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Orphan designation: Mazindol for: Treatment of narcolepsy (updated)
Date of designation: 09/10/2015, Positive, Last updated: 03/08/2022 -
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Orphan designation: Bilayer engineered collagen hydrogel-based skin graft composed of autologous keratinocytes and fibroblasts for: Treatment of partial deep dermal and full-thickness burns (updated)
Date of designation: 14/12/2015, Positive, Last updated: 03/08/2022 -
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Orphan designation: Recombinant human alpha-glucosidase conjugated with multiple copies of synthetic bismannose-6-phosphate-tetra-mannose glycan (avalglucosidase alfa) for: Treatment of glycogen storage disease type II (Pompe's disease) (updated)
Date of designation: 26/03/2014, Withdrawn, Last updated: 02/08/2022 -
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Orphan designation: Pegylated recombinant arginine deiminase (pegargiminase) for: Treatment of malignant mesothelioma (updated)
Date of designation: 15/01/2015, Positive, Last updated: 02/08/2022 -
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Orphan designation: (S)-ethyl 2-amino-3-(4-(2-amino-6((R)-1-(4-chloro-2-(3-methyl-1H-pyrazol-1-yl)phenyl)-2,2,2-trifluoroethoxy)pyrimidin-4-yl)phenyl)propanoate for: Treatment of carcinoid syndrome (updated)
Date of designation: 30/05/2016, Positive, Last updated: 28/07/2022 -
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Orphan designation: Recombinant human acid sphingomyelinase (also known as olipudase alfa) for: Treatment of Niemann-Pick disease (updated)
Date of designation: 24/06/2022, Positive, Last updated: 27/07/2022 -
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Orphan designation: Moxetumomab pasudotox for: Treatment of B-lymphoblastic leukaemia/lymphoma (updated)
Date of designation: 19/07/2022, Withdrawn, Last updated: 25/07/2022 -
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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 (updated)
Date of designation: 19/06/2015, Positive, Last updated: 25/07/2022 -
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Orphan designation: Teclistamab for: Treatment of multiple myeloma (updated)
Date of designation: 19/07/2022, Withdrawn, Last updated: 22/07/2022 -
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Orphan designation: arimoclomol citrate for: Treatment of inclusion body myositis (updated)
Date of designation: 18/07/2022, Withdrawn, Last updated: 19/07/2022 -
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Orphan designation: Arimoclomol for: Treatment of amyotrophic lateral sclerosis (updated)
Date of designation: 18/07/2022, Withdrawn, Last updated: 19/07/2022 -
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Orphan designation: Humanised anti-CD37 monoclonal antibody conjugated to maytansinoid DM1 (naratuximab emtansine) for: Treatment of diffuse large B-cell lymphoma (updated)
Date of designation: 18/07/2022, Withdrawn, Last updated: 19/07/2022 -
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Orphan designation: Sirolimus for: Treatment of tuberous sclerosis (updated)
Date of designation: 20/06/2017, Positive, Last updated: 19/07/2022 -
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Orphan designation: budesonide for: Treatment of primary IgA nephropathy (updated)
Date of designation: 18/11/2016, Positive, Last updated: 18/07/2022 -
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Orphan designation: Autologous CD34+ cells transduced with a lentiviral vector containing the human ADA gene for: Treatment of adenosine-deaminase-deficient severe combined immunodeficiency (updated)
Date of designation: 07/06/2013, Positive, Last updated: 14/07/2022 -
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Orphan designation: Autologous CD34+ cells transfected with lentiviral vector containing the Wiskott-Aldrich syndrome protein gene for: Treatment of Wiskott-Aldrich syndrome (updated)
Date of designation: 06/06/2012, Positive, Last updated: 14/07/2022 -
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Orphan designation: autologous CD34+ haematopoietic stem and progenitor cells genetically modified with the lentiviral vector IDUA LV, encoding for the alpha-L-iduronidase cDNA for: Treatment of mucopolysaccharidosis type I (updated)
Date of designation: 26/10/2018, Positive, Last updated: 14/07/2022 -
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Orphan designation: Autologous CD34+ cells transduced with lentiviral vector encoding the human beta globin gene for: Treatment of beta thalassaemia intermedia and major (updated)
Date of designation: 30/05/2016, Positive, Last updated: 14/07/2022 -
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Orphan designation: Autologous CD34+ cells transduced with a lentiviral vector containing the human SGSH gene for: Treatment of mucopolysaccharidosis, type IIIA (Sanfilippo A syndrome) (updated)
Date of designation: 10/06/2014, Positive, Last updated: 14/07/2022 -
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Orphan designation: Autologous haematopoietic cells genetically modified with a lentiviral vector containing the human gp91(phox) gene for: Treatment of X-linked chronic granulomatous disease (updated)
Date of designation: 09/02/2012, Positive, Last updated: 14/07/2022 -
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Orphan designation: Autologous CD34+ cells transfected with lentiviral vector containing the human arylsulfatase A cDNA for: treatment of metachromatic leukodystrophy (updated)
Date of designation: 13/04/2007, Positive, Last updated: 14/07/2022 -
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Orphan designation: Autologous CD34+ cells transfected with retroviral vector containing adenosine deaminase gene for: Treatment of severe combined immunodeficiency (SCID) due to adenosine deaminase (ADA) deficiency (updated)
Date of designation: 26/08/2005, Positive, Last updated: 14/07/2022 -
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Orphan designation: Replication-incompetent, non-integrating, herpes simplex virus 1 vector expressing the human transglutaminase-1 enzyme for: Treatment of autosomal recessive congenital ichthyosis (updated)
Date of designation: 13/11/2019, Positive, Last updated: 14/07/2022 -
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Orphan designation: genetically modified replication-incompetent herpes simplex virus-1 expressing collagen VII for: Treatment of epidermolysis bullosa (updated)
Date of designation: 16/04/2018, Positive, Last updated: 14/07/2022 -
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Orphan designation: antisense oligonucleotide targeting exon 73 in the COL7A1 gene for: Treatment of epidermolysis bullosa (updated)
Date of designation: 12/10/2017, Positive, Last updated: 14/07/2022