
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Orphan designation: 2-(2-{[2-(1h-benzimidazol-2-yl)ethyl]amino}ethyl)-n-[(3-fluoropyridin-2-yl)methyl]-1,3-oxazole-4-carboxamide trihydrochloride for: Treatment of sickle cell disease
Date of designation: 19/02/2021, Positive, Last updated: 22/02/2022 -
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Orphan designation: Autologous T lymphocyte-enriched population of cells transduced with a lentiviral vector encoding a chimeric antigen receptor targeting human claudin 18.2 antigen with CD28 and CD3-zeta intracellular signalling domains for: Treatment of gastric cancer
Date of designation: 19/02/2021, Positive, Last updated: 23/02/2022 -
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Orphan designation: Autologous CD34+ cells transduced with a lentiviral RNA vector that results in integrated cDNA encoding for functional cystinosin for: Treatment of cystinosis
Date of designation: 19/02/2021, Positive, Last updated: 18/05/2022 -
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Orphan designation: Adeno-associated virus serotype 5 containing the human NR2E3 gene for: Treatment of retinitis pigmentosa
Date of designation: 19/02/2021, Positive, Last updated: 23/02/2022 -
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Orphan designation: Dodecyl creatine ester, dodecyl creatine ester hydrochloride for: Treatment of creatine deficiency syndromes
Date of designation: 19/02/2021, Positive, Last updated: 06/02/2023 -
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Orphan designation: Gly-Arg-Lys-Lys-Arg-Arg-Gln-Arg-Arg-Arg-Cys-Asp-Met-Ala-Glu-His-Thr-Glu-Arg-Leu-Lys-Ala-Asn-Asp-Ser-Leu-Lys-Leu-Ser-Gln-Glu-Tyr-Glu-Ser-Ile-NH2 for: Treatment of spinal cord injury
Date of designation: 19/02/2021, Positive, Last updated: 23/02/2022 -
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Orphan designation: Adeno-associated virus serotype 5 containing the human NR2E3 gene for: Treatment of Leber's congenital amaurosis
Date of designation: 19/02/2021, Positive, Last updated: 24/02/2022 -
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Orphan designation: Adeno-associated viral vector serotype 9 expressing codon-optimized human GBA gene for: Treatment of Gaucher disease
Date of designation: 19/02/2021, Positive, Last updated: 02/03/2022 -
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Orphan designation: (2S)-2-[(2S)-4-cyclohexyl-2-{[(2S)-1-(4-fluorobenzoyl)pyrrolidin-2-yl]formamido}butanamido]-N-(1-{[(1S)-1-{[(1S)-1-({1-[(1-{[2-({1-[(dimethylamino)methyl]cyclobutyl}carbamoyl)ethyl]carbamoyl}-1-methylethyl)carbamoyl]-1-methylethyl}carbamoyl)-3-methylbutyl]carbamoyl}-3-methylbutyl]carbamoyl}-1-methylethyl)-4-methylpentanamide acetate for: Treatment of leishmaniasis
Date of designation: 19/02/2021, Positive, Last updated: 22/02/2022 -
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Orphan designation: Autologous CD34+ hematopoietic stem and progenitor cells transfected with zinc finger nuclease mRNAs SB-mRENH1 and SB-mRENH2 for: Treatment of sickle cell disease
Date of designation: 19/02/2021, Positive, Last updated: 25/04/2022 -
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Orphan designation: 2-[6-(6,7-Dimethoxyquinolin-3-yl)pyridin-3-yl]-N-[3-(1,1,1-trifluoro-2-methylpropan-2-yl)-1,2-oxazol-5-yl]acetamide (zeteletinib) for: Treatment of medullary thyroid carcinoma
Date of designation: 19/02/2021, Positive, Last updated: 13/09/2023 -
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Orphan designation: Tebentafusp for: Treatment of uveal melanoma
Date of designation: 19/02/2021, Positive, Last updated: 22/04/2022