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217 results
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Orphan designation: Expanded human allogeneic mesenchymal adult stem cells extracted from adipose tissue (darvadstrocel) for: Treatment of anal fistula
Date of first decision: 08/10/2009, Positive, Last updated: 26/08/2020 -
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Orphan designation: Brentuximab vedotin for: Treatment of cutaneous T-cell lymphoma
Date of first decision: 11/01/2012, Positive, Last updated: 26/08/2020 -
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Orphan designation: 2,2'-{2-[(1R)-1-({[(2,5-dichlorobenzoyl)amino]acetyl}amino)-3-methylbutyl]-5-oxo-1,3,2-dioxaborolane-4,4-diyl}diacetic acid for: Treatment of multiple myeloma
Date of first decision: 27/09/2011, Positive, Last updated: 26/08/2020 -
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Orphan designation: "Synthetic 47-amino-acid N-myristoylated lipopeptide, derived from the preS region of hepatitis B virus (bulevirtide) for: Treatment of hepatitis delta virus infection
Date of first decision: 19/06/2015, Positive, Last updated: 12/08/2020 -
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Orphan designation: Osilodrostat for: Treatment of Cushing's syndrome
Date of first decision: 15/10/2014, Positive, Last updated: 07/07/2020 -
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Orphan designation: Ex-vivo-expanded autologous human corneal epithelium-containing stem cells for: Corneal lesions, with associated corneal (limbal) stem cell deficiency, due to ocular burns
Date of first decision: 07/11/2008, Positive, Last updated: 23/06/2020 -
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Orphan designation: Beclomethasone 17, 21-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: R)-1-(3-(aminomethyl) phenyl)-N-(5-((3-cyanophenyl)(cyclopropylmethylamino)methyl)-2-fluorophenyl)-3-(trifluoromethyl)-1H-pyrazole-5-carboxamide dihydrochloride (berotralstat) for: Treatment of hereditary angioedema
Date of first decision: 27/06/2018, 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 of neuromyelitis optica spectrum disorders
Date of first decision: 05/08/2013, Positive, Last updated: 20/11/2019