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167 results
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Orphan designation: 1-{3-[3-(4-Chlorophenyl)propoxy]propyl}piperidine, hydrochloride for: Treatment of narcolepsy
Date of designation: 10/07/2007, Positive, Last updated: 20/04/2016 -
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Orphan designation: Gilteritinib for: Treatment of acute myeloid leukaemia
Date of designation: 17/01/2018, Positive, Last updated: 08/11/2019 -
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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: Pegylated recombinant phenylalanine ammonia lyase (pegvaliase) for: Treatment of hyperphenylalaninaemia
Date of designation: 28/01/2010, Positive, Last updated: 29/05/2019 -
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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: defibrotide for: Treatment of hepatic veno-occlusive disease
Date of designation: 29/07/2004, Positive, Last updated: 17/08/2016 -
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Direct healthcare professional communication (DHPC): Defitelio (defibrotide): Do not use for prophylaxis of veno-occlusive disease (VOD) after post-hematopoietic stem-cell transplantation (HSCT)
Active substance: defibrotide, DHPC type: Lack of effect, Safety signal, Last updated: 13/06/2022 -
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Orphan designation: carfilzomib for: Treatment of multiple myeloma
Date of designation: 03/06/2008, Positive, Last updated: 02/12/2015 -
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Orphan designation: 2,2'-{2-[(1R)-1-({[(2,5-dichlorobenzoyl)amino]acetyl}amino)-3-methylbutyl]-5-oxo-1,3,2-dioxaborolane-4,4-diyl}diacetic acid for: Treatment of multiple myeloma
Date of designation: 27/09/2011, Positive, Last updated: 26/08/2020 -
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Direct healthcare professional communication (DHPC): Ondexxya (andexanet alfa): Commercial anti-FXa activity assays are unsuitable for measuring anti-FXa activity following administration of andexanet alfa
Active substance: andexanet alfa, DHPC type: Safety signal, Last updated: 17/06/2020 -
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Direct healthcare professional communication (DHPC): Ondexxya (andexanet alfa): Avoid use of andexanet prior to heparinization
Active substance: andexanet alfa, DHPC type: Adverse event, Last updated: 04/11/2020 -
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Orphan designation: Brentuximab vedotin for: Treatment of cutaneous T-cell lymphoma
Date of designation: 11/01/2012, Positive, Last updated: 26/08/2020 -
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Orphan designation: monoclonal antibody against human CD30 covalently linked to the cytotoxin monomethylauristatin E (brentuximab vedotin) for: Treatment of Hodgkin's lymphoma
Date of designation: 15/01/2009, Positive, Last updated: 26/08/2020 -
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Orphan designation: monoclonal antibody against human CD30 covalently linked to the cytotoxin monomethylauristatin E for: Treatment of peripheral T-cell lymphoma
Date of designation: 21/09/2019, Positive, Last updated: 26/08/2020 -
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Orphan designation: Recombinant human lysosomal acid lipase for: Treatment of lysosomal acid lipase deficiency
Date of designation: 17/12/2010, Positive, Last updated: 20/11/2019 -
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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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Orphan designation: Risdiplam for: Treatment of spinal muscular atrophy
Date of designation: 26/02/2019, Positive, Last updated: 04/05/2021 -
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Orphan designation: (S)-2-nitro-6-(4-trifluoromethoxy)benzyloxy)-6,7-dihydro-5H-imidazo[2,1-b] [1,3] oxazine (pretomanid) for: Treatment of tuberculosis
Date of designation: 29/11/2007, Positive, Last updated: 09/12/2020 -
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Orphan designation: Daratumumab
Date of designation: 17/07/2013, Positive, Last updated: 02/10/2018 -
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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: Nanobody directed towards the human A1 domain of von Willebrand factor (caplacizumab) for: Treatment of thrombotic thrombocytopenic purpura
Date of designation: 30/04/2009, Positive, Last updated: 10/09/2018 -
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Orphan designation: Fenfluramine hydrochloride for: Treatment of Dravet syndrome
Date of designation: 18/12/2013, Positive, Last updated: 08/01/2021 -
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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