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8 results
Categories
Human Remove Human filter
Decision date
2019-04-30T22:00:00Z – 2019-06-01T21:59:59Z Remove 2019-04-30T22:00:00Z – 2019-06-01T21:59:59Z filter
Orphan designation status
Positive Remove Positive filter
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Orphan designation: (S)-3-((3-(1-((6-(3,4-dimethoxyphenyl)pryazin-2-yl)amino)ethyl)phenyl)carbamoyl)-5-methylpridin-1-ium (seralutinib) for: Treatment of pulmonary arterial hypertension
Date of designation: 29/05/2019, Positive, Last updated: 30/09/2022 -
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Orphan designation: (S)-5-(1-(6-chloro-2-oxo-1,2-dihydroquinolin-3-yl)ethylamino-1-methyl-6-oxo-1,6-dihydropyridine-2-carbonitrile (olutasidenib) for: Treatment of acute myeloid leukaemia
Date of designation: 29/05/2019, Positive, Last updated: 23/01/2023 -
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Orphan designation: Allogeneic skin-derived ABCB5-positive mesenchymal stem cells for: Treatment of epidermolysis bullosa
Date of designation: 29/05/2019, Positive, Last updated: 09/08/2019 -
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Orphan designation: Diacerein for: Treatment of epidermolysis bullosa
Date of designation: 29/05/2019, Positive, Last updated: 19/08/2019 -
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Orphan designation: Emixustat hydrochloride for: Treatment of Stargardt's disease
Date of designation: 29/05/2019, Positive, Last updated: 09/08/2019 -
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Orphan designation: Relacorilant for: Treatment of Cushing's syndrome
Date of designation: 29/05/2019, Positive, Last updated: 09/08/2019 -
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Orphan designation: Sodium phenylacetate, Sodium benzoate for: Treatment of citrullinaemia type 1
Date of designation: 29/05/2019, Positive, Last updated: 12/08/2019 -
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Orphan designation: Sodium benzoate for: Treatment of carbamoyl-phosphate synthase-1 deficiency
Date of designation: 29/05/2019, Positive, Last updated: 12/08/2019