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Orphan designation: idursulfase for: Treatment of mucopolysaccharidosis type II (Hunter's syndrome) (updated)
Date of designation: 06/01/2021, Withdrawn, Last updated: 27/01/2023 -
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Orphan designation: Loncastuximab tesirine for: Treatment of diffuse large B-cell lymphoma (updated)
Date of designation: 20/08/2021, Withdrawn, Last updated: 26/01/2023 -
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Orphan designation: propagermanium for: Treatment of focal segmental glomerulosclerosis (updated)
Date of designation: 19/11/2018, Positive, Last updated: 25/01/2023 -
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Orphan designation: Recombinant human mesencephalic astrocyte-derived neurotrophic factor for: Treatment of retinitis pigmentosa (updated)
Date of designation: 24/04/2015, Positive, Last updated: 24/01/2023 -
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Orphan designation: (S)-5-(1-(6-chloro-2-oxo-1,2-dihydroquinolin-3-yl)ethylamino-1-methyl-6-oxo-1,6-dihydropyridine-2-carbonitrile (olutasidenib) for: Treatment of acute myeloid leukaemia (updated)
Date of designation: 29/05/2019, Positive, Last updated: 23/01/2023 -
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Orphan designation: Ramiprilat for: Treatment of Stargardt's disease (updated)
Date of designation: 12/03/2013, Positive, Last updated: 17/01/2023 -
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Orphan designation: Herpes simplex virus 1 expressing the human CFTR gene for: Treatment of cystic fibrosis (updated)
Date of designation: 26/03/2021, Positive, Last updated: 17/01/2023 -
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Orphan designation: Replication-incompetent, non-integrating, herpes simplex virus 1 vector expressing the human transglutaminase-1 enzyme (Inetagugene geperpavec) for: Treatment of autosomal recessive congenital ichthyosis (updated)
Date of designation: 13/11/2019, Positive, Last updated: 13/01/2023 -
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Orphan designation: Allogeneic Epstein-Barr virus specific cytotoxic T lymphocytes for: Treatment of post-transplantation lymphoproliferative disorders (updated)
Date of designation: 21/03/2016, Positive, Last updated: 12/01/2023 -
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Orphan designation: zanubrutinib for: Treatment of marginal zone lymphoma (updated)
Date of designation: 20/05/2021, Withdrawn, Last updated: 05/01/2023 -
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Orphan designation: Pegylated adrenomedullin for: Treatment of acute respiratory distress syndrome (ARDS)
Date of designation: 27/07/2020, Positive, Last updated: 22/12/2022 -
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Orphan designation: Human allogeneic bone-marrow-derived osteoblastic-like cells for: Treatment of non-traumatic osteonecrosis
Date of designation: 05/08/2013, Positive, Last updated: 22/12/2022 -
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Orphan designation: Human allogeneic bone-marrow-derived osteoblastic cells for: Treatment of osteogenesis imperfecta
Date of designation: 10/08/2015, Positive, Last updated: 22/12/2022 -
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Orphan designation: regorafenib for: Treatment of glioma
Date of designation: 28/06/2019, Withdrawn, Last updated: 20/12/2022 -
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Orphan designation: Allogeneic retinal pigment epithelial cells genetically modified with a non-viral vector to express human alpha-L-iduronidase for: Treatment of mucopolysaccharidosis type I
Date of designation: 15/10/2021, Withdrawn, Last updated: 20/12/2022 -
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Orphan designation: bevacizumab for: Treatment of hereditary haemorrhagic telangiectasia
Date of designation: 16/12/2014, Positive, Last updated: 19/12/2022 -
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Orphan designation: 4-[131I] Iodo-L-phenylalanine for: Treatment of glioma
Date of designation: 11/04/2006, Positive, Last updated: 19/12/2022 -
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Orphan designation: Autologous T cells transduced with retroviral vector encoding an anti-CD19 CD28/CD3 zeta chimeric antigen receptor (axicabtagene ciloleucel) for: Treatment of diffuse large B-cell lymphoma
Date of designation: 16/12/2014, Positive, Last updated: 16/12/2022 -
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Orphan designation: pentosan polysulfate sodium for: Treatment of mucopolysaccharidosis VI (Maroteaux-Lamy syndrome)
Date of designation: 21/08/2020, Positive, Last updated: 15/12/2022 -
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Orphan designation: pentosan polysulfate sodium for: Treatment of mucopolysaccharidosis type I
Date of designation: 19/11/2014, Positive, Last updated: 15/12/2022 -
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Orphan designation: edaravone for: Treatment of amyotrophic lateral sclerosis
Date of designation: 16/12/2014, Positive, Last updated: 15/12/2022 -
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Orphan designation: Concizumab for: Treatment of haemophilia B
Date of designation: 12/10/2017, Withdrawn, Last updated: 14/12/2022 -
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Orphan designation: Humanised monoclonal IgG4 antibody against tissue-factor-pathway inhibitor (concizumab) for: Treatment of haemophilia A
Date of designation: 10/10/2012, Withdrawn, Last updated: 14/12/2022 -
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Orphan designation: Alisporivir for: Treatment of collagen VI-related myopathies
Date of designation: 16/03/2022, Positive, Last updated: 14/12/2022 -
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Orphan designation: besilesomab for: Treatment in haematopoietic stem cell transplantation
Date of designation: 17/10/2019, Positive, Last updated: 14/12/2022