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Orphan designation: (E)-2-((2S,4S)-4-(((2R,4S,5S,6S)-4-amino-5-hydroxy-6-methyltetrahydro-2H-pyran-2-yl)oxy)-2,5,12-trihydroxy-7-methoxy-6,11-dioxo-1,2,3,4,6,11-hexahydrotetracen-2-yl)-10-(carboxymethyl)-1-hydroxy-13-(2-(2-(2-((E)-3-(3-((3-hydroxy-3,3 diphosphonopropyl)(methyl)amino)propoxy)benzylidene)hydrazine-1-carbonothioyl)hydrazineyl)-2-oxoethyl)-8-oxo-5-thioxo-3,4,6,7,10,13-hexaazapentadec-2-en-15-oic acid for: Treatment of osteosarcoma
Date of designation: 16/12/2019, Positive, Last updated: 24/02/2020 -
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Orphan designation: 2-(isopropylamino)-3-methyl-5-(6-methyl-5-((2-(1-methyl-1H-pyrazol-4-yl)pyridin-4-yl)oxy)pyridin-2-yl)pyrimidin-4(3H)-one for: Treatment of tenosynovial giant cell tumour, localised and diffuse type
Date of designation: 16/12/2019, Positive, Last updated: 25/08/2021 -
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Orphan designation: 5,7-dichloro-2-((ethylamino)methyl)-8-hydroxy-3-methylquinazolin-4(3H)-one mesilate for: Treatment of multiple system atrophy
Date of designation: 16/12/2019, Positive, Last updated: 27/10/2020 -
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Orphan designation: Adeno-associated virus vector encoding human phenylalanine hydroxylase for: Treatment of phenylalanine hydroxylase deficiency
Date of designation: 16/12/2019, Positive, Last updated: 25/02/2020 -
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Orphan designation: Efgartigimod alfa for: Treatment of immune thrombocytopenia
Date of designation: 16/12/2019, Positive, Last updated: 02/09/2021 -
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Orphan designation: H-Leu-Pro-Pro-Leu-Pro-Tyr-Pro-OH for: Treatment of amyotrophic lateral sclerosis
Date of designation: 16/12/2019, Positive, Last updated: 25/02/2020 -
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Orphan designation: Lactobacillus plantarum for: Treatment of amyotrophic lateral sclerosis
Date of designation: 16/12/2019, Positive, Last updated: 25/02/2020 -
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Orphan designation: navitoclax for: Treatment of myelofibrosis
Date of designation: 16/12/2019, Positive, Last updated: 25/02/2020 -
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Orphan designation: Pamrevlumab for: Treatment of Duchenne muscular dystrophy
Date of designation: 16/12/2019, Positive, Last updated: 13/10/2021 -
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Orphan designation: Synthetic double-stranded siRNA oligonucleotide directed against SERPINA1 mRNA and containing four modified nucleosides which form a ligand cluster of four N-acetylgalactosamine residues for: Treatment of congenital alpha-1 antitrypsin deficiency
Date of designation: 16/12/2019, Positive, Last updated: 24/02/2023