37 results
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Orphan designation: Chimeric 2'-O-(2-methoxyethyl) modified oligonucleotide targeted to huntingtin RNA for: Treatment of Huntington’s disease
Date of designation: 19/03/2015, Positive, Last updated: 30/05/2018 -
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Orphan designation: 2'-O-(2-Methoxyethyl)-modified antisense oligonucleotide targeting exon 13 in the USH2A gene for: Treatment of retinitis pigmentosa
Date of designation: 22/02/2018, Positive, Last updated: 10/04/2018 -
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Orphan designation: Modified messenger ribonucleic acid encoding human argininosuccinate lyase enzyme encapsulated into lipid nanoparticles for: Treatment of argininosuccinic aciduria
Date of designation: 12/12/2017, Positive, Last updated: 23/01/2018 -
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Orphan designation: Adenoviral vector of serotype 5 modified to contain a chimeric sequence consisting of a minimal urokinase-type plasminogen activator receptor promoter preceded by three Notch-responsive elements, and coated with oligopeptide end-modified poly (beta-amino) esters for: Treatment of pancreatic cancer
Date of designation: 16/10/2017, Positive, Last updated: 09/01/2018 -
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Orphan designation: Genetically modified adeno-associated viral vector serotype 9 expressing shRNA as well as a codon-optimised shRNA-insensitive wildtype PABPN1 for: Treatment of oculopharyngeal muscular dystrophy
Date of designation: 12/01/2017, Positive, Last updated: 11/07/2017 -
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Orphan designation: Genetically modified Lactococcus lactis bacteria containing the human trefoil factor 1 gene for: Prevention of oral mucositis in head and neck cancer patients undergoing radiation therapy
Date of designation: 27/09/2011, Positive, Last updated: 29/03/2016 -
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Orphan designation: recombinant modified vaccinia virus Ankara expressing tuberculosis antigen 85A for: Prevention of tuberculosis disease in Bacille-Calmette-Guerin-vaccinated individuals
Date of designation: 28/10/2005, Withdrawn, Last updated: 19/01/2016 -
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Orphan designation: Skin equivalent graft genetically corrected with a COL7A1-encoding SIN retroviral vector for: Treatment of dystrophic epidermolysis bullosa
Date of designation: 30/04/2009, Positive, Last updated: 07/10/2014 -
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Orphan designation: Genetically modified human adenovirus encoding human PH20 hyaluronidase for: Treatment of pancreatic cancer
Date of designation: 21/06/2011, Positive, Last updated: 31/07/2013 -
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Orphan designation: Fluocinolone acetonide for: Treatment of non-infectious uveitis affecting the posterior segment of the eye
Date of designation: 07/03/2005, Positive, Last updated: 30/05/2007 -
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Public data from Article 57 database
Last updated: 30/06/2023 -
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Guidance documents
Last updated: 19/07/2022