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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: 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: 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 -
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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: Osilodrostat for: Treatment of Cushing's syndrome
Date of designation: 15/10/2014, Positive, Last updated: 07/07/2020 -
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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: 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 Lawrence 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: Synthetic double-stranded siRNA oligonucleotide directed against delta-aminolevulinic acid synthase 1 mRNA, covalently linked to a ligand containing three N-acetylgalactosamine residues (givosiran) for: Treatment of acute hepatic porphyria
Date of designation: 29/08/2016, Positive, Last updated: 09/03/2020 -
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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 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: Recombinant human lysosomal acid lipase for: Treatment of lysosomal acid lipase deficiency
Date of designation: 17/12/2010, Positive, Last updated: 20/11/2019 -
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Orphan designation: Eculizumab for: Treatment myasthenia gravis
Date of designation: 29/07/2014, 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: Gilteritinib for: Treatment of acute myeloid leukaemia
Date of designation: 17/01/2018, Positive, Last updated: 08/11/2019 -
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Orphan designation: Lonafarnib for: Treatment of Hutchinson-Gilford progeria
Date of designation: 14/12/2018, Positive, Last updated: 01/08/2019 -
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Orphan designation: Pegylated recombinant phenylalanine ammonia lyase (pegvaliase) for: Treatment of hyperphenylalaninaemia
Date of designation: 28/01/2010, Positive, Last updated: 29/05/2019 -
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Orphan designation: (S)-1-(4-fluorophenyl)-1-(2-(4-(6-(1-methyl-1H-pyrazol-4-yl)pyrrolo[2,1-f][1,2,4]triazin-4-yl)piperazin-yl)pyrimidin-5-yl)ethan-1-amine for: Treatment of gastrointestinal stromal tumours
Date of designation: 17/07/2017, Positive, Last updated: 22/05/2019 -
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Orphan designation: 1-Deoxygalactonojirimycin hydrochloride for: Treatment of Fabry disease
Date of designation: 22/05/2006, Positive, Last updated: 21/05/2019 -
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Orphan designation: Phosphorothioate oligonucleotide targeted to transthyretin (inotersen) for: Treatment of ATTR amyloidosis
Date of designation: 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 designation: 14/09/2007, Positive, Last updated: 26/03/2019 -
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Orphan designation: Adenovirus-associated viral vector serotype 2 containing the human RPE65 gene (voretigene neparvovec) for: Treatment of retinitis pigmentosa
Date of designation: 28/07/2015, Positive, Last updated: 04/03/2019 -
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Orphan designation: Adenovirus-associated viral vector serotype 2 containing the human RPE65 gene (voretigene neparvovec) for: Treatment of Leber's congenital amaurosis
Date of designation: 02/04/2012, Positive, Last updated: 04/03/2019