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85 results
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Orphan designation: Recombinant human IgG1 kappa light chain monoclonal antibody targeting plasma kallikrein (lanadelumab) for: Treatment of hereditary angioedema
Date of designation: 09/10/2015, Positive, Last updated: 04/10/2022 -
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Orphan designation: recombinant adeno-associated viral vector serotype 2 carrying the gene for the human aromatic L-amino acid decarboxylase protein, Eladocagene exuparvovec for: Treatment of aromatic L-amino acid decarboxylase deficiency
Date of designation: 18/11/2016, Positive, Last updated: 10/08/2022 -
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Orphan designation: Carboxypeptidase G2 (glucarpidase) for: Adjunctive treatment in patients at risk of methotrexate toxicity
Date of designation: 03/02/2003, Positive, Last updated: 20/01/2022 -
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Orphan designation: Chimeric monoclonal antibody against GD2 for: Treatment of neuroblastoma
Date of designation: 08/11/2012, Positive, Last updated: 26/08/2021 -
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Orphan designation: Chimeric anti-interleukin-6 monoclonal antibody (siltuximab) for: Treatment of Castleman's disease
Date of designation: 30/11/2007, Positive, Last updated: 26/08/2021 -
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Orphan designation: (2S)-2-{[(2R)-2-[({[3,3-dibutyl-7-(methylthio)-1,1-dioxido-5-phenyl-2,3,4,5-tetrahydro-1,2,5-benzothiadiazepin-8-yl]oxy}acetyl)amino]-2-(4-hydroxyphenyl)acetyl]amino}butanoic acid for: Treatment of progressive familial intrahepatic cholestasis
Date of designation: 17/07/2012, Positive, Last updated: 28/07/2021 -
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Orphan designation: recombinant human interleukin-3 truncated diphtheria toxin fusion protein for: Treatment of blastic plasmacytoid dendritic cell neoplasm
Date of designation: 11/11/2015, Positive, Last updated: 25/01/2021 -
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Orphan designation: Obiltoxaximab for: Treatment of anthrax
Date of designation: 24/08/2018, Positive, Last updated: 27/11/2020 -
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Orphan designation: Recombinant human beta-glucuronidase (vestronidase alfa) for: Treatment of mucopolysaccharidosis type VII (Sly syndrome)
Date of designation: 21/03/2012, Positive, Last updated: 11/11/2020 -
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Orphan designation: Ketoconazole for: Treatment of Cushing's syndrome
Date of designation: 23/04/2012, Positive, Last updated: 22/09/2020 -
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Orphan designation: Metreleptin for: Treatment of familial partial lipodystrophy
Date of designation: 17/07/2012, Positive, Last updated: 02/04/2020 -
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Orphan designation: Metreleptin for: Treatment of Barraquer-Simons syndrome
Date of designation: 17/07/2012, Positive, Last updated: 02/04/2020 -
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Orphan designation: Metreleptin for: Treatment of Berardinelli-Seip syndrome
Date of designation: 17/07/2012, Positive, Last updated: 02/04/2020 -
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Orphan designation: Metreleptin for: Treatment of Lawrence syndrome
Date of designation: 17/07/2012, Positive, Last updated: 02/04/2020 -
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Orphan designation: tafamidis for: Treatment of senile systemic amyloidosis
Date of designation: 08/11/2012, Positive, Last updated: 25/02/2020 -
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Orphan designation: Recombinant human tissue non-specific alkaline phosphatase - Fc - deca-aspartate fusion protein for: Treatment of hypophosphatasia
Date of designation: 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 designation: 17/12/2010, Positive, Last updated: 20/11/2019 -
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Orphan designation: Eculizumab for: Treatment of neuromyelitis optica spectrum disorders
Date of designation: 05/08/2013, Positive, Last updated: 20/11/2019 -
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Orphan designation: Eculizumab for: Treatment myasthenia gravis
Date of designation: 29/07/2014, Positive, Last updated: 20/11/2019 -
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Orphan designation: Eculizumab for: Treatment of atypical haemolytic uraemic syndrome (aHUS)
Date of designation: 24/07/2009, Positive, Last updated: 20/11/2019 -
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Orphan designation: Lonafarnib for: Treatment of Hutchinson-Gilford progeria
Date of designation: 14/12/2018, Positive, Last updated: 01/08/2019 -
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Orphan designation: Phosphorothioate oligonucleotide targeted to transthyretin (inotersen) for: Treatment of ATTR amyloidosis
Date of designation: 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 designation: 14/09/2007, Positive, Last updated: 26/03/2019 -
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Orphan designation: Antisense oligonucleotide targeted to the SMN2 gene (nusinersen) for: Treatment of 5q spinal muscular atrophy
Date of designation: 02/04/2012, Positive, Last updated: 16/10/2018 -
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Orphan designation: Daratumumab for: Treatment of plasma-cell myeloma
Date of designation: 17/07/2013, Positive, Last updated: 02/10/2018