2298 results
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Orphan designation: icatibant acetate for: Treatment of angioedema (updated)
Date of first decision: 18/02/2003, Expired, Last updated: 12/08/2020 -
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Orphan designation: Recombinant human galactocerebrosidase for: Treatment of globoid cell leukodystrophy (Krabbe disease) (updated)
Date of first decision: 27/09/2011, Withdrawn, Last updated: 12/08/2020 -
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Orphan designation: Recombinant human porphobilinogen deaminase for: Treatment of acute intermittent porphyria (updated)
Date of first decision: 12/06/2002, Withdrawn, Last updated: 12/08/2020 -
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Orphan designation: Cabiralizumab for: Treatment of tenosynovial giant cell tumour, localised and diffuse type (updated)
Date of first decision: 12/12/2016, Withdrawn, Last updated: 12/08/2020 -
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Orphan designation: N-[(2S)-2,3-dihydroxypropyl]-3-[(2-fluoro-4-iodophenyl)amino]isonicotinamide hydrochloride for: Treatment of pancreatic cancer (updated)
Date of first decision: 08/10/2009, Withdrawn, Last updated: 12/08/2020 -
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Orphan designation: N-[(2S)-2,3-dihydroxypropyl]-3-[(2-fluoro-4-iodophenyl)amino] isonicotinamide hydrochloride for: Treatment of acute myeloid leukaemia (updated)
Date of first decision: 17/12/2010, Withdrawn, Last updated: 12/08/2020 -
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Orphan designation: Humanised monoclonal antibody to the folate receptor alpha for: Treatment of ovarian cancer (updated)
Date of first decision: 01/04/2008, Positive, Last updated: 11/08/2020 -
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Orphan designation: Amatuximab for: Treatment of malignant mesothelioma (updated)
Date of first decision: 16/01/2014, Positive, Last updated: 11/08/2020 -
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Orphan designation: N-{2-[(6-{[(2,6-dichloro-3,5-dimethoxyphenyl)carbamoyl](methyl)amino}pyrimidin-4-yl)amino]-5-(4-ethylpiperazin-1-yl)phenyl}prop-2-enamide for: Treatment of hepatocellular carcinoma (updated)
Date of first decision: 23/08/2017, Positive, Last updated: 11/08/2020 -
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Orphan designation: Denileukin diftitox for: Treatment of cutaneous T-cell lymphoma (updated)
Date of first decision: 11/12/2001, Positive, Last updated: 11/08/2020 -
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Orphan designation: (6aR, 10aR)-3-(1',1'-dimethylheptyl)-delta-8-tetrahydro-cannabinol-9-carboxylic acid for: Treatment of systemic sclerosis (updated)
Date of first decision: 12/01/2017, Positive, Last updated: 11/08/2020 -
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Orphan designation: (6aR,10aR)-3-(1',1'-dimethylheptyl)-delta-8-tetrahydrocannabinol-9-carboxylic acid- for: Treatment of cystic fibrosis (updated)
Date of first decision: 14/10/2016, Positive, Last updated: 11/08/2020 -
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Orphan designation: cholic acid for: Treatment of inborn errors in primary bile acid synthesis responsive to treatment with cholic acid (updated)
Date of first decision: 29/10/2009, Withdrawn, Last updated: 29/07/2020 -
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Orphan designation: (6aR,10aR)-3-(1,1-dimethylheptyl)-delta8-tetrahydro-cannabinol-9-carboxylic acid for: Treatment of dermatomyositis (updated)
Positive, Last updated: 29/07/2020 -
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Orphan designation: 4-[3-(Methylsulfonyl)phenyl]-1-propylpiperidine x HC1 (pridopidine hydrochloride) for: Treatment of Huntington’s disease (updated)
Date of first decision: 20/06/2005, Positive, Last updated: 28/07/2020 -
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Orphan designation: Marzeptacog alfa (activated) for: Treatment of haemophilia B (updated)
Date of first decision: 01/04/2019, Positive, Last updated: 21/07/2020 -
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Orphan designation: Recombinant human factor IX protein modified with three point mutations (dalcinonacog alfa) for: Treatment of haemophilia B (updated)
Date of first decision: 20/06/2017, Positive, Last updated: 20/07/2020 -
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Orphan designation: Humanised Fc engineered monoclonal antibody against CD19, active ingredient tafasitamab for: Treatment of diffuse large B-cell lymphoma
Date of first decision: 15/01/2015, Positive, Last updated: 13/07/2020 -
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Orphan designation: Oleylphosphocholine for: Treatment of leishmaniasis
Date of first decision: 23/04/2012, Positive, Last updated: 10/07/2020 -
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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: Ciprofloxacin (liposomal) for: Treatment of cystic fibrosis
Date of first decision: 24/07/2009, Positive, Last updated: 26/06/2020 -
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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: 2'-O-methyl phosphorothioate RNA oligonucleotide, 5'-m5CUGm5CUGm5CUGm5CUGm5CUGm5CUGm5CUG-3' for: Treatment of Huntington’s disease
Date of first decision: 12/02/2015, Positive, Last updated: 23/06/2020 -
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Orphan designation: Humanised monoclonal antibody against CD38 for the treatment of plasma cell myeloma (isatuximab) for: Treatment of plasma cell myeloma
Date of first decision: 29/04/2014, Positive, Last updated: 12/06/2020 -
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Orphan designation: propagermanium for: Treatment of focal segmental glomerulosclerosis
Date of first decision: 19/11/2018, Positive, Last updated: 11/06/2020