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Orphan designation: 7-ethyl-10-hydroxy-camptothecin for: Treatment of soft tissue sarcoma (updated)
Date of designation: 25/07/2019, Positive, Last updated: 09/06/2023 -
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Orphan designation: Sirolimus for: Prevention of arteriovenous access dysfunction in haemodialysis patients (updated)
Date of designation: 13/11/2013, Positive, Last updated: 09/06/2023 -
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Orphan designation: Glucagon for: Treatment of noninsulinoma pancreatogenous hypoglycaemia syndrome (updated)
Date of designation: 19/11/2018, Positive, Last updated: 09/06/2023 -
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Orphan designation: Sulfonated monophosphorylated mannose oligosaccharide for: Treatment of hepatocellular carcinoma (updated)
Date of designation: 21/06/2011, Positive, Last updated: 09/06/2023 -
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Orphan designation: Glucagon for: Treatment of congenital hyperinsulinism (updated)
Date of designation: 08/10/2009, Positive, Last updated: 09/06/2023 -
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Orphan designation: Triamcinolone acetonide for: Treatment of non-infectious uveitis (updated)
Date of designation: 21/05/2015, Positive, Last updated: 09/06/2023 -
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Orphan designation: 26 base synthetic single-stranded fully phosphorothioated 2'-O-methyl-RNA and DNA mixmer oligonucleotide-based compound for: Treatment of Dravet syndrome (updated)
Date of designation: 27/02/2017, Positive, Last updated: 09/06/2023 -
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Orphan designation: H-Lys-Lys-Gly-Asp-Asn-Ile-Met-Val-Thr-Phe-Arg-Asn-Gln-Ala-Ser-Arg-Pro-Tyr-Gly-Lys-Lys-OH for: Treatment of haemophilia A (updated)
Date of designation: 10/08/2022, Positive, Last updated: 09/06/2023 -
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Orphan designation: Sirolimus for: Treatment of tuberous sclerosis (updated)
Date of designation: 23/08/2017, Positive, Last updated: 09/06/2023 -
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Orphan designation: Allogeneic multi-virus specific T lymphocytes targeting BK virus, cytomegalovirus, human herpesvirus-6, Epstein Barr virus and adenovirus for: Treatment in haematopoietic stem cell transplantation (updated)
Date of designation: 24/03/2020, Positive, Last updated: 08/06/2023 -
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Orphan designation: A combination of H-Lys-Lys-Gly-Pro-Arg-Cys(SH)-Leu-Thr-Arg-Tyr-Tyr-Ser-Ser-Phe-Val-Asn-Met-Glu-Gly-Lys-Lys-OH and H-Lys-Lys-Gly-Asp-Asn-Ile-Met-Val-Thr-Phe-Arg-Asn-Gln-Ala-Ser-Arg-Pro-Tyr-Gly-Lys-Lys-OH for: Treatment of haemophilia A (updated)
Date of designation: 19/11/2014, Positive, Last updated: 07/06/2023 -
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Orphan designation: Autologous CD34+ cells transduced with a lentiviral vector containing the human RAG1 gene for: Treatment of recombination-activating gene 1 deficient severe combined immunodeficiency (updated)
Date of designation: 26/03/2014, Positive, Last updated: 07/06/2023 -
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Orphan designation: Doxorubicin(6-maleimidocaproyl)hydrazone (aldoxorubicin) for: Treatment of soft tissue sarcoma (updated)
Date of designation: 26/03/2014, Withdrawn, Last updated: 06/06/2023 -
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Orphan designation: Citric acid monohydrate for: Treatment of acute liver failure (updated)
Date of designation: 27/06/2016, Positive, Last updated: 05/06/2023 -
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Orphan designation: 4,7,10,13,16,19-docosahexaenoic acid for: Treatment of retinitis pigmentosa (updated)
Date of designation: 04/11/2006, Positive, Last updated: 05/06/2023 -
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Orphan designation: 5'-ASCSASTSCSASGSTSCSTSGSASUSASASGSCSTSA-3' (lademirsen) for: Treatment of Alport syndrome (updated)
Date of designation: 19/03/2015, Withdrawn, Last updated: 05/06/2023 -
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Orphan designation: Viltolarsen for: Treatment of Duchenne muscular dystrophy (updated)
Date of designation: 04/06/2020, Positive, Last updated: 01/06/2023 -
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Orphan designation: Recombinant human acid alpha-glucosidase for: Treatment of glycogen storage disease type II (Pompe's disease) (updated)
Date of designation: 21/03/2018, Withdrawn, Last updated: 30/05/2023 -
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Orphan designation: Ivosidenib for: Treatment of biliary tract cancer (updated)
Date of designation: 21/03/2018, Positive, Last updated: 12/05/2023 -
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Orphan designation: Ivosidenib for: Treatment of acute myeloid leukaemia (updated)
Date of designation: 12/12/2016, Positive, Last updated: 12/05/2023 -
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Orphan designation: Govorestat for: Treatment of galactosaemia
Date of designation: 21/06/2022, Positive, Last updated: 10/05/2023 -
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Orphan designation: Pegunigalsidase alfa for: Treatment of Fabry disease
Date of designation: 12/12/2017, Withdrawn, Last updated: 08/05/2023 -
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Orphan designation: N-[(2S)-5-{[(1R, 2S)-2-(4-fluorophenyl)cyclopropyl]amino}-1-(4-methylpiperazin-1-yl)-1-oxopentan-2-yl]-4-(1H-1,2,3-triazol-1-yl)benzamide, bis-tosilate salt (Bomedemstat ditosilate) for: Treatment of myelofibrosis
Date of designation: 14/10/2016, Positive, Last updated: 08/05/2023 -
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Orphan designation: 1-[(3S)-3-{4-amino-3-[(3,5-dimethoxyphenyl)ethynyl]-1H-pyrazolo[3,4-d]pyrimidin-1-yl}pyrrolidin-1-yl]-2-propen-1-one (futibatinib) for: Treatment of biliary tract cancer
Date of designation: 01/04/2019, Positive, Last updated: 08/05/2023 -
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Orphan designation: Nimorazole for: Treatment of squamous-cell carcinoma of the head and neck in patients undergoing radiotherapy
Date of designation: 23/02/2011, Positive, Last updated: 08/05/2023