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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 for: Treatment of pulmonary arterial hypertension
Date of first decision: 29/05/2019, Positive, Last updated: 09/08/2019 -
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Orphan designation: Emixustat hydrochloride for: Treatment of Stargardt's disease
Date of first decision: 29/05/2019, Positive, Last updated: 09/08/2019 -
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Orphan designation: Diacerein for: Treatment of epidermolysis bullosa
Date of first decision: 29/05/2019, Positive, Last updated: 19/08/2019 -
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Orphan designation: Zanubrutinib for: Treatment of lymphoplasmacytic lymphoma
Date of first decision: 29/05/2019, Positive, Last updated: 12/08/2019 -
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Orphan designation: Allogeneic skin-derived ABCB5-positive mesenchymal stem cells for: Treatment of epidermolysis bullosa
Date of first decision: 29/05/2019, Positive, Last updated: 09/08/2019 -
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Orphan designation: Relacorilant for: Treatment of Cushing's syndrome
Date of first decision: 29/05/2019, Positive, Last updated: 09/08/2019 -
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Orphan designation: Sodium benzoate, sodium phenylacetate for: Treatment of carbamoyl-phosphate synthase-1 deficiency
Date of first decision: 29/05/2019, Positive, Last updated: 12/08/2019 -
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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 for: Treatment of acute myeloid leukaemia
Date of first decision: 29/05/2019, Positive, Last updated: 07/05/2020 -
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Orphan designation: N-(trans-3-(5-((R)-1-hydroxyethyl)-1,3,4-oxadiazol-2-yl)cyclobutyl)-3-phenylisoxazole-5-carboxamide for: Treatment of cystic fibrosis
Date of first decision: 29/05/2019, Positive, Last updated: 09/08/2019 -
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Orphan designation: Sodium benzoate, sodium phenylacetate for: Treatment of citrullinaemia type 1
Date of first decision: 29/05/2019, Positive, Last updated: 12/08/2019