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259 results
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Orphan designation: Metreleptin for: Treatment of Barraquer-Simons syndrome
Date of first decision: 17/07/2012, Positive, Last updated: 02/04/2020 -
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Orphan designation: Metreleptin for: Treatment of Lawrence syndrome
Date of first decision: 17/07/2012, Positive, Last updated: 02/04/2020 -
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Orphan designation: Metreleptin for: Treatment of Berardinelli-Seip syndrome
Date of first decision: 17/07/2012, Positive, Last updated: 02/04/2020 -
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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 first decision: 29/08/2016, Positive, Last updated: 09/03/2020 -
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Orphan designation: N-methyl D-(2,3,4,5,6-pentahydroxy-hexyl)-ammonium; 2-(3,5-dichloro-phenyl)-benzoxazole-6-carboxylate (tafamidis) for: Treatment of familial amyloid polyneuropathy
Date of first decision: 28/08/2006, Positive, Last updated: 25/02/2020 -
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Orphan designation: tafamidis for: Treatment of senile systemic amyloidosis
Date of first decision: 08/11/2012, Positive, Last updated: 25/02/2020 -
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Orphan designation: polatuzumab vedotin for: Treatment of diffuse large B-cell lymphoma
Date of first decision: 16/04/2018, Positive, Last updated: 27/01/2020 -
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Orphan designation: Recombinant human tissue non-specific alkaline phosphatase - Fc - deca-aspartate fusion protein for: Treatment of hypophosphatasia
Date of first decision: 04/12/2008, Positive, Last updated: 20/11/2019 -
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Orphan designation: Recombinant human lysosomal acid lipase for: Treatment of lysosomal acid lipase deficiency
Date of first decision: 17/12/2010, Positive, Last updated: 20/11/2019 -
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Orphan designation: Gilteritinib for: Treatment of acute myeloid leukaemia
Date of first decision: 17/01/2018, Positive, Last updated: 08/11/2019 -
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Orphan designation: Glyceryl tri-(4-phenylbutyrate) for: Treatment of hyperargininaemia
Date of first decision: 10/06/2010, Positive, Last updated: 12/07/2019 -
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Orphan designation: Glyceryl tri-(4-phenylbutyrate) for: Treatment of argininosuccinic aciduria
Date of first decision: 10/06/2010, Positive, Last updated: 12/07/2019 -
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Orphan designation: Glyceryl tri-(4-phenylbutyrate) for: Ornithine translocase deficiency (hyperornithinaemia-hyperammonaemia homocitrullinuria (HHH) syndrom
Date of first decision: 10/06/2010, Positive, Last updated: 12/07/2019 -
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Orphan designation: Glyceryl tri-(4-phenylbutyrate) for: Treatment of citrullinaemia type 1
Date of first decision: 10/06/2010, Positive, Last updated: 12/07/2019 -
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Orphan designation: Glyceryl tri-(4-phenylbutyrate) for: Treatment of ornithine carbamoyltransferase deficiency
Date of first decision: 10/06/2010, Positive, Last updated: 12/07/2019 -
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Orphan designation: Pomalidomide for: Treatment of multiple myeloma
Date of first decision: 08/10/2009, Positive, Last updated: 18/06/2019 -
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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
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
Date of first decision: 28/01/2010, Positive, Last updated: 29/05/2019 -
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Orphan designation: (S)-1-(4-fluorophenyl)-1-(2-(4-(6-(1-methyl-1H-pyrazol-4-yl)pyrrolo[2,1-f][1,2,4]triazin-4-yl)piperazin-yl)pyrimidin-5-yl)ethan-1-amine for: Treatment of gastrointestinal stromal tumours
Date of first decision: 17/07/2017, Positive, Last updated: 22/05/2019 -
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Orphan designation: 1-Deoxygalactonojirimycin hydrochloride for: Treatment of Fabry disease
Date of first decision: 22/05/2006, Positive, Last updated: 21/05/2019 -
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Orphan designation: Phosphorothioate oligonucleotide targeted to apolipoprotein C-III (volanesorsen) for: Treatment of familial chylomicronaemia syndrome
Date of first decision: 19/02/2014, Positive, Last updated: 20/05/2019 -
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Orphan designation: Phosphorothioate oligonucleotide targeted to transthyretin (inotersen) for: Treatment of ATTR amyloidosis
Date of first decision: 26/03/2014, Positive, Last updated: 17/05/2019 -
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Orphan designation: Human coagulation factor X for: Treatment of hereditary factor X deficiency
Date of first decision: 14/09/2007, Positive, Last updated: 26/03/2019 -
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Orphan designation: Adenovirus-associated viral vector serotype 2 containing the human RPE65 gene (voretigene neparvovec) for: Treatment of retinitis pigmentosa
Date of first decision: 28/07/2015, Positive, Last updated: 04/03/2019 -
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Orphan designation: Adenovirus-associated viral vector serotype 2 containing the human RPE65 gene (voretigene neparvovec) for: Treatment of Leber's congenital amaurosis
Date of first decision: 02/04/2012, Positive, Last updated: 04/03/2019