Latest orphan designations and/or marketing authorisations
6 June 2012
Treatment of meningioma
N-hydroxy-4-(3-methyl-2-(S)-phenyl-butyrylamino) benzamide
Treatment of schwannoma
N-hydroxy-4-(3-methyl-2-(S)-phenyl-butyrylamino) benzamide
Treatment of Wiskott-Aldrich syndrome
Autologous CD34+ cells transfected with lentiviral vector containing the Wiskott-Aldrich syndrome protein gene
Treatment of cytomegalovirus disease in patients with impaired cell mediated immunity
Letermovir
Treatment of pancreatic cancer
Polyinosine-polycytidylic acid coupled with the polycationic polyethyleneimine
Treatment of ovarian cancer
1-(4-{4-amino-7-[1-(2-hydroxyethyl)-1H-pyrazol-4-yl] thieno [3,2-c]pyridin-3-yl}phenyl)-3-(3-fluorophenyl) urea
Treatment of glioma
Adenovirus-associated vector containing human Fas-c gene
Treatment of adrenoleukodystrophy
Autologous haematopoietic stem cells transduced with lentiviral vector Lenti-D encoding the human ABCD1 cDNA
Date
25 May 2012List
Treatment of soft tissue sarcomaYttrium (90Y)-DTPA-radiolabelled chimeric monoclonal antibody against frizzled homologue 10
Detailed information on European orphan drug designation applications is available on the EMA website
A full list of designated and authorised orphan drugs in Europe available at: ec.europa.eu
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