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23 results
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Orphan designation: Methotrexate for: Treatment of retinal detachment
Date of designation: 04/06/2020, Positive, Last updated: 19/08/2020 -
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Orphan designation: Autologous T lymphocyte-enriched population of cells transduced with a lentiviral vector encoding a chimeric antigen receptor targeting human B cell maturation antigen with 4-1BB and CD3-zeta intracellular signalling domains for: Treatment of multiple myeloma
Date of designation: 04/06/2020, Positive, Last updated: 19/08/2020 -
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Orphan designation: Rilzabrutinib for: Treatment of immune thrombocytopenia
Date of designation: 04/06/2020, Positive, Last updated: 14/02/2022 -
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Orphan designation: Lumacaftor for: Treatment of non-traumatic subarachnoid haemorrhage
Date of designation: 04/06/2020, Positive, Last updated: 19/08/2020 -
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Orphan designation: Lutetium (177Lu) lilotomab satetraxetan for: Treatment of marginal zone lymphoma
Date of designation: 04/06/2020, Positive, Last updated: 19/08/2020 -
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Orphan designation: (4-{(2S,4S)-4-ethoxy-1-[(5-methoxy-7-methyl-1H-indol-4-yl)methyl]piperidin-2-yl}benzoic acid-hydrogen chloride(1/1)) for: Treatment of paroxysmal nocturnal haemoglobinuria
Date of designation: 04/06/2020, Positive, Last updated: 19/08/2020 -
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Orphan designation: Ile-Ala-Leu-Ile-Leu-Glu-Pro-Ile-Cys-Cys-Gln-Glu-Arg-Ala-Ala-(discrete-polyethylene glycol)24 for: Treatment of neonatal encephalopathy
Date of designation: 04/06/2020, Positive, Last updated: 19/08/2020 -
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Orphan designation: Sodium phenylbutyrate, Tauroursodeoxycholic acid for: Treatment of amyotrophic lateral sclerosis
Date of designation: 04/06/2020, Positive, Last updated: 05/01/2022 -
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Orphan designation: 1-((2S,4S)-2-((((S)-(4-bromophenoxy)(((S)-1-oxo-1-(((S)-pentan-2-yl)oxy)propan-2-yl)amino)phosphoryl)oxy)methyl)-1,3-dioxolan-4-yl)-2-oxo-1,2-dihydropyrimidin-4-aminium chloride for: Treatment of hepatocellular carcinoma
Date of designation: 04/06/2020, Positive, Last updated: 19/08/2020 -
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Orphan designation: Autologous CD4+ and CD8+ T cells transduced with a lentiviral vector encoding an affinity enhanced T cell receptor specific to MAGE-A4 for: Treatment of soft tissue sarcoma
Date of designation: 04/06/2020, Positive, Last updated: 19/08/2020 -
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Orphan designation: Adeno-associated virus serotype 9 containing the human ASPA gene for: Treatment of Canavan disease
Date of designation: 04/06/2020, Positive, Last updated: 06/04/2021 -
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Orphan designation: Magrolimab for: Treatment of myelodysplastic syndromes
Date of designation: 26/06/2020, Positive, Last updated: 16/10/2020 -
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Orphan designation: Nomacopan for: Treatment of bullous pemphigoid
Date of designation: 26/06/2020, Positive, Last updated: 26/01/2022 -
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Orphan designation: stiripentol for: Treatment of primary hyperoxaluria
Date of designation: 26/06/2020, Positive, Last updated: 22/09/2020 -
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Orphan designation: Onfekafusp alfa for: Treatment of glioma
Date of designation: 26/06/2020, Positive, Last updated: 22/09/2020 -
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Orphan designation: Anti-CD123 IgG1 humanised monoclonal antibody conjugated to N1-(2-(2,5-dioxo-2,5-dihydro-1H-pyrrol-1-yl)ethyl)-N6-((S)-1-(((S)-1-((3-((((S)-8-methoxy-6-oxo-11,12,12a,13-tetrahydro-6H-benzo[5,6][1,4]diazepino[1,2-a]indol-9-yl)oxy)methyl)-5-((((S)-8-methoxy-6-oxo-12a,13-dihydro-6Hbenzo[5,6][1,4]diazepino[1,2-a]indol-9-yl)oxy)methyl)phenyl)amino)-1-oxopropan-2-yl)amino)-1-oxopropan-2-yl)adipamide for: Treatment of blastic plasmacytoid dendritic cell neoplasm
Date of designation: 26/06/2020, Positive, Last updated: 23/09/2020 -
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Orphan designation: (+)-Epicatechin for: Treatment of Becker muscular dystrophy
Date of designation: 26/06/2020, Positive, Last updated: 23/09/2020 -
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Orphan designation: Sodium cromoglicate for: Treatment of idiopathic pulmonary fibrosis
Date of designation: 26/06/2020, Withdrawn, Last updated: 08/12/2020 -
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Orphan designation: Lys40(NODAGA-68Ga)NH2-exendin-4 for: Diagnosis of insulinoma
Date of designation: 26/06/2020, Positive, Last updated: 24/09/2020 -
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Orphan designation: Axicabtagene ciloleucel for: Treatment of marginal zone lymphoma
Date of designation: 26/06/2020, Positive, Last updated: 15/09/2021 -
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Orphan designation: Adeno-associated virus serotype HSC15 expressing human arylsulfatase A gene for: treatment of metachromatic leukodystrophy
Date of designation: 26/06/2020, Positive, Last updated: 21/10/2022 -
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Orphan designation: Refusal of orphan designation for the treatment of eumycetoma
Date of refusal of designation: 18/06/2020, Negative, Last updated: 19/11/2020 -
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Orphan designation: Viltolarsen for: Treatment of Duchenne muscular dystrophy (updated)
Date of designation: 04/06/2020, Positive, Last updated: 01/06/2023