2184 results
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Orphan designation: Tazemetostat for: Treatment of diffuse large B-cell lymphoma (updated)
Date of first decision: 21/03/2018, Positive, Last updated: 25/06/2019 -
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Orphan designation: Tazemetostat for: Treatment of follicular lymphoma (updated)
Date of first decision: 21/03/2018, Positive, Last updated: 25/06/2019 -
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Orphan designation: Tazemetostat for: Treatment of malignant mesothelioma (updated)
Date of first decision: 21/03/2018, Positive, Last updated: 25/06/2019 -
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Orphan designation: Recombinant human anti-interferon gamma monoclonal antibody (emapalumab) for: Treatment of haemophagocytic lymphohistiocytosis (updated)
Date of first decision: 09/06/2010, Positive, Last updated: 19/06/2019 -
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Orphan designation: Autologous mesenchymal stromal cells on a decellularised tracheal scaffold from a cadaveric donor for: Treatment of tracheal stenosis (updated)
Date of first decision: 29/08/2016, Positive, Last updated: 19/06/2019 -
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Orphan designation: Recombinant adeno-associated viral vector containing human acid alfa-glucosidase-gene for: Treatment of glycogen storage disease type II (Pompe's disease) (updated)
Date of first decision: 04/07/2012, Positive, Last updated: 18/06/2019 -
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Orphan designation: Deferiprone for: Treatment of sickle cell disease (updated)
Date of first decision: 23/02/2011, Positive, Last updated: 18/06/2019 -
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Orphan designation: Pomalidomide for: Treatment of multiple myeloma (updated)
Date of first decision: 08/10/2009, Positive, Last updated: 18/06/2019 -
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Orphan designation: 4-[2-(6-Methylpyridin-2-yl)-5,6-dihydro-4H-pyrrolo[1,2-b]pyrazol-3-yl]-quinoline-6-carboxamide monohydrate for: Treatment of glioma (updated)
Date of first decision: 26/04/2013, Positive, Last updated: 17/06/2019 -
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Orphan designation: 4-[2-(6-Methylpyridin-2-yl)-5,6-dihydro-4H-pyrrolo[1,2-b]pyrazol-3-yl]-quinoline-6-carboxamide monohydrate for: Treatment of hepatocellular carcinoma (updated)
Date of first decision: 12/03/2013, Withdrawn, Last updated: 17/06/2019 -
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Orphan designation: Human/murine chimeric monoclonal antibody against endoglin (carotuximab) for: Treatment of soft tissue sarcoma (updated)
Date of first decision: 28/04/2016, Withdrawn, Last updated: 17/06/2019 -
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Orphan designation: Eculizumab for: Treatment of neuromyelitis optica spectrum disorders (updated)
Date of first decision: 05/08/2013, Positive, Last updated: 14/06/2019 -
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Orphan designation: Recombinant human hepatocarcinoma-intestine-pancreas, pancreatic associated protein for: Treatment of acute liver failure (updated)
Date of first decision: 11/02/2009, Positive, Last updated: 07/06/2019 -
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Orphan designation: adeno-associated viral vector serotype hu68 containing the human SMN1 gene for: Treatment of spinal muscular atrophy (updated)
Date of first decision: 24/08/2018, Positive, Last updated: 05/06/2019 -
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Orphan designation: 3-pentylbenzeneacetic acid sodium salt for: Treatment of Alström syndrome (updated)
Date of first decision: 12/01/2017, Positive, Last updated: 05/06/2019 -
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Orphan designation: 3-pentylbenzeneacetic acid sodium salt for: Treatment of idiopathic pulmonary fibrosis (updated)
Date of first decision: 09/10/2015, Positive, Last updated: 05/06/2019 -
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Orphan designation: 1-(2-hydroxyethyl)-8-{[5-(4-methylpiperazin-1-yl)-2-(trifluoromethoxy) phenyl]amino}-4,5-dihydro-1H-pyrazolo[4,3-h]quinazoline-3-carboxamide fumarate salt for: Treatment of acute myeloid leukaemia (updated)
Date of first decision: 24/08/2018, Positive, Last updated: 05/06/2019 -
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Orphan designation: Fenfluramine hydrochloride for: Treatment of Lennox-Gastaut syndrome (updated)
Date of first decision: 27/02/2017, Positive, Last updated: 03/06/2019 -
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Orphan designation: Fenfluramine hydrochloride for: Treatment of Dravet syndrome (updated)
Date of first decision: 16/01/2014, Positive, Last updated: 03/06/2019 -
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Orphan designation: Autologous CD34+ haematopoietic stem cells transduced with lentiviral vector encoding the human betaA-T87Q-globin gene for: Treatment of beta thalassaemia intermedia and major (updated)
Date of first decision: 24/01/2013, Positive, Last updated: 03/06/2019 -
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Orphan designation: Pegylated recombinant phenylalanine ammonia lyase (pegvaliase) for: Treatment of hyperphenylalaninaemia (updated)
Date of first decision: 28/01/2010, Positive, Last updated: 29/05/2019 -
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Orphan designation: avapritinib for: Treatment of mastocytosis
Date of first decision: 26/10/2018, Positive, Last updated: 27/05/2019 -
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Orphan designation: Human plasma-derived alpha-1 proteinase inhibitor for: Treatment of graft-versus-host disease
Date of first decision: 19/03/2015, Positive, Last updated: 27/05/2019 -
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Orphan designation: Recombinant protein consisting of modified human growth hormone releasing hormone and the translocation and endopeptidase domains of botulinum toxin serotype D for: Treatment of acromegaly
Date of first decision: 11/01/2012, Withdrawn, Last updated: 23/05/2019 -
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Orphan designation: PEGylated recombinant factor VIII (turoctocog alfa pegol) for: Treatment of haemophilia A
Date of first decision: 26/04/2012, Withdrawn, Last updated: 23/05/2019