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1758 results
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Orphan designation: Carboxypeptidase G2 (glucarpidase) for: Adjunctive treatment in patients at risk of methotrexate toxicity (updated)
Date of designation: 03/02/2003, Positive, Last updated: 20/01/2022 -
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Orphan designation: Denileukin diftitox for: Treatment of cutaneous T-cell lymphoma (updated)
Date of designation: 11/12/2001, Positive, Last updated: 19/01/2022 -
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Orphan designation: chimeric fusion protein of recombinant human alpha-N-acetylglucosaminidase and human insulin-like growth factor 2 (tralesinidase alfa) for: Treatment of mucopolysaccharidosis type IIIB (updated)
Date of designation: 15/01/2015, Positive, Last updated: 14/01/2022 -
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Orphan designation: Resiquimod for: Treatment of cutaneous T-cell lymphoma (updated)
Date of designation: 28/04/2016, Positive, Last updated: 13/01/2022 -
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Orphan designation: Treprostinil diethanolamine for: Treatment of pulmonary arterial hypertension and chronic thromboembolic pulmonary hypertension (updated)
Date of designation: 26/08/2005, Positive, Last updated: 13/01/2022 -
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Orphan designation: artesunate for: Treatment of malaria (updated)
Date of designation: 28/02/2020, Positive, Last updated: 13/01/2022 -
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Orphan designation: Sparsentan for: Treatment of primary IgA nephropathy (updated)
Date of designation: 19/10/2020, Positive, Last updated: 12/01/2022 -
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Orphan designation: 4'-[(2-butyl-4-oxo-1,3-diazaspiro[4.4]non-1-en-3-yl)methyl]-N-(4,5-dimethyl-3-isoxazolyl)-2'-(ethoxymethyl)-[1,1'-biphenyl]-2-sulfonamide for: Treatment of focal segmental glomerulosclerosis (updated)
Date of designation: 11/11/2015, Positive, Last updated: 12/01/2022 -
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Orphan designation: Human plasminogen for: Treatment of plasminogen deficiency (updated)
Date of designation: 28/07/2015, Positive, Last updated: 06/01/2022 -
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Orphan designation: 2-((4S)-6-(4-chlorophenyl)-1-methyl-4H-benzo[C]isoxazolo[4,5-e]azepin-4-yl)acetamide monohydrate for: Treatment of myelofibrosis (updated)
Date of designation: 28/02/2020, Positive, Last updated: 05/01/2022 -
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Orphan designation: Sodium phenylbutyrate, Tauroursodeoxycholic acid for: Treatment of amyotrophic lateral sclerosis (updated)
Date of designation: 04/06/2020, Positive, Last updated: 05/01/2022 -
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Orphan designation: erlotinib for: Treatment of patients with Olmsted syndrome (updated)
Date of designation: 09/12/2020, Positive, Last updated: 05/01/2022 -
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Orphan designation: Sirolimus for: Treatment of perivascular epithelioid cell tumours
Date of designation: 19/07/2021, Positive, Last updated: 21/12/2021 -
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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: Tislelizumab for: Treatment of oesophageal cancer
Date of designation: 13/11/2020, Positive, Last updated: 14/12/2021 -
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Orphan designation: 2-Hydroxypropyl-β-cyclodextrin for: Treatment of Niemann-Pick's disease, type C
Date of designation: 26/04/2013, Positive, Last updated: 14/12/2021 -
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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: (4R,5R)-1-[[4-[[4-[3,3-dibutyl-7-(dimethylamino)-2,3,4,5- tetrahydro-4-hydroxy-1,1-dioxido-1-benzothiepin-5-yl]phenoxy]methyl]phenyl]methyl]-4-aza-1-azoniabicyclo[2.2.2]octane chloride (maralixibat chloride) for: Treatment of progressive familial intrahepatic cholestasis
Date of designation: 16/01/2014, Positive, Last updated: 03/12/2021 -
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Orphan designation: Glycosylation independent lysosomal targeting (GILT)-tagged recombinant human acid alpha glucosidase for: Treatment of glycogen storage disease type II (Pompe's disease)
Date of designation: 27/10/2011, Positive, Last updated: 30/11/2021 -
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Orphan designation: 3-(3-(3,5-Dimethyl-1H-pyrazol-4-yl)propoxy)-4-fluorobenzoic acid for: Treatment of ATTR amyloidosis
Date of designation: 19/11/2018, Positive, Last updated: 30/11/2021 -
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Orphan designation: Sirolimus for: Treatment of tuberous sclerosis
Date of designation: 23/08/2017, Positive, Last updated: 30/11/2021 -
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Orphan designation: CD34+ haematopoietic stem and progenitor cells with CD3+ T-cells for: Treatment in solid organ transplantation
Date of designation: 24/08/2018, Positive, Last updated: 30/11/2021 -
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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: Humanised Fc engineered monoclonal antibody against CD19 (tafasitamab) for: Treatment of chronic lymphocytic leukaemia / small lymphocytic lymphoma
Date of designation: 04/07/2014, Positive, Last updated: 18/11/2021 -
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Orphan designation: poly(oxy-1,2-ethanediyl), alpha-(carboxymethyl)-omega-methoxy-, amide with arginase 1 [cobalt cofactor] (synthetic human) (1:10), trimer (pegzilarginase) for: Treatment of hyperargininaemia
Date of designation: 14/07/2016, Positive, Last updated: 11/11/2021