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45 results
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Orphan designation: ciclosporin for: Treatment of vernal keratoconjunctivitis
Date of first decision: 06/04/2006, Positive, Last updated: 12/07/2018 -
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Orphan designation: Synthetic double-stranded siRNA oligonucleotide directed against transthyretin mRNA (also known as patisiran) for: Treatment of transthyretin-mediated amyloidosis
Date of first decision: 06/04/2017, Positive, Last updated: 30/05/2018 -
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Orphan designation: Recombinant human alpha-Mannosidase (velmanase alfa) for: Treatment of alpha-mannosidosis
Date of first decision: 26/01/2005, Positive, -
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Orphan designation: budesonide for: Treatment of eosinophilic oesophagitis
Date of first decision: 05/08/2013, Positive, Last updated: 18/01/2018 -
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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 first decision: 09/10/2015, Positive, Last updated: 28/09/2017 -
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Orphan designation: Chenodeoxycholic acid for: Treatment of inborn errors in primary bile acid synthesis
Date of first decision: 16/12/2014, Positive, Last updated: 29/06/2017 -
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Orphan designation: defibrotide for: Treatment of hepatic veno-occlusive disease
Date of first decision: 29/07/2004, Positive, Last updated: 17/08/2016 -
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Orphan designation: cholic acid for: Treatment of inborn errors in primary bile acid synthesis
Date of first decision: 18/12/2002, Positive, Last updated: 12/04/2016 -
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Orphan designation: idebenone for: Treatment of Leber's hereditary optic neuropathy
Date of first decision: 16/02/2007, Positive, Last updated: 11/04/2016 -
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Orphan designation: idebenone for: Treatment of Duchenne muscular dystrophy
Date of first decision: 20/03/2007, Positive, Last updated: 11/04/2016 -
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Orphan designation: [Nle4, D-Phe7]-alfa-melanocyte stimulating hormone (afamelanotide) for: Treatment of erythropoietic protoporphyria
Date of first decision: 08/05/2008, Positive, Last updated: 11/04/2016 -
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Orphan designation: Recombinant human tripeptidyl-peptidase 1 for: Treatment of neuronal ceroid lipofuscinosis type 2
Date of first decision: 12/03/2013, Positive, Last updated: 09/02/2016 -
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Orphan designation: Recombinant human nerve growth factor (cenegermin) for: Treatment of neurotrophic keratitis
Date of first decision: 14/12/2015, Positive, Last updated: 02/02/2016 -
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Orphan designation: carfilzomib for: Treatment of multiple myeloma
Date of first decision: 03/06/2008, Positive, Last updated: 02/12/2015 -
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Orphan designation: Benzamide, 3-(2-imidazo[1,2-b]pyridazin-3-ylethynyl)-4-methyl-N-[4-[(4-methyl-1-piperazinyl)methyl]-3-(trifluoromethyl)phenyl] (ponatinib) for: Treatment of chronic myeloid leukaemia
Date of first decision: 02/02/2010, Positive, Last updated: 23/03/2015 -
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Orphan designation: Benzamide, 3-(2-imidazo[1,2-b]pyridazin-3-ylethynyl)-4-methyl-N-[4-[(4-methyl-1-piperazinyl)methyl]-3-(trifluoromethyl)phenyl] (ponatinib) for: Treatment of acute lymphoblastic leukaemia
Date of first decision: 02/02/2010, Positive, Last updated: 23/03/2015 -
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Orphan designation: Chimeric anti-interleukin-6 monoclonal antibody (siltuximab) for: Treatment of Castleman's disease
Date of first decision: 30/11/2007, Positive, Last updated: 11/07/2014 -
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Orphan designation: Adeno-associated viral vector expressing lipoprotein lipase for: Treatment of lipoprotein-lipase deficiency
Date of first decision: 09/03/2004, Positive, Last updated: 17/10/2013 -
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Orphan designation: velaglucerase alfa for: Treatment of Gaucher disease
Date of first decision: 06/06/2010, Positive, Last updated: 23/06/2010 -
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Orphan designation: Risdiplam for: Treatment of spinal muscular atrophy
Date of first decision: 26/02/2019, Positive, Last updated: 09/04/2019