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Orphan designation: Hemopexin for: Treatment of sickle cell disease
Date of designation: 27/07/2020, Positive, Last updated: 19/11/2020 -
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Orphan designation: C-type natriuretic peptide conjugated to multi-arm polyethylene glycol carrier through a cleavable linker for: Treatment of achondroplasia
Date of designation: 27/07/2020, Positive, Last updated: 19/11/2020 -
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Orphan designation: Fasudil hydrochloride for: Treatment of non-traumatic subarachnoid haemorrhage
Date of designation: 27/07/2020, Positive, Last updated: 19/11/2020 -
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Orphan designation: Pegylated adrenomedullin for: Treatment of acute respiratory distress syndrome (ARDS)
Date of designation: 27/07/2020, Positive, Last updated: 22/12/2022 -
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Orphan designation: Triheptanoin for: Treatment of carnitine-acylcarnitine translocase deficiency
Date of designation: 27/07/2020, Positive, Last updated: 19/11/2020 -
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Orphan designation: Adeno-associated viral vector expressing acid alpha-glucosidase gene for: Treatment of glycogen storage disease type II (Pompe's disease)
Date of designation: 27/07/2020, Positive, Last updated: 08/12/2020 -
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Orphan designation: Refusal of orphan designation for the treatment of intracerebral haemorrhage
Date of refusal of designation: 06/07/2020, Negative, Last updated: 19/11/2020 -
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Orphan designation: Maralixibat chloride for: Treatment of biliary atresia
Date of designation: 27/07/2020, Positive, Last updated: 24/09/2021 -
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Orphan designation: Imetelstat sodium for: Treatment of myelodysplastic syndromes
Date of designation: 27/07/2020, Positive, Last updated: 19/11/2020 -
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Orphan designation: Retinol palmitate for: Prevention of bronchopulmonary dysplasia
Date of designation: 27/07/2020, Positive, Last updated: 19/11/2020 -
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Orphan designation: Tinostamustine for: Treatment of T-cell prolymphocytic leukaemia
Date of designation: 27/07/2020, Positive, Last updated: 19/11/2020