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18 results
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Orphan designation: Anti-CD38 IgG4 human monoclonal antibody for: Treatment of amyotrophic lateral sclerosis
Date of designation: 12/11/2021, Positive, Last updated: 07/06/2022 -
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Orphan designation: Linerixibat for: Treatment of primary biliary cholangitis
Date of designation: 12/11/2021, Positive, Last updated: 29/11/2022 -
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Orphan designation: 2-[1-(3-{6-[(1E)-(hydroxyimino)methyl]-5-methyl-4-oxo-7-propyl-3H,4H-pyrrolo[2,1-f][1,2,4]triazin-2-yl}-4-propoxybenzenesulfonyl)piperidin-4-yl]ethyl nitrate for: Treatment of systemic sclerosis
Date of designation: 12/11/2021, Positive, Last updated: 07/06/2022 -
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Orphan designation: (14S)-8-[3-(2-{dispiro[2.0.24.13]heptan-7-yl}ethoxy)-1H-pyrazol-1-yl]-12,12-dimethyl-2lamba6-thia-3,9,11,18,23-penta-azatetracyclo[17.3.1.111,14.05,10]tetracosa-1(22),5,7,9,19(23),20-hexaene-2,2,4-trione calcium salt hydrate, deutivacaftor, tezacaftor for: Treatment of cystic fibrosis
Date of designation: 12/11/2021, Positive, Last updated: 07/06/2022 -
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Orphan designation: D-lactic acid, glycolic acid for: Treatment of amyotrophic lateral sclerosis
Date of designation: 12/11/2021, Positive, Last updated: 07/06/2022 -
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Orphan designation: Nadunolimab for: Treatment of pancreatic cancer
Date of designation: 12/11/2021, Positive, Last updated: 08/06/2022 -
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Orphan designation: Mosunetuzumab for: Treatment of follicular lymphoma
Date of designation: 12/11/2021, Positive, Last updated: 23/06/2022 -
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Orphan designation: paclitaxel, polyoligo(ethylene glycol)methacrylate-co-poly(vinylbenzyldithiodibutyric acid-gemcitabine) for: Treatment of pancreatic cancer
Date of designation: 12/11/2021, Positive, Last updated: 08/06/2022 -
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Orphan designation: Autologous CD34+ enriched cells transduced with a self-inactivating lentiviral vector containing the codon-optimized RPS19 gene for: Treatment of Diamond-Blackfan anaemia
Date of designation: 12/11/2021, Positive, Last updated: 11/11/2022 -
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Orphan designation: Fingolimod for: Treatment of adrenoleukodystrophy
Date of designation: 12/11/2021, Positive, Last updated: 11/11/2022 -
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Orphan designation: Udonitrectag lysine for: Treatment in solid organ transplantation
Date of designation: 12/11/2021, Positive, Last updated: 08/06/2022 -
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Orphan designation: Cannabidiol, Dronabinol for: Treatment of complex regional pain syndrome
Date of designation: 12/11/2021, Positive, Last updated: 08/06/2022 -
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Orphan designation: 5-fluoro-4-(4-fluoro-2-methoxyphenyl)-N-{4-[(S-methylsulfonimidoyl)methyl] pyridin-2-yl}pyridin-2-amine for: Treatment of diffuse large B-cell lymphoma
Date of designation: 12/11/2021, Positive, Last updated: 09/06/2022 -
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Orphan designation: Rebastinib for: Treatment of ovarian cancer
Date of designation: 12/11/2021, Positive, Last updated: 07/02/2023 -
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Orphan designation: Ibrexafungerp for: Treatment of invasive candidiasis
Date of designation: 12/11/2021, Positive, Last updated: 09/06/2022 -
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Orphan designation: Soticlestat for: Treatment of Lennox-Gastaut syndrome
Date of designation: 12/11/2021, Positive, Last updated: 09/06/2022 -
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Orphan designation: Olverembatinib for: Treatment of chronic myeloid leukaemia
Date of designation: 12/11/2021, Positive, Last updated: 15/06/2022 -
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Orphan designation: Adeno-associated viral vector serotype 9 containing the human SURF1 gene for: Treatment of Leigh syndrome
Date of designation: 12/11/2021, Positive, Last updated: 15/06/2022