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Medicine
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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: 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: 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: 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 -
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Orphan designation: (4R,5R)-1-[[4-[[4-[3,3-dibutyl-7-(dimethylamino)-2,3,4,5- tetrahydro-4-hydroxy-1,1-dioxido-1-benzothiepin-5-yl]phenoxy]methyl]phenyl]methyl]-4-aza-1-azoniabicyclo[2.2.2]octane chloride (maralixibat chloride) for: Treatment of Alagille syndrome
Date of designation: 18/12/2013, Positive, Last updated: 14/12/2022 -
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Orphan designation: 3-[4-(1H-imidazol-1-ylmethyl)phenyl]-5-(2-methylpropyl)thiophene-2-[(N-butyloxylcarbamate)-sulphonamide] sodium salt for: Treatment of idiopathic pulmonary fibrosis
Date of designation: 14/07/2016, Positive, Last updated: 08/12/2022 -
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Orphan designation: mitapivat sulfate for: Treatment of pyruvate kinase deficiency
Date of designation: 22/04/2020, Positive, Last updated: 07/12/2022 -
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Orphan designation: Lentiviral vector encoding human coagulation factor IX for: Treatment of haemophilia B
Date of designation: 26/02/2019, Positive, Last updated: 02/12/2022 -
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Orphan designation: Autologous peripheral blood T cells CD4 and CD8 selected and CD3 and CD28 activated transduced with retroviral vector expressing anti-CD19 CD28/CD3-zeta chimeric antigen receptor and cultured (brexucabtagene autoleucel) for: Treatment of acute lymphoblastic leukaemia
Date of designation: 19/10/2020, Positive, Last updated: 02/12/2022 -
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Orphan designation: 1-(3-{4-[3,4-difluoro-2-(trifluoromethyl)phenyl]piperidine-1-carbonyl}-1H,4H,5H,6H,7H-pyrazolo[3,4- c]pyridin-6-yl)ethan-1-one (Tinlarebant) for: Treatment of Stargardt's disease
Date of designation: 25/05/2018, Positive, Last updated: 01/12/2022 -
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Orphan designation: A combination of H-Lys-Lys-Gly-Pro-Arg-Cys(SH)-Leu-Thr-Arg-Tyr-Tyr-Ser-Ser-Phe-Val-Asn-Met-Glu-Gly-Lys-Lys-OH and H-Lys-Lys-Gly-Asp-Asn-Ile-Met-Val-Thr-Phe-Arg-Asn-Gln-Ala-Ser-Arg-Pro-Tyr-Gly-Lys-Lys-OH for: Treatment of haemophilia A
Date of designation: 19/11/2014, Positive, Last updated: 29/11/2022