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532 results
Medicine
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Orphan designation: cholic acid for: Treatment of inborn errors in primary bile acid synthesis responsive to treatment with cholic acid (updated)
Date of first decision: 29/10/2009, Withdrawn, Last updated: 29/07/2020 -
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Orphan designation: nintedanib for: Treatment of idiopathic pulmonary fibrosis
Date of first decision: 26/04/2013, Withdrawn, Last updated: 09/06/2020 -
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Orphan designation: Autologous Epstein-Barr virus specific T cells derived from peripheral blood mononuclear cells, expanded ex vivo for: Treatment of post-transplantation lymphoproliferative disorders
Date of first decision: 14/07/2016, Withdrawn, Last updated: 25/05/2020 -
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Orphan designation: Autologous Epstein-Barr virus specific T cells derived from peripheral blood mononuclear cells, expanded ex vivo- for: Treatment of extranodal NK/T-cell lymphoma, nasal type
Date of first decision: 14/07/2016, Withdrawn, Last updated: 25/05/2020 -
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Orphan designation: Mertansine functionalised gold nanoconjugate for: Treatment of hepatocellular carcinoma
Date of first decision: 22/02/2018, Withdrawn, Last updated: 20/05/2020 -
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Orphan designation: Entolimod for: Treatment of acute radiation syndrome
Date of first decision: 11/01/2016, Withdrawn, Last updated: 07/05/2020 -
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Orphan designation: thiotepa for: Conditioning treatment prior to haematopoietic-progenitor-cell transplantation
Date of first decision: 29/01/2007, Withdrawn, Last updated: 20/04/2020 -
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Orphan designation: 2-(7-ethoxy-4-(3-fluorophenyl)-1-oxophthalazin-2(1H)-yl)-N-methyl-N-(2-methylbenzo[d]oxazol-6-yl) acetamide for: Treatment of cystic fibrosis
Date of first decision: 21/05/2015, Withdrawn, Last updated: 16/04/2020 -
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Orphan designation: nitisinone for: Treatment of alkaptonuria
Date of first decision: 13/03/2002, Withdrawn, Last updated: 01/04/2020 -
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Orphan designation: Humanised recombinant IgG4 anti-human tau antibody (tilavonemab) for: Treatment of progressive supranuclear palsy
Date of first decision: 28/04/2016, Withdrawn, Last updated: 01/04/2020 -
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Orphan designation: Human monoclonal IgG2 antibody against tissue factor pathway inhibitor for: Treatment of haemophilia A
Date of first decision: 22/02/2018, Withdrawn, Last updated: 26/03/2020 -
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Orphan designation: Yttrium (90Y)-DOTA-radiolabelled humanized monoclonal antibody against mucin 1 for: Treatment of pancreatic cancer
Date of first decision: 06/02/2009, Withdrawn, Last updated: 06/03/2020 -
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Orphan designation: Veltuzumab for: Treatment of chronic lymphocytic leukaemia
Date of first decision: 29/01/2010, Withdrawn, Last updated: 06/03/2020 -
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Orphan designation: 3,4-diaminopyridine phosphate (amifampridine) for: Treatment of Lambert-Eaton myasthenic syndrome
Date of first decision: 19/12/2002, Withdrawn, Last updated: 04/03/2020 -
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Orphan designation: Milatuzumab for: Treatment of chronic lymphocytic leukaemia
Date of first decision: 19/01/2009, Withdrawn, Last updated: 18/02/2020 -
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Orphan designation: Milatuzumab for: Treatment of multiple myeloma
Date of first decision: 19/01/2009, Withdrawn, Last updated: 18/02/2020 -
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Orphan designation: 505 amino acid protein, corresponding to amino acids 2-506 of the wild-type human histidyl-tRNA synthetase for: Treatment of limb-girdle muscular dystrophy
Date of first decision: 27/02/2017, Withdrawn, Last updated: 12/02/2020 -
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Orphan designation: 505 amino acid protein, corresponding to amino acids 2-506 of the wild type human histidyl-tRNA synthetase for: treatment of facioscapulohumeral muscular dystrophy
Date of first decision: 12/02/2015, Withdrawn, Last updated: 12/02/2020 -
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Orphan designation: Adenovirus-specific T-cells derived from allogeneic donor leukocytes, expanded ex vivo for: Treatment of adenovirus infection in allogeneic haematopoietic stem cell transplant recipients
Date of first decision: 16/01/2014, Withdrawn, Last updated: 12/02/2020 -
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Orphan designation: lenalidomide for: Treatment of follicular lymphoma
Date of first decision: 24/01/2013, Withdrawn, Last updated: 11/02/2020 -
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Orphan designation: Beraprost sodium for: Treatment of pulmonary arterial hypertension
Date of first decision: 10/07/2008, Withdrawn, Last updated: 11/02/2020 -
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Orphan designation: Herpes simplex 1 virus-thymidine kinase and truncated low affinity nerve growth factor receptor transfected donor lymphocytes for: Adjunctive treatment in haematopoietic cell transplantation
Date of first decision: 20/10/2003, Withdrawn, Last updated: 16/01/2020 -
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Orphan designation: Triheptanoin for: Treatment of glucose transporter type-1 deficiency syndrome
Date of first decision: 21/05/2015, Withdrawn, Last updated: 16/01/2020 -
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Orphan designation: 6-(2-hydroxy-2-methylpropoxy)-4-(6-(6-((6-methoxypyridin-3-yl)methyl)-3,6-diazabicyclo[3.1.1]heptan-3-yl)pyridin-3-yl)pyrazolo[1,5-a]pyridine-3-carbonitrile for: Treatment of medullary thyroid carcinoma
Date of first decision: 26/10/2018, Withdrawn, Last updated: 16/01/2020 -
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Orphan designation: Olaratumab for: Treatment of soft tissue sarcoma
Date of first decision: 12/02/2015, Withdrawn, Last updated: 15/01/2020