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12 results
Decision date
2019-05-31T22:00:00Z – 2019-07-01T21:59:59Z Remove 2019-05-31T22:00:00Z – 2019-07-01T21:59:59Z filter
Orphan designation status
Positive Remove Positive filter
Categories
Human Remove Human filter
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Orphan designation: Sodium benzoate, Sodium phenylacetate for: Treatment of hyperargininaemia
Date of first decision: 28/06/2019, Positive, Last updated: 14/10/2019 -
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Orphan designation: (S)-6-hydroxy-2,5,7,8-tetramethyl-N-((R)-piperidine-3-yl)chroman-2-carboxamide hydrochloride for: Treatment of maternally-inherited diabetes and deafness
Date of first decision: 28/06/2019, Positive, Last updated: 02/10/2019 -
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Orphan designation: regorafenib for: Treatment of glioma
Date of first decision: 28/06/2019, Positive, Last updated: 14/10/2019 -
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Orphan designation: Sodium benzoate, Sodium phenylacetate for: Treatment of argininosuccinic aciduria
Date of first decision: 28/06/2019, Positive, Last updated: 14/10/2019 -
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Orphan designation: Reldesemtiv for: Treatment of spinal muscular atrophy
Date of first decision: 28/06/2019, Positive, Last updated: 02/10/2019 -
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Orphan designation: Gaboxadol monohydrate for: Treatment of Angelman syndrome
Date of first decision: 28/06/2019, Positive, Last updated: 07/10/2019 -
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Orphan designation: Recombinant adeno-associated viral vector containing a bioengineered capsid serotype AAV-rh74 and a codon-optimised expression cassette to drive the expression of a secretable form of human acid alpha-glucosidase for: Treatment of glycogen storage disease type II (Pompe's disease)
Date of first decision: 28/06/2019, Positive, Last updated: 10/10/2019 -
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Orphan designation: 2-(2-{[2-(1H-benzimidazol-2-yl)ethyl]amino}ethyl)-N-[(3-fluoropyridine-2-yl)methyl]-1,3-oxazole-4-carboxamide trihydrochloride for: Treatment of beta-thalassaemia intermedia and major
Date of first decision: 28/06/2019, Positive, Last updated: 02/10/2019 -
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Orphan designation: Imidazolyl ethanamide pentandioic acid for: Treatment of acute radiation syndrome
Date of first decision: 28/06/2019, Positive, Last updated: 04/05/2020 -
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Orphan designation: rasagiline for: Treatment of Duchenne muscular dystrophy
Date of first decision: 28/06/2019, Positive, Last updated: 10/10/2019 -
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Orphan designation: 5'-cEtG-sp-cEt5MeU-sp-cEt5MeU-sp-dT-sp-dA-sp-dT-sp-dT-sp-dA-sp-dT-sp-dA-sp-dG-sp-dG-sp-dG-sp-cEt5MeC-sp-cEt5MeU-sp-cEt5MeU-3' (also known as DYN101) for: Treatment of centronuclear myopathies
Date of first decision: 28/06/2019, Positive, Last updated: 03/10/2019 -
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Orphan designation: Recombinant human coagulation factor VIII Fc - von Willebrand factor - XTEN fusion protein for: Treatment of haemophilia A
Date of first decision: 28/06/2019, Positive, Last updated: 14/10/2019