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88 results
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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: Chimeric monoclonal antibody against GD2 for: Treatment of neuroblastoma
Date of designation: 08/11/2012, Positive, Last updated: 26/08/2021 -
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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: 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: idebenone for: Treatment of Leber's hereditary optic neuropathy
Date of designation: 16/02/2007, Positive, Last updated: 11/04/2016 -
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Orphan designation: idebenone for: Treatment of Duchenne muscular dystrophy
Date of designation: 20/03/2007, Positive, Last updated: 11/04/2016 -
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Orphan designation: Recombinant human tissue non-specific alkaline phosphatase - Fc - deca-aspartate fusion protein for: Treatment of hypophosphatasia
Date of designation: 04/12/2008, Positive, Last updated: 20/11/2019 -
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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: Metreleptin for: Treatment of familial partial lipodystrophy
Date of designation: 17/07/2012, Positive, Last updated: 02/04/2020 -
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Orphan designation: Metreleptin for: Treatment of Barraquer-Simons syndrome
Date of designation: 17/07/2012, Positive, Last updated: 02/04/2020 -
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Orphan designation: Metreleptin for: Treatment of Berardinelli-Seip syndrome
Date of designation: 17/07/2012, Positive, Last updated: 02/04/2020 -
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Orphan designation: Metreleptin for: Treatment of Lawrence syndrome
Date of designation: 17/07/2012, Positive, Last updated: 02/04/2020 -
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Direct healthcare professional communication (DHPC): Myalepta (metreleptin): inconsistencies in the package leaflet
Active substance: Metreleptin, DHPC type: Quality defect, Last updated: 18/06/2020 -
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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: tafamidis for: Treatment of senile systemic amyloidosis
Date of designation: 08/11/2012, Positive, Last updated: 25/02/2020 -
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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: Chenodeoxycholic acid for: Treatment of inborn errors in primary bile acid synthesis
Date of designation: 16/12/2014, Positive, Last updated: 29/06/2017 -
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Orphan designation: Recombinant human beta-glucuronidase (vestronidase alfa) for: Treatment of mucopolysaccharidosis type VII (Sly syndrome)
Date of designation: 21/03/2012, Positive, Last updated: 11/11/2020 -
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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: [Nle4, D-Phe7]-alfa-melanocyte stimulating hormone (afamelanotide) for: Treatment of erythropoietic protoporphyria
Date of designation: 08/05/2008, Positive, Last updated: 11/04/2016