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212 results
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Orphan designation: Beclomethasone 17, 21-dipropionate (oral use) (beclometasone dipropionate) for: Treatment of intestinal graft-versus-host disease
Date of first decision: 13/03/2002, Positive, Last updated: 08/06/2020 -
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Orphan designation: pasireotide for: Treatment of acromegaly
Date of first decision: 08/10/2009, Positive, Last updated: 13/05/2020 -
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Orphan designation: pasireotide for: Treatment of Cushing's disease
Date of first decision: 08/10/2009, Positive, Last updated: 13/05/2020 -
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Orphan designation: Treprostinil sodium for: Treatment of chronic thromboembolic pulmonary hypertension
Date of first decision: 08/02/2013, Positive, Last updated: 08/04/2020 -
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Orphan designation: Metreleptin for: Treatment of familial partial lipodystrophy
Date of first decision: 17/07/2012, Positive, Last updated: 02/04/2020 -
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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: Carboxypeptidase G2 for: Adjunctive treatment in patients at risk of methotrexate toxicity
Date of first decision: 03/02/2003, 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: Deferiprone for: Treatment of neurodegeneration with brain iron accumulation
Date of first decision: 27/06/2018, Positive, Last updated: 04/02/2020 -
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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: Ivosidenib for: Treatment of acute myeloid leukaemia
Date of first decision: 12/12/2016, Positive, Last updated: 04/12/2019 -
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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: Eculizumab for: Treatment myasthenia gravis
Date of first decision: 29/07/2014, Positive, Last updated: 20/11/2019 -
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Orphan designation: Eculizumab for: Treatment of neuromyelitis optica spectrum disorders
Date of first decision: 05/08/2013, Positive, Last updated: 20/11/2019 -
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Orphan designation: Eculizumab for: Treatment of atypical haemolytic uraemic syndrome (aHUS)
Date of first decision: 24/07/2009, 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: 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: 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: Ornithine translocase deficiency (hyperornithinaemia-hyperammonaemia homocitrullinuria (HHH) syndrom
Date of first decision: 10/06/2010, Positive, Last updated: 12/07/2019