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2479 results
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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Date of designation: 31/01/2008, Withdrawn, Last updated: 04/05/2022 -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Date of designation: 19/11/2018, Withdrawn, Last updated: 04/05/2022 -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Date of designation: 25/05/2018, Withdrawn, Last updated: 04/05/2022 -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Date of designation: 17/07/2017, Withdrawn, Last updated: 04/05/2022 -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
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
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
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
Date of designation: 19/02/2021, Positive, Last updated: 25/04/2022 -
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Orphan designation: Tebentafusp for: Treatment of uveal melanoma
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
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 -
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Orphan designation: 26 base synthetic single-stranded fully phosphorothioated 2'-O-methyl-RNA and DNA mixmer oligonucleotide-based compound for: Treatment of Dravet syndrome
Date of designation: 27/02/2017, Positive, Last updated: 11/04/2022 -
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Orphan designation: [Gly2]-recombinant human glucagon-like peptide for: Treatment of short bowel syndrome
Date of designation: 12/12/2001, Positive, Last updated: 08/04/2022 -
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Orphan designation: (5R,5aR,8aR,9S)-9-[[4,6-O-[(R)-Ethylidene]-β-D-glucopyranosyl]-oxy]-5-(4-({[(2,2-dimethyl-1,3-dioxolan-4-yl)methoxy]carbonyl}oxy)-3,5-dimethoxyphenyl)-5,8,8a,9-tetrahydroisobenzofuro[5,6-f][1,3]benzodioxol-6(5aH)-one (etoposide toniribate) for: Treatment of biliary tract cancer
Date of designation: 04/06/2014, Positive, Last updated: 08/04/2022 -
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Orphan designation: Loncastuximab tesirine for: Treatment of diffuse large B-cell lymphoma
Date of designation: 20/08/2021, Positive, Last updated: 08/04/2022 -
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Orphan designation: Allogeneic donor-derived ex-vivo expanded T lymphocytes transduced with a retroviral vector containing inducible caspase 9 and truncated CD19 for: Treatment in haematopoietic stem cell transplantation
Date of designation: 27/06/2016, Withdrawn, Last updated: 07/04/2022 -
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Orphan designation: Rimiducid for: Treatment of graft-versus-host disease
Date of designation: 30/05/2016, Withdrawn, Last updated: 07/04/2022 -
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Orphan designation: Molgramostim for: Treatment of acute respiratory distress syndrome (ARDS)
Date of designation: 27/06/2016, Positive, Last updated: 06/04/2022