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179 results
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Orphan designation: Synthetic double-stranded siRNA oligonucleotide directed against delta-aminolevulinic acid synthase 1 mRNA, covalently linked to a ligand containing three N-acetylgalactosamine residues (givosiran) for: Treatment of acute hepatic porphyria
Date of designation: 29/08/2016, Positive, Last updated: 09/03/2020 -
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Orphan designation: Humanised Fc engineered monoclonal antibody against CD19 (tafasitamab) for: Treatment of diffuse large B-cell lymphoma
Date of designation: 15/01/2015, Positive, Last updated: 08/09/2021 -
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Orphan designation: Glasdegib maleate for: Treatment of acute myeloid leukaemia
Date of designation: 16/10/2017, Positive, Last updated: 09/01/2018 -
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Direct healthcare professional communication (DHPC): Picato (ingenol mebutate): Suspension of the marketing authorisation due to risk of skin malignancy
Active substance: Ingenol mebutate, DHPC type: Interim measures, Referral - Article 20 procedure, Last updated: 14/02/2020 -
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Orphan designation: Somapacitan for: Treatment of growth hormone deficiency
Date of designation: 24/08/2018, Positive, Last updated: 15/04/2021 -
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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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Direct healthcare professional communication (DHPC): Strimvelis® (autologous CD34+ enriched cell fraction that contains CD34+ cells transduced with retroviral vector that encodes for the human adenosine deaminase [ADA] cDNA sequence): first case of lymphoid T cell leukaemia after insertional oncogenesis
Active substance: autologous CD34+ enriched cell fraction that contains CD34+ cells transduced with retroviral vector that encodes for the human adenosine deaminase (ADA) cDNA sequence from human haematopoietic stem/progenitor (CD34+) cells, DHPC type: Adverse event, Last updated: 22/03/2021 -
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Orphan designation: Recombinant human acid sphingomyelinase (also known as olipudase alfa) for: Treatment of Niemann-Pick disease
Date of designation: 24/06/2022, Positive, Last updated: 27/07/2022 -
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Orphan designation: Recombinant human tripeptidyl-peptidase 1 for: Treatment of neuronal ceroid lipofuscinosis type 2
Date of designation: 12/03/2013, Positive, Last updated: 09/02/2016 -
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Orphan designation: Adenovirus-associated viral vector serotype 2 containing the human RPE65 gene (voretigene neparvovec) for: Treatment of retinitis pigmentosa
Date of designation: 28/07/2015, Positive, Last updated: 04/03/2019 -
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Orphan designation: Adenovirus-associated viral vector serotype 2 containing the human RPE65 gene (voretigene neparvovec) for: Treatment of Leber's congenital amaurosis
Date of designation: 02/04/2012, Positive, Last updated: 04/03/2019 -
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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 CD34+ cells transfected with lentiviral vector containing the human arylsulfatase A cDNA for: treatment of metachromatic leukodystrophy
Date of designation: 13/04/2007, Positive, Last updated: 14/07/2022 -
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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: 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: (2S)-2-{[(2R)-2-[({[3,3-dibutyl-7-(methylthio)-1,1-dioxido-5-phenyl-2,3,4,5-tetrahydro-1,2,5-benzothiadiazepin-8-yl]oxy}acetyl)amino]-2-(4-hydroxyphenyl)acetyl]amino}butanoic acid for: Treatment of progressive familial intrahepatic cholestasis
Date of designation: 17/07/2012, Positive, Last updated: 28/07/2021 -
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Orphan designation: 1-Deoxygalactonojirimycin hydrochloride for: Treatment of Fabry disease
Date of designation: 22/05/2006, Positive, Last updated: 21/05/2019 -
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Orphan designation: Humanised monoclonal antibody targeting B-cell maturation antigen conjugated with maleimidocaproyl monomethyl auristatin F for: Treatment of multiple myeloma
Date of designation: 16/10/2017, Positive, Last updated: 17/09/2020 -
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Orphan designation: Synthetic double-stranded siRNA oligonucleotide directed against hydroxyacid oxidase 1 mRNA and covalently linked to a ligand containing three N-acetylgalactosamine residues (lumasiran) for: Treatment of primary hyperoxaluria
Date of designation: 21/03/2016, Positive, Last updated: 25/11/2020 -
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Orphan designation: (R)-2-Methyl-6-nitro-2-{4-[4-(4-trifluoromethoxyphenoxy)piperidin-1-yl]phenoxymethyl}-2,3-dihydroimidazo[2,1-b]oxazole (delamanid) for: Treatment of tuberculosis
Date of designation: 02/02/2008, Positive, Last updated: 18/06/2014 -
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Orphan designation: Benzamide, 3-(2-imidazo[1,2-b]pyridazin-3-ylethynyl)-4-methyl-N-[4-[(4-methyl-1-piperazinyl)methyl]-3-(trifluoromethyl)phenyl] (ponatinib) for: Treatment of chronic myeloid leukaemia
Date of designation: 02/02/2010, Positive, Last updated: 23/03/2015 -
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Orphan designation: Benzamide, 3-(2-imidazo[1,2-b]pyridazin-3-ylethynyl)-4-methyl-N-[4-[(4-methyl-1-piperazinyl)methyl]-3-(trifluoromethyl)phenyl] (ponatinib) for: Treatment of acute lymphoblastic leukaemia
Date of designation: 02/02/2010, Positive, Last updated: 23/03/2015 -
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Orphan designation: idebenone for: Treatment of Leber's hereditary optic neuropathy
Date of designation: 16/02/2007, Positive, Last updated: 11/04/2016 -
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Orphan designation: [Nle4, D-Phe7]-alfa-melanocyte stimulating hormone (afamelanotide) for: Treatment of erythropoietic protoporphyria
Date of designation: 08/05/2008, Positive, Last updated: 11/04/2016 -
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Orphan designation: Autologous T cells transduced with lentiviral vector containing a chimeric antigen receptor directed against CD19 (tisagenlecleucel) for: Treatment of diffuse large B-cell lymphoma
Date of designation: 14/10/2016, Positive, Last updated: 13/06/2018