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Medicine
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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 mantle cell lymphoma (updated)
Date of designation: 09/10/2015, Withdrawn, Last updated: 12/01/2022 -
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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 acute lymphoblastic leukaemia (updated)
Date of designation: 11/11/2015, Withdrawn, Last updated: 12/01/2022 -
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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 chronic lymphocytic leukaemia / small lymphocytic lymphoma (updated)
Date of designation: 11/11/2015, Withdrawn, Last updated: 12/01/2022 -
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Orphan designation: Zanubrutinib for: Treatment of lymphoplasmacytic lymphoma
Date of designation: 29/05/2019, Withdrawn, Last updated: 15/12/2021 -
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Orphan designation: N-[4-[[(2-amino-3,4-dihydro-4-oxo-6-pteridinyl)methyl]amino]benzoyl]-D-gamma-glutamyl-(2S)-2-amino-beta-alanyl-L-alpha-aspartyl-L-cysteine (etarfolatide) for: Diagnosis of positive folate-receptor status in ovarian cancer
Date of designation: 10/09/2012, Withdrawn, Last updated: 07/12/2021 -
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Orphan designation: Autologous CD34+ haematopoietic stem cells transduced with lentiviral vector encoding the human beta A-T87Q-globin gene for: The treatment of sickle cell disease
Date of designation: 29/04/2014, Withdrawn, Last updated: 07/12/2021 -
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Orphan designation: Ibrutinib for: Treatment of follicular lymphoma
Date of designation: 16/01/2014, Withdrawn, Last updated: 23/11/2021 -
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Orphan designation: Ibrutinib for: Treatment of marginal zone lymphoma
Date of designation: 10/08/2015, Withdrawn, Last updated: 23/11/2021 -
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Orphan designation: Ibrutinib for: Treatment of graft-versus-host disease
Date of designation: 18/11/2016, Withdrawn, Last updated: 23/11/2021 -
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Orphan designation: Ibrutinib for: Treatment of diffuse large B-cell lymphoma
Date of designation: 13/11/2013, Withdrawn, Last updated: 23/11/2021 -
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Orphan designation: Polyphenyl(disodium 3-O-sulfo-beta-D-glucopyranuronate)-(1->3)-beta-D-galactopyranoside for: Treatment of anti-MAG neuropathy
Date of designation: 17/07/2017, Withdrawn, Last updated: 15/11/2021 -
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Orphan designation: Agammaglobulinaemia tyrosine kinase for: Treatment of pemphigus
Date of designation: 12/12/2017, Withdrawn, Last updated: 15/11/2021 -
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Orphan designation: (E)-2,4,6-trimethoxystyryl-3-carboxymethylamino-4-methoxybenzyl-sulfone sodium salt (rigosertib sodium) for: Treatment of myelodysplastic syndromes
Date of designation: 26/04/2012, Withdrawn, Last updated: 27/10/2021 -
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Orphan designation: Ibrutinib for: Treatment of lymphoplasmacytic lymphoma
Date of designation: 29/04/2014, Withdrawn, Last updated: 22/10/2021 -
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Orphan designation: 1-[(3R)-3-[4-amino-3-(4-phenoxyphenyl)-1H-pyrazolo[3,4 d]pyrimidin-1-yl]-1-piperidinyl]-2-propen-1-one (ibrutinib) for: Treatment of mantle cell lymphoma
Date of designation: 12/03/2013, Withdrawn, Last updated: 22/10/2021 -
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Orphan designation: 1-[(3R)-3-[4-amino-3-(4-phenoxyphenyl)-1H-pyrazolo[3,4 d]pyrimidin-1-yl]-1-piperidinyl]-2-propen-1-one (ibrutinib) for: Treatment of chronic lymphocytic leukaemia
Date of designation: 26/04/2012, Withdrawn, Last updated: 21/10/2021 -
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Orphan designation: Palovarotene for: Treatment of multiple osteochondromas
Date of designation: 27/06/2018, Withdrawn, Last updated: 19/10/2021 -
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Orphan designation: Seletalisib for: Treatment of activated phosphoinositide 3-kinase delta syndrome
Date of designation: 22/02/2018, Withdrawn, Last updated: 19/10/2021 -
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Orphan designation: Venglustat for: Treatment of autosomal dominant polycystic kidney disease
Date of designation: 14/12/2018, Withdrawn, Last updated: 12/10/2021 -
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Orphan designation: eptacog alfa (activated) for: Treatment of diffuse alveolar haemorrhage
Date of designation: 14/12/2005, Withdrawn, Last updated: 21/09/2021 -
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Orphan designation: drotrecogin alfa (activated) for: Treatment of acute respiratory distress syndrome (ARDS)
Date of designation: 22/09/2008, Withdrawn, Last updated: 21/09/2021 -
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Orphan designation: Recombinant human heparan-N-sulfatase for: Treatment of mucopolysaccharidosis, type IIIA (Sanfilippo A syndrome)
Date of designation: 07/11/2008, Withdrawn, Last updated: 02/09/2021 -
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Orphan designation: Chimeric IgG monoclonal antibody cG250 (girentuximab) for: Treatment of renal-cell carcinoma
Date of designation: 19/03/2002, Withdrawn, Last updated: 02/09/2021 -
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Orphan designation: Fosbretabulin tromethamine for: Treatment of ovarian cancer
Date of designation: 17/07/2013, Withdrawn, Last updated: 02/09/2021 -
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Orphan designation: Reduced oxidised N-acetyl heparin (roneparstat) for: Treatment of plasma cell myeloma
Date of designation: 21/05/2015, Withdrawn, Last updated: 02/09/2021