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Orphan designation: Alpha-L-iduronidase fused to Fab fragment of a humanised monoclonal antibody targeting human transferrin receptor for: Treatment of mucopolysaccharidosis type I
Date of designation: 26/03/2021, Positive, Last updated: 19/01/2022 -
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Orphan designation: Lefitolimod for: Treatment of small cell lung cancer
Date of designation: 26/03/2021, Positive, Last updated: 19/05/2022 -
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Orphan designation: 5-fluoro-4-(7'-fluoro-2'-methylspiro[cyclopentane-1,3'-indol]-5'-yl)-N-(5-(1-methylpiperidin-4-yl)pyridin-2-yl)pyrimidin-2-amine for: Treatment of glioma
Date of designation: 26/03/2021, Positive, Last updated: 25/02/2022 -
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Orphan designation: Adeno-associated virus serotype hu68 containing the human GALC gene for: Treatment of Krabbe disease
Date of designation: 26/03/2021, Positive, Last updated: 19/01/2022 -
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Orphan designation: Ilixadencel for: Treatment of gastrointestinal stromal tumours
Date of designation: 26/03/2021, Positive, Last updated: 28/09/2022 -
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Orphan designation: Messenger RNA encoding Cas9, single guide RNA targeting the human TTR gene for: Treatment of ATTR amyloidosis
Date of designation: 26/03/2021, Positive, Last updated: 19/01/2022 -
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Orphan designation: Alpelisib for: Treatment of PIK3CA related overgrowth spectrum
Date of designation: 26/03/2021, Positive, Last updated: 19/01/2022 -
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Orphan designation: Herpes simplex virus 1 expressing the human CFTR gene for: Treatment of cystic fibrosis
Date of designation: 26/03/2021, Positive, Last updated: 17/01/2023 -
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Orphan designation: Lorcaserin hydrochloride for: Treatment of Dravet syndrome
Date of designation: 26/03/2021, Positive, Last updated: 19/01/2022 -
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Orphan designation: Vatiquinone for: Treatment of Friedreich's ataxia
Date of designation: 26/03/2021, Positive, Last updated: 19/01/2022