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2577 results
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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Date of designation: 16/03/2014, Withdrawn, Last updated: 10/05/2022 -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Date of designation: 14/09/2007, Withdrawn, Last updated: 10/05/2022 -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Date of designation: 31/01/2008, Withdrawn, Last updated: 04/05/2022 -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Date of designation: 19/11/2018, Withdrawn, Last updated: 04/05/2022 -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 -
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Orphan designation: Autologous CD34+ hematopoietic stem and progenitor cells transfected with zinc finger nuclease mRNAs SB-mRENH1 and SB-mRENH2 for: Treatment of sickle cell disease (updated)
Date of designation: 19/02/2021, Positive, Last updated: 25/04/2022 -
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Orphan designation: Tebentafusp for: Treatment of uveal melanoma (updated)
Date of designation: 19/02/2021, Positive, Last updated: 22/04/2022 -
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Orphan designation: Entinostat for: Treatment of Hodgkin's lymphoma (updated)
Date of designation: 10/06/2010, Positive, Last updated: 19/04/2022 -
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Orphan designation: polyethylene glycol-modified human recombinant truncated cystathionine beta-synthase (pegtibatinase) for: Treatment of homocystinuria
Date of designation: 30/05/2016, Positive, Last updated: 13/04/2022 -
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Orphan designation: Mocravimod for: Treatment in haematopoietic stem cell transplantation
Date of designation: 15/10/2021, Positive, Last updated: 13/04/2022 -
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Orphan designation: Encapsulated human retinal pigment epithelial cell line transfected with plasmid vector expressing human ciliary neurotrophic factor for: Treatment of retinitis pigmentosa
Date of designation: 24/01/2013, Positive, Last updated: 12/04/2022 -
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Orphan designation: Encapsulated human retinal pigment epithelial cell line transfected with plasmid vector expressing human ciliary neurotropic factor for: Treatment of macular telangiectasia type 2
Date of designation: 08/11/2012, Positive, Last updated: 12/04/2022 -
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Orphan designation: Leniolisib for: Treatment of activated phosphoinositide 3-kinase delta syndrome
Date of designation: 19/10/2020, Positive, Last updated: 11/04/2022