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15 results
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Orphan designation: Refusal of orphan designation for the treatment of non-small cell lung cancer with EGFR alterations
Date of refusal of designation: 28/06/2021, Negative, Last updated: 24/01/2022 -
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Orphan designation: Humanised IgG2 monoclonal antibody against TNFSF13 for: Treatment of primary IgA nephropathy
Date of designation: 21/06/2021, Positive, Last updated: 11/05/2022 -
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Orphan designation: L-ergothioneine for: Treatment of cystinuria
Date of designation: 21/06/2021, Positive, Last updated: 11/05/2022 -
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Orphan designation: Tislelizumab for: Treatment of nasopharyngeal cancer
Date of designation: 21/06/2021, Positive, Last updated: 11/05/2022 -
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Orphan designation: Adeno-associated virus serotype 9 expressing human CLN5 for: Treatment of neuronal ceroid lipofuscinosis
Date of designation: 21/06/2021, Positive, Last updated: 11/05/2022 -
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Orphan designation: Allogenic placenta-derived mesenchymal stromal cells secretome for: Treatment of pre-eclampsia
Date of designation: 21/06/2021, Positive, Last updated: 11/05/2022 -
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Orphan designation: imatinib for: Treatment of pulmonary arterial hypertension
Date of designation: 21/06/2021, Positive, Last updated: 11/05/2022 -
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Orphan designation: (S)-5-amino-3-(4-((5-fluoro-2-methoxybenzamido)methyl)phenyl)-1-(1,1,1-trifluoropropane-2-yl)-1H-pyrazole-4-carboxamide for: Treatment of mantle cell lymphoma
Date of designation: 21/06/2021, Positive, Last updated: 11/05/2022 -
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Orphan designation: Bomedemstat ditosilate for: Treatment of essential thrombocythaemia
Date of designation: 21/06/2021, Positive, Last updated: 11/05/2022 -
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Orphan designation: Hydrocortisone hydrogen succinate for: Prevention of bronchopulmonary dysplasia
Date of designation: 21/06/2021, Positive, Last updated: 11/05/2022 -
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Orphan designation: melatonin for: Prevention of retinopathy of prematurity
Date of designation: 21/06/2021, Positive, Last updated: 11/05/2022 -
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Orphan designation: Humanised monoclonal antibody derivative against fibroblast growth factor receptor 3 for: Treatment of achondroplasia
Date of designation: 21/06/2021, Positive, Last updated: 11/05/2022 -
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Orphan designation: H-D-valyl1-D-alanyl-D-glutamyl-D-alanyl-D-arginyl5-D-glutamyl-D-glutamyl-D-leucyl-D-glutamyl-D-arginyl10-D-leucyl-D-glutamyl-D-alanyl-D-arginyl-D-leucyl15-glycyl-D-glutaminyl-D-alanyl-D-arginyl-glycyl20-D-glutamyl-D-leucyl-D-lysyl-D-lysyl-D-tryptophyl25-D-lysyl-D-methionyl-D-arginyl-D-arginyl-D-asparaginyl30-D-glutaminyl-D-phenylalanyl-D-tryptophyl-D-leucyl-D-lysyl35-D-leucyl-D-glutaminyl-D-arginine for: Treatment of glioma
Date of designation: 21/06/2021, Positive, Last updated: 11/05/2022 -
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Orphan designation: Adeno-associated virus serotype 9 containing the human FXN gene isoform 1 for: Treatment of Friedreich's ataxia
Date of designation: 21/06/2021, Withdrawn, Last updated: 11/05/2022 -
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Orphan designation: Adeno-associated viral vector LK03 encoding human methylmalonyl-CoA mutase for: Treatment of methylmalonic acidaemia
Date of designation: 21/06/2021, Positive, Last updated: 11/05/2022