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24 results
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Orphan designation: Synthetic double-stranded siRNA oligonucleotide directed against apolipoprotein C-III mRNA and covalently linked to a ligand containing three N-acetylgalactosamine residues for: Treatment of familial chylomicronaemia syndrome
Date of designation: 19/07/2021, Positive, Last updated: 12/11/2021 -
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Orphan designation: Saroglitazar magnesium for: Treatment of primary biliary cholangitis
Date of designation: 19/07/2021, Positive, Last updated: 12/11/2021 -
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Orphan designation: 1-(4-(6-chloropyridazin-3-yl)piperazin-1-yl)-2-(4-cyclopropyl-3-fluorophenyl)ethan-1-one for: Treatment of pantothenate kinase-associated neurodegeneration
Date of designation: 19/07/2021, Positive, Last updated: 05/09/2022 -
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Orphan designation: 1-(4-(6-chloropyridazin-3-yl)piperazin-1-yl)-2-(4-cyclopropyl-3-fluorophenyl)ethan-1-one for: Treatment of propionic acidaemia
Date of designation: 19/07/2021, Positive, Last updated: 05/09/2022 -
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Orphan designation: Itolizumab for: Treatment of graft-versus-host disease
Date of designation: 19/07/2021, Positive, Last updated: 12/11/2021 -
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Orphan designation: Eftansomatropin alfa for: Treatment of growth hormone deficiency
Date of designation: 19/07/2021, Positive, Last updated: 12/11/2021 -
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Orphan designation: Lutetium (177Lu) omburtamab barzuxetan for: Treatment of medulloblastoma
Date of designation: 19/07/2021, Positive, Last updated: 12/11/2021 -
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Orphan designation: melatonin for: Treatment of non-traumatic spontaneous intracerebral haemorrhage
Date of designation: 19/07/2021, Positive, Last updated: 12/11/2021 -
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Orphan designation: Vodobatinib for: Treatment of chronic myeloid leukaemia
Date of designation: 19/07/2021, Positive, Last updated: 12/11/2021 -
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Orphan designation: Pridopidine hydrochloride for: Treatment of amyotrophic lateral sclerosis
Date of designation: 19/07/2021, Positive, Last updated: 17/02/2022 -
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Orphan designation: Adeno-associated viral vector serotype Anc80 containing the 3' portion of human OTOF gene, adeno-associated viral vector serotype Anc80 containing the 5' portion of human OTOF gene for: Treatment of otoferlin gene-mediated hearing loss
Date of designation: 19/07/2021, Positive, Last updated: 16/11/2021 -
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Orphan designation: Adeno-associated virus vector serotype 1 containing the human GRN gene for: Treatment of frontotemporal dementia
Date of designation: 19/07/2021, Positive, Last updated: 16/11/2021 -
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Orphan designation: recombinant human ectonucleotide pyrophosphatase/phosphodiesterase 1 fused to the Fc fragment of IgG1 for: Treatment of adenosine triphosphate binding cassette transporter protein subfamily C member 6 deficiency
Date of designation: 19/07/2021, Positive, Last updated: 16/11/2021 -
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Orphan designation: mRNA encoding the human glycogen debranching enzyme for: Treatment of glycogen storage disease type III
Date of designation: 19/07/2021, Positive, Last updated: 16/11/2021 -
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Orphan designation: adeno-associated viral vector serotype 9 containing the human SLC13A5 gene for: Treatment of SLC13A5-epileptic encephalopathy deficiencies
Date of designation: 19/07/2021, Positive, Last updated: 16/11/2021 -
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Orphan designation: autologous CD34+ hematopoietic stem and progenitor cells genetically modified with the lentiviral vector encoding for the human palmitoyl-protein thioesterase 1 gene for: Treatment of neuronal ceroid lipofuscinosis
Date of designation: 19/07/2021, Positive, Last updated: 16/11/2021 -
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Orphan designation: 3,5-diiodothyropropionic acid for: Treatment of Allan-Herndon-Dudley syndrome
Date of designation: 19/07/2021, Positive, Last updated: 16/11/2021 -
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Orphan designation: tisagenlecleucel for: Treatment of follicular lymphoma
Date of designation: 19/07/2021, Positive, Last updated: 19/05/2022 -
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Orphan designation: Zanidatamab for: Treatment of biliary tract cancer
Date of designation: 19/07/2021, Positive, Last updated: 27/06/2023 -
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Orphan designation: Sirolimus for: Treatment of perivascular epithelioid cell tumours
Date of designation: 19/07/2021, Positive, Last updated: 21/12/2021 -
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Orphan designation: Infigratinib for: Treatment of achondroplasia
Date of designation: 19/07/2021, Positive, Last updated: 16/11/2021 -
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Orphan designation: humanised IgG2k Fc-modified bispecific monoclonal antibody against CD3 and BCMA for: Treatment of multiple myeloma
Date of designation: 19/07/2021, Positive, Last updated: 16/11/2021 -
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Orphan designation: 2-Methoxy-5-[(1Z)-2-(3,4,5-trimethoxyphenyl)ethenyl]-phenol (fosbretabulin tromethamine) for: Treatment of anaplastic thyroid cancer
Date of designation: 21/07/2021, Withdrawn, Last updated: 21/07/2021 -
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Orphan designation: Allogeneic anti-Epstein Barr virus cytotoxic T lymphocytes expanded ex vivo for: Treatment of post-transplant lymphoproliferative disorder
Date of designation: 21/07/2021, Withdrawn, Last updated: 21/07/2021