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2577 results
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Orphan designation: avapritinib for: Treatment of mastocytosis (updated)
Date of designation: 26/10/2018, Positive, Last updated: 18/05/2022 -
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Orphan designation: N-Hydroxy-4-(3-methyl-2-(S)-phenyl-butyrylamino) benzamide for: Treatment of schwannoma (updated)
Date of designation: 06/06/2012, Positive, Last updated: 18/05/2022 -
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Orphan designation: N-Hydroxy-4-(3-methyl-2-(S)-phenyl-butyrylamino) benzamide for: Treatment of neurofibromatosis type 2 (updated)
Date of designation: 26/04/2012, Positive, Last updated: 18/05/2022 -
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Orphan designation: N-Hydroxy-4-(3-methyl-2-(S)-phenyl-butyrylamino) benzamide for: Treatment of meningioma (updated)
Date of designation: 06/06/2012, Positive, Last updated: 18/05/2022 -
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Orphan designation: Autologous CD34+ cells transduced with a lentiviral vector encoding glucosylceramidase beta for: Treatment of Gaucher disease (updated)
Date of designation: 21/08/2020, Positive, Last updated: 18/05/2022 -
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Orphan designation: Autologous CD34+ cells transduced with a lentiviral RNA vector that results in integrated cDNA encoding for functional cystinosin for: Treatment of cystinosis (updated)
Date of designation: 19/02/2021, Positive, Last updated: 18/05/2022 -
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Orphan designation: synthetic human hepcidin for: Treatment of sickle cell disease (updated)
Date of designation: 18/11/2016, Withdrawn, Last updated: 18/05/2022 -
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Orphan designation: Synthetic hepcidin for: Treatment of beta thalassaemia intermedia and major (updated)
Date of designation: 09/10/2015, Withdrawn, Last updated: 18/05/2022 -
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Orphan designation: 6-{[(1R,2S)-2-aminocyclohexyl]amino}-7-fluoro-4-(1-methyl-1H-pyrazol-4-yl)-1,2-dihydro-3H-pyrrolo[3,4-c]pyridin-3-one monocitrate (also known as mivavotinib) for: Treatment of acute myeloid leukaemia (updated)
Date of designation: 22/02/2018, Positive, Last updated: 16/05/2022 -
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Orphan designation: pentosan polysulfate sodium for: Treatment of interstitial cystitis (updated)
Date of designation: 30/05/2016, Positive, Last updated: 16/05/2022 -
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Orphan designation: 1-[(3S)-3-{4-amino-3-[(3,5-dimethoxyphenyl)ethynyl]-1H-pyrazolo[3,4-d]pyrimidin-1-yl}pyrrolidin-1-yl]-2-propen-1-one (futibatinib) for: Treatment of biliary tract cancer (updated)
Date of designation: 01/04/2019, Positive, Last updated: 10/05/2022 -
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Orphan designation: Obeticholic acid for: Treatment of primary sclerosing cholangitis (updated)
Date of designation: 16/03/2014, Withdrawn, Last updated: 10/05/2022 -
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Orphan designation: N-(2-amino-phenyl)-4-[(4-pyridin-3-yl-pyrimidin-2-ylamino)-methyl] benzamide (mocetinostat) for: Treatment of Hodgkin's lymphoma (updated)
Date of designation: 14/09/2007, Withdrawn, Last updated: 10/05/2022 -
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Orphan designation: Mannitolum for: Treatment of cystic fibrosis (updated)
Date of designation: 07/11/2005, Expired, Last updated: 05/05/2022 -
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Orphan designation: Mercaptopurine for: Treatment of acute lymphoblastic leukaemia (updated)
Date of designation: 30/04/2009, Expired, Last updated: 05/05/2022 -
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Orphan designation: N- (2-Amino-phenyl)-4-[(4-pyridin-3-yl-pyrimidin-2-ylamino)-methyl] benzamide (mocetinostat) for: Treatment of acute myeloid leukaemia (updated)
Date of designation: 31/01/2008, Withdrawn, Last updated: 04/05/2022 -
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Orphan designation: Lisocabtagene maraleucel for: Treatment of primary mediastinal large B-cell lymphoma (updated)
Date of designation: 19/11/2018, Withdrawn, Last updated: 04/05/2022 -
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Orphan designation: Autologous CD4+ and CD8+ T cells expressing a CD19-specific chimeric antigen receptor (lisocabtagene maraleucel) for: Treatment of follicular lymphoma (updated)
Date of designation: 25/05/2018, Withdrawn, Last updated: 04/05/2022 -
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Orphan designation: Autologous CD4+ and CD8+ T cells expressing a CD19-specific chimeric antigen receptor (lisocabtagene maraleucel) for: Treatment of diffuse large B-cell lymphoma (updated)
Date of designation: 17/07/2017, Withdrawn, Last updated: 04/05/2022 -
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Orphan designation: Human culture expanded autologous mesenchymal stromal cells for: Treatment of amyotrophic lateral sclerosis (updated)
Date of designation: 24/04/2019, Positive, Last updated: 03/05/2022 -
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Orphan designation: Adeno-associated virus vector serotype 9 encoding the human GRN gene for: Treatment of frontotemporal dementia (updated)
Date of designation: 10/12/2021, Positive, Last updated: 03/05/2022 -
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Orphan designation: Unesbulin for: Treatment of soft tissue sarcoma (updated)
Date of designation: 10/12/2021, Positive, Last updated: 03/05/2022 -
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Orphan designation: Autologous CD34+ cells transduced ex vivo with a lentiviral vector containing a modified gamma-globin gene for: Treatment of sickle cell disease (updated)
Date of designation: 13/11/2020, Positive, Last updated: 26/04/2022 -
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Orphan designation: sotatercept for: Treatment of pulmonary arterial hypertension (updated)
Date of designation: 09/12/2020, Positive, Last updated: 26/04/2022 -
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Orphan designation: recombinant human interleukin-3 truncated diphtheria toxin fusion protein for: Treatment of acute myeloid leukaemia (updated)
Date of designation: 09/10/2015, Positive, Last updated: 25/04/2022