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132 results
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Orphan designation: synthetic double-stranded siRNA oligonucleotide targeted against transthyretin mRNA, with six phosphorothioate linkages in the backbone, and nine 2'-fluoro and thirty-five 2'-O-methyl nucleoside residues in the sequence, which is covalently linked via a phosphodiester group to a ligand containing three N-acetylgalactosamine residues (Vutrisiran) for: Treatment of transthyretin-mediated amyloidosis (ATTR amyloidosis)
Date of designation: 25/05/2018, Positive, Last updated: 12/10/2022 -
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Orphan designation: [Gly2]-recombinant human glucagon-like peptide for: Treatment of short bowel syndrome
Date of designation: 12/12/2001, Positive, Last updated: 12/10/2022 -
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Orphan designation: Recombinant human IgG1 kappa light chain monoclonal antibody targeting plasma kallikrein (lanadelumab) for: Treatment of hereditary angioedema
Date of designation: 09/10/2015, Positive, Last updated: 04/10/2022 -
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Orphan designation: Recombinant human parathyroid hormone for: Treatment of hypoparathyroidism
Date of designation: 16/01/2014, Positive, Last updated: 04/10/2022 -
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Orphan designation: Setmelanotide for: Treatment of Bardet-Biedl syndrome
Date of designation: 21/08/2019, Positive, Last updated: 30/09/2022 -
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Orphan designation: Adeno-associated viral vector serotype 5 containing a B-domain deleted variant of human coagulation factor VIII gene for: Treatment of haemophilia A
Date of designation: 21/03/2016, Positive, Last updated: 28/09/2022 -
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Orphan designation: Budesonide for: Treatment of primary IgA nephropathy
Date of designation: 18/11/2016, Positive, Last updated: 14/09/2022 -
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Orphan designation: Efgartigimod alfa for: Treatment of myasthenia gravis
Date of designation: 10/08/2022, Positive, Last updated: 12/09/2022 -
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Orphan designation: Asciminib for: Treatment of chronic myeloid leukaemia
Date of designation: 24/03/2020, Positive, Last updated: 07/09/2022 -
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Orphan designation: recombinant adeno-associated viral vector serotype 2 carrying the gene for the human aromatic L-amino acid decarboxylase protein, Eladocagene exuparvovec for: Treatment of aromatic L-amino acid decarboxylase deficiency
Date of designation: 18/11/2016, Positive, Last updated: 10/08/2022 -
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Orphan designation: Adeno-associated viral vector serotype 9 containing the human SMN gene (onasemnogene abeparvovec) for: Treatment of spinal muscular atrophy
Date of designation: 19/06/2015, Positive, Last updated: 25/07/2022 -
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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: 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: Tebentafusp for: Treatment of uveal melanoma
Date of designation: 19/02/2021, Positive, Last updated: 22/04/2022 -
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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