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2652 results
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Orphan designation: Fenfluramine hydrochloride for: Treatment of Lennox-Gastaut syndrome (updated)
Date of designation: 27/02/2017, Positive, Last updated: 07/02/2023 -
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Orphan designation: Berzosertib for: Treatment of small cell lung cancer (updated)
Date of designation: 18/07/2022, Withdrawn, Last updated: 07/02/2023 -
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Orphan designation: Rebastinib for: Treatment of ovarian cancer (updated)
Date of designation: 12/11/2021, Positive, Last updated: 07/02/2023 -
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Orphan designation: Dodecyl creatine ester, dodecyl creatine ester hydrochloride for: Treatment of creatine deficiency syndromes (updated)
Date of designation: 19/02/2021, Positive, Last updated: 06/02/2023 -
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Orphan designation: polyethylene glycol-modified human recombinant truncated cystathionine beta-synthase (pegtibatinase) for: Treatment of homocystinuria (updated)
Date of designation: 30/05/2016, Positive, Last updated: 06/02/2023 -
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Orphan designation: Volanesorsen sodium for: Treatment of familial partial lipodystrophy (updated)
Date of designation: 14/07/2016, Positive, Last updated: 02/02/2023 -
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Orphan designation: Phosphorothioate oligonucleotide targeted to apolipoprotein C-III (volanesorsen) for: Treatment of familial chylomicronaemia syndrome (updated)
Date of designation: 19/02/2014, Positive, Last updated: 02/02/2023 -
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Orphan designation: Allogeneic Epstein-Barr virus specific cytotoxic T lymphocytes (Tabelecleucel) for: Treatment of post-transplantation lymphoproliferative disorders (updated)
Date of designation: 21/03/2016, Positive, Last updated: 30/01/2023 -
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Orphan designation: Ex-vivo-expanded autologous keratinocytes transduced with retroviral vector containing the COL7A1 gene (prademagene zamikeracel) for: Treatment of epidermolysis bullosa (updated)
Date of designation: 27/02/2017, Positive, Last updated: 30/01/2023 -
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Orphan designation: Bardoxolone methyl for: Treatment of autosomal dominant polycystic kidney disease (updated)
Date of designation: 20/08/2021, Positive, Last updated: 30/01/2023 -
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Orphan designation: Omaveloxolone for: Treatment of Friedreich’s ataxia (updated)
Date of designation: 27/06/2018, Positive, Last updated: 30/01/2023 -
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Orphan designation: Bardoxolone methyl for: Treatment of Alport syndrome (updated)
Date of designation: 25/05/2018, Positive, Last updated: 30/01/2023 -
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Orphan designation: Rilonacept for: Treatment of idiopathic pericarditis (updated)
Date of designation: 06/01/2021, Positive, Last updated: 30/01/2023 -
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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: zanubrutinib for: Treatment of marginal zone lymphoma
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