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Orphan designation: Autologous CD34+ cells transfected with retroviral vector containing adenosine deaminase gene for: Treatment of severe combined immunodeficiency (SCID) due to adenosine deaminase (ADA) deficiency
Date of designation: 26/08/2005, Positive, Last updated: 14/07/2022 -
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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 follicular lymphoma
Date of designation: 11/11/2015, Positive, Last updated: 12/07/2022 -
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Orphan designation: (2R,3S)-2-(4-cyclopentylaminophenyl)-1-(2-fluoro-6-methylbenzoyl)piperidine-3-carboxylic acid(4-methyl-3-trifluoromethylphenyl)amNide for: Treatment of microscopic polyangiitis (avacopan)
Date of designation: 19/11/2014, Positive, Last updated: 04/07/2022 -
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Orphan designation: (2R,3S)-2-(4-cyclopentylaminophenyl)-1-(2-fluoro-6-methylbenzoyl)piperidine-3-carboxylic acid(4-methyl-3-trifluoromethylphenyl)amide (avacopan) for: Treatment of granulomatosis with polyangiitis
Date of designation: 19/11/2014, Positive, Last updated: 04/07/2022 -
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Orphan designation: Setmelanotide for: Treatment of leptin receptor deficiency
Date of designation: 19/11/2018, Positive, Last updated: 01/07/2022 -
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Orphan designation: Setmelanotide for: Treatment of pro-opiomelanocortin deficiency
Date of designation: 14/07/2016, Positive, Last updated: 01/07/2022 -
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Orphan designation: Mosunetuzumab for: Treatment of follicular lymphoma
Date of designation: 12/11/2021, Positive, Last updated: 23/06/2022 -
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Orphan designation: Autologous human T cells genetically modified ex-vivo with a lentiviral vector encoding a chimeric antigen receptor for B-cell maturation antigen for: Treatment of multiple myeloma
Date of designation: 28/02/2020, Positive, Last updated: 13/06/2022 -
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Orphan designation: polatuzumab vedotin for: Treatment of diffuse large B-cell lymphoma
Date of designation: 16/04/2018, Positive, Last updated: 08/06/2022 -
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Orphan designation: tisagenlecleucel for: Treatment of follicular lymphoma
Date of designation: 19/07/2021, Positive, Last updated: 19/05/2022 -
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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: 2-hydroxy-6-((2-(1-isopropyl-1H-pyrazol-5-yl)pyridin-3-yl)methoxy)benzaldehyde (voxelotor) for: Treatment of sickle cell disease
Date of designation: 18/11/2016, Positive, Last updated: 17/03/2022 -
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Orphan designation: Recombinant modified human growth hormone for: Treatment of growth hormone deficiency
Date of designation: 24/01/2013, Positive, Last updated: 03/03/2022 -
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Orphan designation: lonapegsomatropin for: Treatment of growth hormone deficiency
Date of designation: 17/10/2019, Positive, Last updated: 17/02/2022 -
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Orphan designation: [5-(5-chloro-1H-pyrrolo[2,3-b]pyridin-3-ylmethyl)-pyridin-2-yl]-(6-trifluoromethyl-pyridin-3-ylmethyl)-amine hydrochloride for: Treatment of tenosynovial giant cell tumour, localised and diffuse type
Date of designation: 19/03/2015, Withdrawn, Last updated: 02/02/2022 -
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Orphan designation: ivacaftor, tezacaftor for: Treatment of cystic fibrosis
Date of designation: 27/02/2017, Positive, Last updated: 25/01/2022 -
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Orphan designation: Ivacaftor, N-(1,3-dimethyl-1H-pyrazole-4-sulfonyl)-6-[3-(3,3,3-trifluoro-2,2-dimethylpropoxy)-1H-pyrazol-1-yl]-2-[(4S)-2,2,4-trimethylpyrrolidin-1-yl]pyridine-3-carboxamide, tezacaftor (ivacaftor / tezacaftor / elexacaftor) for: Treatment of cystic fibrosis
Date of designation: 14/12/2018, Positive, Last updated: 25/01/2022 -
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Orphan designation: Carboxypeptidase G2 (glucarpidase) for: Adjunctive treatment in patients at risk of methotrexate toxicity
Date of designation: 03/02/2003, Positive, Last updated: 20/01/2022 -
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Orphan designation: artesunate for: Treatment of malaria
Date of designation: 28/02/2020, Positive, Last updated: 13/01/2022 -
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Orphan designation: Poly(oxy-1,2-ethanediyl),.alpha.-hydro-.omega.-hydroxy-,15,15'-diester with N-acetyl-L-isoleucyl-L-cysteinyl-L-valyl-1-methyl-L-tryptophyl-L-glutaminyl-L-.alpha.-aspartyl-L-tryptophylglycyl-L-alanyl-L-histidyl-L-arginyl-L-cysteinyl-L-threonyl-2-[2-(2-aminoethoxy)ethoxy]acetyl-N6-carboxy-L-lysinamide cyclic (2.fwdarw.12)-(disulfide); where two identical synthetic peptide domains are covalently linked at the ends of the polyethylene glycol chain for: Treatment of paroxysmal nocturnal haemoglobinuria
Date of designation: 22/05/2017, Positive, Last updated: 16/12/2021 -
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Orphan designation: Autologous T lymphocyte-enriched population of cells transduced with a lentiviral vector encoding a chimeric antigen receptor targeting human B cell maturation antigen with 4-1BB and CD3-zeta intracellular signalling domains (idecabtagene vicleucel) for: Treatment of multiple myeloma
Date of designation: 20/04/2017, Positive, Last updated: 03/12/2021 -
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Orphan designation: 1-[4-bromo-5-[1-ethyl-7-(methylamino)-2-oxo-1,2-dihydro-1,6-naphthyridin-3-yl]-2-fluorophenyl]-3-phenylurea for: Treatment of gastrointestinal stromal tumours
Date of designation: 08/11/2017, Positive, Last updated: 23/11/2021 -
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Orphan designation: N-tert-butyl-3-[(5-methyl-2-{[4-(2-pyrrolidin-1-ylethoxy)phenyl]amino}pyrimidin-4-yl)amino] benzenesulfonamide dihydrochloride monohydrate (fedratinib) for: Treatment of post-polycythaemia vera myelofibrosis
Date of designation: 26/11/2010, Positive, Last updated: 17/09/2021 -
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Orphan designation: N-tert-butyl-3-[(5-methyl-2-{[4-(2-pyrrolidin-1-ylethoxy)phenyl]amino}pyrimidin-4-yl)amino] benzenesulfonamide dihydrochloride monohydrate (fedratinib) for: Treatment of post-essential thrombocythaemia myelofibrosis
Date of designation: 26/11/2010, Positive, Last updated: 17/09/2021 -
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Orphan designation: N-tert-butyl-3-[(5-methyl-2-{[4-(2-pyrrolidin-1-ylethoxy)phenyl]amino}pyrimidin-4-yl)amino] benzenesulfonamide dihydrochloride monohydrate (fedratinib) for: Treatment of primary myelofibrosis
Date of designation: 01/10/2010, Positive, Last updated: 17/09/2021