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Orphan designation: autologous peripheral blood T cells CD4 and CD8 selected and CD3 and CD28 activated transduced with retroviral vector expressing anti CD19 CD28/CD3-zeta chimeric antigen receptor and cultured (brexucabtagene autoleucel) for: Treatment of mantle cell lymphoma
Date of designation: 13/11/2019, Positive, Last updated: 15/09/2021 -
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Orphan designation: Eculizumab for: Treatment of atypical haemolytic uraemic syndrome (aHUS)
Date of designation: 24/07/2009, Positive, Last updated: 20/11/2019 -
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Orphan designation: Eculizumab for: Treatment of neuromyelitis optica spectrum disorders
Date of designation: 05/08/2013, Positive, Last updated: 20/11/2019 -
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Orphan designation: Human coagulation factor X for: Treatment of hereditary factor X deficiency
Date of designation: 14/09/2007, Positive, Last updated: 26/03/2019 -
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Orphan designation: Recombinant human tripeptidyl-peptidase 1 for: Treatment of neuronal ceroid lipofuscinosis type 2
Date of designation: 12/03/2013, Positive, Last updated: 09/02/2016 -
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Orphan designation: Humanised monoclonal antibody against P-selectin (crizanlizumab) for: Treatment of sickle cell disease
Date of designation: 09/08/2012, Positive, Last updated: 24/11/2020 -
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Orphan designation: Ketoconazole for: Treatment of Cushing's syndrome
Date of designation: 23/04/2012, Positive, Last updated: 22/09/2020 -
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Orphan designation: Recombinant IgG degrading enzyme of Streptococcus pyogenes (imlifidase) for: Prevention of graft rejection following solid organ transplantation
Date of designation: 12/01/2017, Positive, Last updated: 28/08/2020 -
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Orphan designation: Phosphorothioate oligonucleotide targeted to apolipoprotein C-III (volanesorsen) for: Treatment of familial chylomicronaemia syndrome
Date of designation: 19/02/2014, Positive, Last updated: 20/05/2019 -
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Orphan designation: Chimeric anti-interleukin-6 monoclonal antibody (siltuximab) for: Treatment of Castleman's disease
Date of designation: 30/11/2007, Positive, Last updated: 26/08/2021 -
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Orphan designation: (1R,2S) 6-bromo-alpha-[2-(dimethylamino)ethyl]-2-methoxy-alpha-(1-naphthyl)-beta-phenyl-3-quinolineethanol bedaquiline for: Treatment of tuberculosis
Date of designation: 26/08/2005, Positive, Last updated: 01/04/2014 -
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Orphan designation: Adeno-associated viral vector serotype 9 containing the human SMN gene (onasemnogene abeparvovec) for: Treatment of spinal muscular atrophy
Date of designation: 19/06/2015, Positive, Last updated: 10/12/2020 -
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Orphan designation: velaglucerase alfa for: Treatment of Gaucher disease
Date of designation: 06/06/2010, Positive, Last updated: 24/09/2021 -
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Orphan designation: Recombinant human monoclonal IgG1 antibody for fibroblast growth factor 23 (burosumab) for: Treatment of X-linked hypophosphataemia
Date of designation: 15/10/2014, Positive, Last updated: 22/10/2018 -
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Orphan designation: Ex-vivo-expanded autologous human corneal epithelium-containing stem cells for: Corneal lesions, with associated corneal (limbal) stem cell deficiency, due to ocular burns
Date of designation: 07/11/2008, Positive, Last updated: 23/06/2020 -
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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 designation: 06/04/2017, Positive, Last updated: 30/05/2018 -
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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 designation: 09/10/2015, Positive, Last updated: 20/09/2021 -
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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 designation: 24/01/2013, Positive, Last updated: 03/06/2019 -
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Orphan designation: ciclosporin for: Treatment of vernal keratoconjunctivitis
Date of designation: 06/04/2006, Positive, Last updated: 12/07/2018 -
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Orphan designation: Antisense oligonucleotide targeted to the SMN2 gene (nusinersen) for: Treatment of 5q spinal muscular atrophy
Date of designation: 02/04/2012, Positive, Last updated: 16/10/2018 -
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Orphan designation: budesonide for: Treatment of eosinophilic oesophagitis
Date of designation: 05/08/2013, Positive, Last updated: 18/01/2018 -
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Orphan designation: (S)-2-nitro-6-(4-trifluoromethoxy)benzyloxy)-6,7-dihydro-5H-imidazo[2,1-b] [1,3] oxazine (pretomanid) for: Treatment of tuberculosis
Date of designation: 29/11/2007, Positive, Last updated: 09/12/2020 -
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Orphan designation: 3-[5-(2-Fluoro-phenyl)-[1,2,4]oxadiazole-3-yl]-benzoic acid (ataluren) for: Treatment of Duchenne muscular dystrophy
Date of designation: 27/05/2005, Positive, Last updated: 10/03/2015 -
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Orphan designation: Daratumumab
Date of designation: 17/07/2013, Positive, Last updated: 02/10/2018 -
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Orphan designation: Humanised monoclonal antibody targeting B-cell maturation antigen conjugated with maleimidocaproyl monomethyl auristatin F for: Treatment of multiple myeloma
Date of designation: 16/10/2017, Positive, Last updated: 17/09/2020