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24 results
Medicine
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Orphan designation: Eculizumab for: Treatment of neuromyelitis optica spectrum disorders
Date of first decision: 05/08/2013, Positive, Last updated: 04/09/2019 -
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Orphan designation: Autologous CD34+ haematopoietic stem cells transduced with lentiviral vector encoding the human betaA-T87Q-globin gene for: Treatment of beta thalassaemia intermedia and major
Date of first decision: 24/01/2013, Positive, Last updated: 03/06/2019 -
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Orphan designation: Phosphorothioate oligonucleotide targeted to transthyretin (inotersen) for: Treatment of ATTR amyloidosis
Date of first decision: 26/03/2014, Positive, Last updated: 17/05/2019 -
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Orphan designation: Human coagulation factor X for: Treatment of hereditary factor X deficiency
Date of first decision: 14/09/2007, Positive, Last updated: 26/03/2019 -
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Orphan designation: N-(2,4-di-tert-butyl-5-hydroxyphenyl)-1,4-dihydro-4-oxoquinoline-3-carboxamide (ivacaftor) for: Treatment of cystic fibrosis
Date of first decision: 08/07/2008, Positive, Last updated: 08/11/2018 -
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Orphan designation: Antisense oligonucleotide targeted to the SMN2 gene (nusinersen) for: Treatment of 5q spinal muscular atrophy
Date of first decision: 02/04/2012, Positive, Last updated: 16/10/2018 -
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Orphan designation: Daratumumab
Date of first decision: 17/07/2013, Positive, Last updated: 02/10/2018 -
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Orphan designation: ciclosporin for: Treatment of vernal keratoconjunctivitis
Date of first decision: 06/04/2006, Positive, Last updated: 12/07/2018 -
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Orphan designation: Synthetic double-stranded siRNA oligonucleotide directed against transthyretin mRNA (also known as patisiran) for: Treatment of transthyretin-mediated amyloidosis
Date of first decision: 06/04/2017, Positive, Last updated: 30/05/2018 -
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Orphan designation: budesonide for: Treatment of eosinophilic oesophagitis
Date of first decision: 05/08/2013, Positive, Last updated: 18/01/2018 -
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Orphan designation: Recombinant human IgG1 kappa light chain monoclonal antibody targeting plasma kallikrein (lanadelumab) for: Treatment of hereditary angioedema
Date of first decision: 09/10/2015, Positive, Last updated: 28/09/2017 -
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Orphan designation: Eculizumab for: Treatment myasthenia gravis
Date of first decision: 29/07/2014, Positive, Last updated: 26/09/2017 -
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Orphan designation: Olaratumab for: Treatment of soft tissue sarcoma
Date of first decision: 12/02/2015, Positive, -
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Orphan designation: Eculizumab for: Treatment of atypical haemolytic uraemic syndrome (aHUS)
Date of first decision: 24/07/2009, Positive, Last updated: 30/09/2016 -
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Orphan designation: Recombinant human tripeptidyl-peptidase 1 for: Treatment of neuronal ceroid lipofuscinosis type 2
Date of first decision: 12/03/2013, Positive, Last updated: 09/02/2016 -
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Orphan designation: Recombinant human nerve growth factor (cenegermin) for: Treatment of neurotrophic keratitis
Date of first decision: 14/12/2015, Positive, Last updated: 02/02/2016 -
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Orphan designation: Recombinant human lysosomal acid lipase for: Treatment of lysosomal acid lipase deficiency
Date of first decision: 17/12/2010, Positive, Last updated: 11/01/2016 -
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Orphan designation: carfilzomib for: Treatment of multiple myeloma
Date of first decision: 03/06/2008, Positive, Last updated: 02/12/2015 -
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Orphan designation: Benzamide, 3-(2-imidazo[1,2-b]pyridazin-3-ylethynyl)-4-methyl-N-[4-[(4-methyl-1-piperazinyl)methyl]-3-(trifluoromethyl)phenyl] (ponatinib) for: Treatment of chronic myeloid leukaemia
Date of first decision: 02/02/2010, Positive, Last updated: 23/03/2015 -
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Orphan designation: Benzamide, 3-(2-imidazo[1,2-b]pyridazin-3-ylethynyl)-4-methyl-N-[4-[(4-methyl-1-piperazinyl)methyl]-3-(trifluoromethyl)phenyl] (ponatinib) for: Treatment of acute lymphoblastic leukaemia
Date of first decision: 02/02/2010, Positive, Last updated: 23/03/2015 -
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Orphan designation: Ketoconazole for: Treatment of Cushing's syndrome
Date of first decision: 23/04/2012, Positive, Last updated: 12/03/2015 -
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Orphan designation: nintedanib for: Treatment of idiopathic pulmonary fibrosis
Date of first decision: 26/04/2013, Positive, Last updated: 12/03/2015 -
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Orphan designation: Chimeric anti-interleukin-6 monoclonal antibody (siltuximab) for: Treatment of Castleman's disease
Date of first decision: 30/11/2007, Positive, Last updated: 11/07/2014 -
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Orphan designation: velaglucerase alfa for: Treatment of Gaucher disease
Date of first decision: 06/06/2010, Positive, Last updated: 23/06/2010