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2317 results
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Orphan designation: Recombinant IgG degrading enzyme of Streptococcus pyogenes (imlifidase) for: Prevention of graft rejection following solid organ transplantation (updated)
Date of first decision: 12/01/2017, Positive, Last updated: 28/08/2020 -
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Orphan designation: Rilzabrutinib for: Treatment of immune thrombocytopenia (updated)
Date of first decision: 04/06/2020, Positive, Last updated: 27/08/2020 -
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Orphan designation: (16E)-14-methyl-20-oxa-5,7,14,26-tetraaza-tetracyclo[19.3.1.1(2,6).1(8,12)]heptacosa-1(25),2(26),3,5,8(27),9,11,16,21,23-decaene–citric acid for: Treatment of glioma (updated)
Date of first decision: 17/10/2019, Positive, Last updated: 27/08/2020 -
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Orphan designation: gallium citrate for: Treatment of cystic fibrosis (updated)
Date of first decision: 21/08/2019, Positive, Last updated: 27/08/2020 -
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Orphan designation: Autologous stromal vascular cell fraction from adipose tissue for: Treatment of systemic sclerosis (updated)
Date of first decision: 28/04/2016, Withdrawn, Last updated: 27/08/2020 -
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Orphan designation: Autologous adult live cultured osteoblasts for: Treatment of non-traumatic osteonecrosis (updated)
Date of first decision: 26/02/2019, Positive, Last updated: 27/08/2020 -
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Orphan designation: Agammaglobulinaemia tyrosine kinase for: Treatment of pemphigus (updated)
Date of first decision: 12/12/2017, Positive, Last updated: 27/08/2020 -
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Orphan designation: P-ethoxy growth factor receptor-bound protein 2 antisense oligonucleotide for: Treatment of acute myeloid leukaemia (updated)
Date of first decision: 14/10/2016, Positive, Last updated: 27/08/2020 -
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Orphan designation: Recombinant human monoclonal antibody to insulin receptor for: Treatment of congenital hyperinsulinism (updated)
Date of first decision: 14/07/2016, Positive, Last updated: 27/08/2020 -
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Orphan designation: Human monoclonal antibody targeting Staphylococcus aureus alpha-toxin (tosatoxumab) for: Treatment of pneumonia caused by Staphylococcus aureus (updated)
Date of first decision: 05/03/2012, Positive, Last updated: 27/08/2020 -
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Orphan designation: Multilamellar microvesicle comprising phosphatidylcholine, sphingomyelin, phosphatidylethanolamine, phosphatidylserine, phosphatidylinositol and cholesterol for: Treatment of cystic fibrosis (updated)
Date of first decision: 30/08/2011, Positive, Last updated: 27/08/2020 -
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Orphan designation: Synthetic double-stranded siRNA oligonucleotide directed against p53 mRNA (teprasiran) for: Prevention of delayed graft function after renal transplantation (updated)
Date of first decision: 06/06/2010, Positive, Last updated: 27/08/2020 -
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Orphan designation: Human monoclonal antibody against Pseudomonas aeruginosa serotype O11 (Panobacumab) for: Treatment of pneumonia caused by serotype O11 Pseudomonas aeruginosa (updated)
Date of first decision: 29/06/2006, Positive, Last updated: 27/08/2020 -
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Orphan designation: Pevonedistat for: Treatment of acute myeloid leukaemia (updated)
Date of first decision: 25/07/2019, Positive, Last updated: 26/08/2020 -
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Orphan designation: Pevonedistat for: Treatment of myelodysplastic syndromes (updated)
Date of first decision: 14/12/2018, Positive, Last updated: 26/08/2020 -
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Orphan designation: monoclonal antibody against human CD30 covalently linked to the cytotoxin monomethylauristatin E (brentuximab vedotin) for: Adjunctive treatment in haematopoietic cell transplantation (updated)
Date of first decision: 15/01/2009, Positive, Last updated: 26/08/2020 -
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Orphan designation: monoclonal antibody against human CD30 covalently linked to the cytotoxin monomethylauristatin E for: Treatment of peripheral T-cell lymphoma (updated)
Date of first decision: 21/09/2019, Positive, Last updated: 26/08/2020 -
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Orphan designation: Ixazomib for: Treatment of systemic light chain amyloidosis (updated)
Date of first decision: 08/11/2012, Positive, Last updated: 26/08/2020 -
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Orphan designation: Expanded human allogeneic mesenchymal adult stem cells extracted from adipose tissue (darvadstrocel) for: Treatment of anal fistula (updated)
Date of first decision: 08/10/2009, Positive, Last updated: 26/08/2020 -
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Orphan designation: Brentuximab vedotin for: Treatment of cutaneous T-cell lymphoma (updated)
Date of first decision: 11/01/2012, Positive, Last updated: 26/08/2020 -
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Orphan designation: Balipodect for: Adjunctive treatment in haematopoietic cell transplantation (updated)
Date of first decision: 14/04/2019, Positive, Last updated: 26/08/2020 -
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Orphan designation: 5-{(1R,2R)-2-[(cyclopropylmethyl)amino]cyclopropyl}-N-(tetrahydro-2H-pyran-4-yl)thiophene-3-carboxamide monohydrochloride for: Treatment of Kabuki syndrome (updated)
Date of first decision: 19/11/2018, Positive, Last updated: 26/08/2020 -
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Orphan designation: 2,2'-{2-[(1R)-1-({[(2,5-dichlorobenzoyl)amino]acetyl}amino)-3-methylbutyl]-5-oxo-1,3,2-dioxaborolane-4,4-diyl}diacetic acid for: Treatment of multiple myeloma (updated)
Date of first decision: 27/09/2011, Positive, Last updated: 26/08/2020 -
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Orphan designation: 6-{[(1R,2S)-2-aminocyclohexyl]amino}-7-fluoro-4-(1-methyl-1H-pyrazol-4-yl)-1,2-dihydro-3H-pyrrolo[3,4-c]pyridin-3-one monocitrate for: Treatment of acute myeloid leukaemia (updated)
Date of first decision: 22/02/2018, Positive, Last updated: 26/08/2020 -
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Orphan designation: 6'-(R)-methyl-5-O-(5-amino-5,6-dideoxy-α-L-talofuranosyl)-paromamine sulfate for: Treatment of mucopolysaccharidosis type I (updated)
Date of first decision: 22/09/2016, Positive, Last updated: 25/08/2020