2216 results
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Orphan designation: drotrecogin alfa (activated) for: Treatment of acute respiratory distress syndrome (ARDS) (updated)
Date of first decision: 22/09/2008, Positive, Last updated: 14/11/2019 -
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Orphan designation: eptacog alfa (activated) for: Treatment of diffuse alveolar haemorrhage (updated)
Date of first decision: 14/12/2005, Positive, Last updated: 14/11/2019 -
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Orphan designation: Gilteritinib for: Treatment of acute myeloid leukaemia (updated)
Date of first decision: 17/01/2018, Positive, Last updated: 08/11/2019 -
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Orphan designation: (S)-N-(5-((R)-2-(2,5-difluorophenyl)pyrrolidin-1-yl)pyrazolo[1,5-a]pyrimidin-3-yl)-3-hydroxypyrrolidine-1-carboxamide hydrogen sulfate (larotrectinib) for: Treatment of soft tissue sarcoma (updated)
Date of first decision: 11/01/2016, Withdrawn, Last updated: 24/10/2019 -
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Orphan designation: Larotrectinib for: Treatment of salivary gland cancer (updated)
Date of first decision: 21/03/2018, Withdrawn, Last updated: 24/10/2019 -
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Orphan designation: Larotrectinib for: Treatment of glioma (updated)
Date of first decision: 19/11/2018, Withdrawn, Last updated: 24/10/2019 -
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Orphan designation: Larotrectinib for: Treatment of papillary thyroid cancer (updated)
Date of first decision: 19/11/2018, Withdrawn, Last updated: 24/10/2019 -
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Orphan designation: Recombinant human monoclonal antibody to human Nogo-A protein of the IgG4/kappa class (atinumab) for: Treatment of spinal cord injury
Date of first decision: 19/01/2009, Withdrawn, Last updated: 16/10/2019 -
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Orphan designation: Aztreonam lysinate (inhalation use) for: Treatment of Gram-negative bacterial lung infection in cystic fibrosis
Date of first decision: 21/06/2004, Expired, Last updated: 16/10/2019 -
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Orphan designation: Recombinant human soluble Fc-gamma receptor II b for: Treatment of idiopathic thrombocytopenic purpura
Date of first decision: 02/08/2007, Withdrawn, Last updated: 16/10/2019 -
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Orphan designation: pentetrazol for: Treatment of idiopathic hypersomnia
Date of first decision: 11/11/2015, Positive, Last updated: 16/10/2019 -
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Orphan designation: Recombinant human acid ceramidase for: Treatment of Farber disease
Date of first decision: 19/02/2014, Positive, Last updated: 16/10/2019 -
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Orphan designation: Recombinant human acid ceramidase for: Treatment of cystic fibrosis
Date of first decision: 10/08/2015, Positive, Last updated: 16/10/2019 -
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Orphan designation: Ibudilast for: Treatment of amyotrophic lateral sclerosis
Date of first decision: 12/12/2016, Positive, Last updated: 16/10/2019 -
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Orphan designation: Recombinant human monoclonal IgG1 antibody against programmed death ligand-1 (avelumab) for: Treatment of Merkel cell carcinoma
Date of first decision: 14/12/2015, Withdrawn, Last updated: 15/10/2019 -
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Orphan designation: avelumab for: Treatment of gastric cancer
Date of first decision: 12/12/2016, Withdrawn, Last updated: 15/10/2019 -
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Orphan designation: Humanised recombinant monoclonal antibody against epidermal growth factor receptor conjugated to maleimidocaproyl monomethylauristatin F(depatuxizumab mafodotin for: Treatment of glioma
Date of first decision: 29/07/2014, Withdrawn, Last updated: 15/10/2019 -
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Orphan designation: Glucopyranosyl lipid A for: Treatment of follicular lymphoma
Date of first decision: 16/10/2017, Withdrawn, Last updated: 15/10/2019 -
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Orphan designation: Glucopyranosyl lipid A stable emulsion and recombinant New York oesophageal squamous cell carcinoma-1 protein for: Treatment of soft tissue sarcoma
Date of first decision: 21/03/2016, Withdrawn, Last updated: 15/10/2019 -
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Orphan designation: Sindbis virus envelope pseudotyped lentiviral vector encoding New York oesophageal squamous cell carcinoma-1 protein for: Treatment of soft tissue sarcoma
Date of first decision: 21/03/2016, Withdrawn, Last updated: 15/10/2019 -
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Orphan designation: Dry extract from birch bark (DER 0.1-0.2:1), extraction solvent n-heptane 95% (V/V) for: Treatment of epidermolysis bullosa
Date of first decision: 23/02/2011, Positive, Last updated: 11/10/2019 -
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Orphan designation: Cannabidiol for: Treatment of Lennox-Gastaut syndrome
Date of first decision: 20/03/2017, Positive, Last updated: 04/10/2019 -
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Orphan designation: Cannabidiol for: Treatment of Dravet syndrome
Date of first decision: 15/10/2014, Positive, Last updated: 04/10/2019 -
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Orphan designation: S[+] apomorphine for: Treatment of amyotrophic lateral sclerosis
Date of first decision: 09/02/2012, Positive, Last updated: 03/10/2019 -
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Orphan designation: Adeno-associated viral vector serotype 9 containing the human SMN gene for: Treatment of spinal muscular atrophy
Date of first decision: 19/06/2015, Positive, Last updated: 03/10/2019