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1713 results
Medicine
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Orphan designation: dimethyl fumarate for: Adjunctive treatment in haematopoietic cell transplantation (updated)
Date of first decision: 21/03/2018, Positive, Last updated: 15/01/2021 -
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Orphan designation: Fenfluramine hydrochloride for: Treatment of Dravet syndrome (updated)
Date of first decision: 18/12/2013, Positive, Last updated: 08/01/2021 -
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Orphan designation: Autologous CD34+ cells transfected with lentiviral vector containing the human arylsulfatase A cDNA for: treatment of metachromatic leukodystrophy (updated)
Date of first decision: 13/04/2007, Positive, Last updated: 22/12/2020 -
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Orphan designation: Miransertib for: Treatment of Proteus syndrome (updated)
Date of first decision: 21/03/2018, Positive, Last updated: 22/12/2020 -
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Orphan designation: Obeticholic acid for: Treatment of primary sclerosing cholangitis (updated)
Date of first decision: 16/03/2014, Positive, Last updated: 22/12/2020 -
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Orphan designation: Ibutamoren mesilate for: Treatment of growth hormone deficiency (updated)
Date of first decision: 20/06/2017, Positive, Last updated: 22/12/2020 -
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Orphan designation: Bardoxolone methyl for: Treatment of Alport syndrome
Date of first decision: 25/05/2018, Positive, Last updated: 21/12/2020 -
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Orphan designation: Treosulfan for: Conditioning treatment prior to haematopoietic-progenitor-cell transplantation
Date of first decision: 22/02/2004, Positive, Last updated: 11/12/2020 -
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Orphan designation: Adeno-associated viral vector serotype 9 containing the human SMN gene (onasemnogene abeparvovec) for: Treatment of spinal muscular atrophy
Date of first decision: 19/06/2015, Positive, Last updated: 10/12/2020 -
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Orphan designation: (S)-2-nitro-6-(4-trifluoromethoxy)benzyloxy)-6,7-dihydro-5H-imidazo[2,1-b] [1,3] oxazine (pretomanid) for: Treatment of tuberculosis
Date of first decision: 29/11/2007, Positive, Last updated: 09/12/2020 -
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Orphan designation: Adeno-associated viral vector expressing acid alpha-glucosidase gene for: Treatment of glycogen storage disease type II (Pompe's disease)
Date of first decision: 27/07/2020, Positive, Last updated: 08/12/2020 -
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Orphan designation: 5-aminolevulinic acid for: Treatment of glioma
Date of first decision: 12/01/2017, Positive, Last updated: 08/12/2020 -
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Orphan designation: Encapsulated human retinal pigment epithelial cell line transfected with plasmid vector expressing human ciliary neurotrophic factor for: Treatment of retinitis pigmentosa
Date of first decision: 24/01/2013, Positive, Last updated: 07/12/2020 -
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Orphan designation: Recombinant adeno-associated viral vector serotype 5 encoding Staphylococcus aureus Cas9 endonuclease and two guide RNAs complementary to two regions of intron 26 of the CEP290 gene for: Treatment of Leber's congenital amaurosis
Date of first decision: 16/10/2017, Positive, Last updated: 30/11/2020 -
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Orphan designation: Recombinant human bone morphogenetic protein 4 for: Treatment of glioma
Date of first decision: 15/10/2014, Positive, Last updated: 30/11/2020 -
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Orphan designation: Obiltoxaximab for: Treatment of anthrax
Date of first decision: 24/08/2018, Positive, Last updated: 27/11/2020 -
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Orphan designation: Picropodophyllin for: Treatment of glioma
Date of first decision: 23/08/2017, Positive, Last updated: 27/11/2020 -
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Orphan designation: Synthetic double-stranded siRNA oligonucleotide directed against hydroxyacid oxidase 1 mRNA and covalently linked to a ligand containing three N-acetylgalactosamine residues (lumasiran) for: Treatment of primary hyperoxaluria
Date of first decision: 21/03/2016, Positive, Last updated: 25/11/2020 -
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Orphan designation: Hydroxychloroquine for: Treatment of antiphospholipid syndrome
Date of first decision: 12/01/2017, Positive, Last updated: 24/11/2020 -
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Orphan designation: synthetic 15-amino-acid macrocyclic peptide acylated with a polyethyleneglycol palmitoylated linker (zilucoplan) for: Treatment of paroxysmal nocturnal haemoglobinuria
Date of first decision: 14/10/2016, Positive, Last updated: 24/11/2020 -
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Orphan designation: Humanised monoclonal antibody against P-selectin (crizanlizumab) for: Treatment of sickle cell disease
Date of first decision: 09/08/2012, Positive, Last updated: 24/11/2020 -
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Orphan designation: Recombinant human beta-glucuronidase (vestronidase alfa) for: Treatment of mucopolysaccharidosis type VII (Sly syndrome)
Date of first decision: 21/03/2012, Positive, Last updated: 11/11/2020 -
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Orphan designation: Adeno-associated viral vector serotype rh10 containing the human factor IX gene for the treatment of haemophilia B- for: Treatment of haemophilia B
Date of first decision: 14/12/2015, Positive, Last updated: 11/11/2020 -
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Orphan designation: Triheptanoin for: Treatment of carnitine palmitoyltransferase II deficiency
Date of first decision: 28/07/2015, Positive, Last updated: 11/11/2020 -
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Orphan designation: Triheptanoin for: Treatment of mitochondrial trifunctional protein deficiency
Date of first decision: 28/07/2015, Positive, Last updated: 11/11/2020