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716 results
Orphan designation status
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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Date of designation: 26/03/2014, Withdrawn, Last updated: 06/06/2023 -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: 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Date of designation: 19/03/2015, Withdrawn, Last updated: 05/06/2023 -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: 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Date of designation: 21/03/2018, Withdrawn, Last updated: 30/05/2023 -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: 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Date of designation: 12/12/2017, Withdrawn, Last updated: 08/05/2023 -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: Rozanolixizumab for: Treatment of immune thrombocytopenia
Date of designation: 11/01/2019, Withdrawn, Last updated: 08/05/2023 -
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Orphan designation: 3-(3-(3,5-bis(trifluoromethyl)phenyl)-1h-pyrazol-1-yl)propanoic acid for: Treatment of Stargardt's disease
Date of designation: 24/04/2019, Withdrawn, Last updated: 13/04/2023 -
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Orphan designation: (R)-1-[1-(4-acetoxy-3,3-dimethyl-2-oxo-butyl)-2-oxo-5-(pyridin-2-yl)-2,3-dihydro-1H-benzo[e][1,4]diazepin-3-yl]-3-(3-methylamino-phenyl)-urea (ceclazepide) for: Treatment of gastro-entero-pancreatic neuroendocrine tumours
Date of designation: 14/12/2015, Withdrawn, Last updated: 04/04/2023 -
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Orphan designation: Soluble recombinant human fibroblast growth factor receptor 3 for: Treatment of achondroplasia
Date of designation: 27/02/2017, Withdrawn, Last updated: 24/03/2023 -
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Orphan designation: miglustat for: Treatment of glycogen storage disease type II (Pompe's disease)
Date of designation: 11/01/2019, Withdrawn, Last updated: 24/03/2023 -
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Orphan designation: 2-hydroxy-N,N,N-trimethylethan-1-aminium (Z)-4-(5-((3-benzyl-4-oxo-2-thioxothiazolidin-5-ylidene)methyl)furan-2-yl)benzoate for: Treatment of pancreatic cancer
Date of designation: 24/03/2020, Withdrawn, Last updated: 16/02/2023 -
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Orphan designation: Berzosertib for: Treatment of small cell lung cancer
Date of designation: 18/07/2022, Withdrawn, Last updated: 07/02/2023 -
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Orphan designation: idursulfase for: Treatment of mucopolysaccharidosis type II (Hunter's syndrome)
Date of designation: 06/01/2021, Withdrawn, Last updated: 27/01/2023 -
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Orphan designation: Loncastuximab tesirine for: Treatment of diffuse large B-cell lymphoma
Date of designation: 20/08/2021, Withdrawn, Last updated: 26/01/2023 -
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Orphan designation: zanubrutinib for: Treatment of marginal zone lymphoma
Date of designation: 20/05/2021, Withdrawn, Last updated: 05/01/2023 -
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Orphan designation: regorafenib for: Treatment of glioma
Date of designation: 28/06/2019, Withdrawn, Last updated: 20/12/2022 -
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Orphan designation: Allogeneic retinal pigment epithelial cells genetically modified with a non-viral vector to express human alpha-L-iduronidase for: Treatment of mucopolysaccharidosis type I
Date of designation: 15/10/2021, Withdrawn, Last updated: 20/12/2022 -
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Orphan designation: Concizumab for: Treatment of haemophilia B
Date of designation: 12/10/2017, Withdrawn, Last updated: 14/12/2022 -
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Orphan designation: Humanised monoclonal IgG4 antibody against tissue-factor-pathway inhibitor (concizumab) for: Treatment of haemophilia A
Date of designation: 10/10/2012, Withdrawn, Last updated: 14/12/2022 -
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Orphan designation: Parsaclisib for: Treatment of marginal zone lymphoma
Date of designation: 25/07/2019, Withdrawn, Last updated: 13/12/2022 -
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Orphan designation: Tremelimumab for: Treatment of hepatocellular carcinoma
Date of designation: 09/12/2020, Withdrawn, Last updated: 09/12/2022 -
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Orphan designation: Allogeneic retinal pigment epithelial cells genetically modified with a non-viral vector to express beta-domain deleted human factor VIII for: Treatment of haemophilia A
Date of designation: 09/12/2020, Withdrawn, Last updated: 05/12/2022 -
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Orphan designation: Autologous CD34+ haematopoietic stem cells transduced with lentiviral vector encoding the human betaA-T87Q-globin gene (betibeglogene autotemcel) for: Treatment of beta thalassaemia intermedia and major
Date of designation: 24/01/2013, Withdrawn, Last updated: 30/11/2022 -
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Orphan designation: 1-(2-hydroxyethyl)-8-{[5-(4-methylpiperazin-1-yl)-2-(trifluoromethoxy) phenyl]amino}-4,5-dihydro-1H-pyrazolo[4,3-h]quinazoline-3-carboxamide fumarate salt (onvansertib) for: Treatment of acute myeloid leukaemia
Date of designation: 24/08/2018, Withdrawn, Last updated: 22/11/2022 -
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Orphan designation: 5-{(1R,2R)-2-[(cyclopropylmethyl)amino]cyclopropyl}-N-(tetrahydro-2H-pyran-4-yl)thiophene-3-carboxamide monohydrochloride for: Treatment of Kabuki syndrome
Date of designation: 19/11/2018, Withdrawn, Last updated: 22/11/2022 -
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Orphan designation: 6-((3S,4S)-4-methyl-1-(pyrimidin-2-yl-methyl)pyrrolidin-3-yl)-3-tetrahydropyran-4-yl-7H-imidazo(1,5-a)pyrazin-8-one (Tovinontrine) for: Treatment of sickle cell disease
Date of designation: 21/08/2020, Withdrawn, Last updated: 08/11/2022