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589 results
Orphan designation status
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Orphan designation: Adeno-associated viral vector serotype 2/2 containing a gene encoding the channelrhodopsin-2 protein for: Treatment of retinitis pigmentosa (updated)
Date of designation: 14/10/2016, Withdrawn, Last updated: 11/06/2021 -
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Orphan designation: Adeno-associated viral vector expressing lipoprotein lipase (alipogene tiparvovec) for: Treatment of lipoprotein-lipase deficiency (updated)
Date of designation: 09/03/2004, Withdrawn, Last updated: 31/05/2021 -
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Orphan designation: Recombinant human alpha-1-microglobulin for: Treatment of pre-eclampsia
Date of designation: 04/07/2014, Withdrawn, Last updated: 11/05/2021 -
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Orphan designation: tripotassium citrate monohydrate, Potassium hydrogen carbonate for: Treatment of distal renal tubular acidosis
Date of designation: 20/06/2017, Withdrawn, Last updated: 03/05/2021 -
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Orphan designation: Codon-optimised human ornithine transcarbamylase mRNA complexed with lipid-based nanoparticles for: Treatment of ornithine transcarbamylase deficiency
Date of designation: 27/06/2018, Withdrawn, Last updated: 28/04/2021 -
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Orphan designation: Ixazomib for: Treatment of systemic light chain amyloidosis
Date of designation: 08/11/2012, Withdrawn, Last updated: 09/04/2021 -
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Orphan designation: 2-((2-ethyl-6-(4-(2-(3-hydroxyazetidin-1-yl)-2-oxoethyl)-piperazin-1-yl)-8-methylimidazo[1,2-alpha]pyridin-3-yl)-(methyl)amino)-4-(4-fluorophenyl)-thiazole-5-carbonitrile (ziritaxestat) for: Treatment of idiopathic pulmonary fibrosis
Date of designation: 29/08/2016, Withdrawn, Last updated: 09/04/2021 -
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Orphan designation: Chimeric monoclonal antibody against claudin 6 for: Treatment of ovarian cancer
Date of designation: 24/01/2013, Withdrawn, Last updated: 08/04/2021 -
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Orphan designation: Recombinant human glutamate oxaloacetate transaminase 1 for: Treatment of glioma
Date of designation: 12/02/2015, Withdrawn, Last updated: 07/04/2021 -
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Orphan designation: romilkimab for: Treatment of systemic sclerosis
Date of designation: 09/01/2020, Withdrawn, Last updated: 06/04/2021 -
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Orphan designation: ziritaxestat for: Treatment of systemic sclerosis
Date of designation: 09/01/2020, Withdrawn, Last updated: 06/04/2021 -
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Orphan designation: Terguride for: Treatment of systemic sclerosis
Date of designation: 24/01/2013, Withdrawn, Last updated: 22/03/2021 -
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Orphan designation: milciclib maleate for: Treatment of malignant thymoma
Date of designation: 08/11/2012, Withdrawn, Last updated: 22/03/2021 -
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Orphan designation: Allogeneic umbilical cord blood CD34+ cells cultured ex vivo with Notch ligand Delta1 (dilanubicel) for: Treatment in haematopoietic stem cell transplantation
Date of designation: 17/01/2018, Withdrawn, Last updated: 22/03/2021 -
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Orphan designation: Levosimendan for: Treatment of amyotrophic lateral sclerosis
Date of designation: 22/02/2018, Withdrawn, Last updated: 19/03/2021 -
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Orphan designation: Humanised single-chain monoclonal antibody against CD37 (otlertuzumab) for: Treatment chronic lymphocytic leukaemia
Date of designation: 06/12/2012, Withdrawn, Last updated: 19/03/2021 -
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Orphan designation: Adeno-associated viral vector serotype 8 containing the human UGT1A1 gene (volrubigene ralaparvovec) for: Treatment of Crigler-Najjar syndrome
Date of designation: 22/08/2014, Withdrawn, Last updated: 19/03/2021 -
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Orphan designation: Nimorazole maleate for: Treatment of squamous-cell carcinoma of the head and neck in patients undergoing radiotherapy
Date of designation: 09/02/2012, Withdrawn, Last updated: 19/03/2021 -
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Orphan designation: Three chimeric human/murine monoclonal antibodies against the Ebola (Zaire) surface glycoprotein (porgaviximab) for: Treatment for Ebola virus disease
Date of designation: 09/10/2015, Withdrawn, Last updated: 19/03/2021 -
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Orphan designation: 5-(Ethylsulfonyl)-2-(naphthalen-2-yl)benzo[d]oxazole (ezutromid) for: Treatment of Duchenne muscular dystrophy
Date of designation: 04/12/2008, Withdrawn, Last updated: 10/02/2021 -
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Orphan designation: Human monoclonal antibody against Pseudomonas aeruginosa IATS-O1 for: Treatment of pneumonia caused by serotype O1 Pseudomonas aeruginosa
Date of designation: 28/01/2010, Withdrawn, Last updated: 03/02/2021 -
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Orphan designation: RNA, [P-deoxy-P-(dimethylamino)] (2',3'-dideoxy-2',3'-imino-2',3'-seco) (2'a→5') (C-m5U-m5U-A-C-A-G-G-C-m5U-C-C-A-A-m5U-A-G-m5U-G-G-m5U-C-A-G-m5U), 5' [P-[4-[[2-[2-(2-hydroxyethoxy)ethoxy]ethoxy]carbonyl]-1-piperazinyl]-N,N-dimethylaminophosphonamidate], 3'-[2'a-[N2-acetyl-L-arginyl-6-aminohexanoyl-L-arginyl-L-arginyl-β-alanyl-L-arginyl-L-arginyl-6-aminohexanoyl-L-arginyl-L-arginyl-β-alanyl-L-arginyl-6-aminohexanoyl-β-alanyl], octahydrochloride for: Treatment of Duchenne muscular dystrophy
Date of designation: 02/02/2010, Withdrawn, Last updated: 03/02/2021 -
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Orphan designation: Sodium thiosulfate for: Treatment of calciphylaxis
Date of designation: 23/02/2011, Withdrawn, Last updated: 03/02/2021 -
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Orphan designation: Adeno-associated viral vector serotype 8 containing the human UGT1A1 gene (volrubigene ralaparvovec) for: Treatment of Crigler-Najjar syndrome
Date of designation: 18/11/2016, Withdrawn, Last updated: 03/02/2021 -
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Orphan designation: Heparin-activated recombinant human fibroblast growth factor 1 (on a biodegradable device made from alpha-calcium sulfate hemihydrate) for: Treatment of traumatic spinal cord injury
Date of designation: 27/07/2010, Withdrawn, Last updated: 03/02/2021