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Orphan designation: Melphalan flufenamide for: Treatment of plasma cell myeloma
Date of designation: 19/03/2015, Withdrawn, Last updated: 22/08/2022 -
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Orphan designation: autologous dendritic cells pulsed with killed ovarian cancer cells and matured by TLR3 ligand ex vivo for: Treatment of ovarian cancer
Date of designation: 16/04/2018, Withdrawn, Last updated: 17/08/2022 -
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Orphan designation: Recombinant truncated N-terminal fragment of human lens epithelium-derived growth factor for: Treatment of retinitis pigmentosa
Date of designation: 23/08/2017, Withdrawn, Last updated: 12/08/2022 -
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Orphan designation: allogeneic peripheral blood mononuclear cells incubated ex-vivo with 16, 16-dimethyl prostaglandin E2, dexamethasone for: Treatment in haematopoietic stem cell transplantation
Date of designation: 18/11/2016, Withdrawn, Last updated: 10/08/2022 -
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Orphan designation: recombinant adeno-associated viral vector serotype 2 carrying the gene for the human aromatic L-amino acid decarboxylase protein, Eladocagene exuparvovec for: Treatment of aromatic L-amino acid decarboxylase deficiency
Date of designation: 18/11/2016, Positive, Last updated: 10/08/2022 -
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Orphan designation: 2-Chloro-N6-(3-iodobenzyl)adenosine-5'-N-methyluronamide (namodenoson) for: Treatment of hepatocellular carcinoma
Date of designation: 09/10/2015, Positive, Last updated: 10/08/2022 -
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Orphan designation: Adenovirus associated viral vector serotype 4 containing the human RPE65 gene for: Treatment of retinitis pigmentosa
Date of designation: 14/11/2007, Positive, Last updated: 09/08/2022 -
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Orphan designation: Adenovirus associated viral vector serotype 4 containing the human RPE65 gene for: Treatment of Leber's congenital amaurosis
Date of designation: 22/10/2007, Positive, Last updated: 09/08/2022 -
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Orphan designation: adenovirus associated viral vector serotype 5 containing the human pde6β gene for: Treatment of retinitis pigmentosa
Date of designation: 19/06/2013, Positive, Last updated: 09/08/2022 -
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Orphan designation: Adeno-associated viral vector serotype 5 containing the human RLBP1 gene for: Treatment of retinitis pigmentosa
Date of designation: 14/10/2016, Positive, Last updated: 09/08/2022 -
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Orphan designation: Mazindol for: Treatment of narcolepsy
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
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)
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
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
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
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
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
Date of designation: 19/06/2015, Positive, Last updated: 25/07/2022 -
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Orphan designation: Teclistamab for: Treatment of multiple myeloma
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
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
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
Date of designation: 18/07/2022, Withdrawn, Last updated: 19/07/2022 -
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Orphan designation: Sirolimus for: Treatment of tuberous sclerosis
Date of designation: 20/06/2017, Positive, Last updated: 19/07/2022 -
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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
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
Date of designation: 06/06/2012, Positive, Last updated: 14/07/2022