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20 results
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Orphan designation: Autologous CD4+ and CD8+ T cells genetically modified with a lentiviral vector encoding a B-cell maturation antigen-specific chimeric antigen receptor for: Treatment of multiple myeloma
Date of designation: 09/12/2020, Withdrawn, Last updated: 26/05/2021 -
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Orphan designation: 4-[(3S)-3-aminopyrrolidin-1-yl]-6-cyano-5-(3,5-difluorophenyl)-N-[(2S)-1,1,1-trifluoropropan-2-yl]pyridine-3-carboxamide for: Treatment of congenital hyperinsulinism
Date of designation: 09/12/2020, Positive, Last updated: 27/05/2021 -
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Orphan designation: perflubron for: Treatment of respiratory distress syndrome
Date of designation: 09/12/2020, Positive, Last updated: 16/09/2021 -
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Orphan designation: Sulindac for: Treatment of fragile X syndrome
Date of designation: 09/12/2020, Positive, Last updated: 12/10/2021 -
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Orphan designation: Adeno-associated virus serotype rh74 containing the human sarcoglycan beta gene for: Treatment of limb-girdle muscular dystrophy
Date of designation: 09/12/2020, Positive, Last updated: 19/05/2021 -
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Orphan designation: Human interleukin 12 fused with immunoglobulin G4 C-terminal Fc fragment for: Treatment of glioma
Date of designation: 09/12/2020, Positive, Last updated: 19/05/2021 -
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Orphan designation: sotatercept for: Treatment of pulmonary arterial hypertension
Date of designation: 09/12/2020, Positive, Last updated: 26/04/2022 -
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Orphan designation: Aspacytarabine for: Treatment of acute myeloid leukaemia
Date of designation: 09/12/2020, Positive, Last updated: 21/05/2021 -
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Orphan designation: Humanised IgG1 monoclonal antibody against the extracellular domain of receptor tyrosine kinase-like orphan receptor 1 coupled via a proteolytically cleavable maleimidocaproyl-valine-citrulline-para-aminobenzoate linker to monomethyl auristatin E for: Treatment of mantle cell lymphoma
Date of designation: 09/12/2020, Positive, Last updated: 25/05/2021 -
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Orphan designation: Trametinib dimethyl sulfoxide for: Treatment of glioma
Date of designation: 09/12/2020, Positive, Last updated: 25/05/2021 -
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Orphan designation: 2'-O-(2-methoxyethyl) phosphorothioate antisense oligonucleotide targeting CD49d RNA for: Treatment of Duchenne muscular dystrophy
Date of designation: 09/12/2020, Positive, Last updated: 25/05/2021 -
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Orphan designation: 2’-O-(2-methoxyethyl) modified antisense oligonucleotide targeting glycogen synthase 1 pre‑mRNA for: Treatment of progressive myoclonic epilepsy type 2 (Lafora disease)
Date of designation: 09/12/2020, Positive, Last updated: 27/05/2021 -
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Orphan designation: Adeno-associated viral vector serotype 9 encoding human ATP7B for: Treatment of Wilson's disease
Date of designation: 09/12/2020, Positive, Last updated: 27/05/2021 -
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Orphan designation: Synthetic oligonucleotide selectively targeting UBE3A antisense RNA transcripts for: Treatment of Angelman syndrome
Date of designation: 09/12/2020, Positive, Last updated: 27/05/2021 -
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Orphan designation: erlotinib for: Treatment of patients with Olmsted syndrome
Date of designation: 09/12/2020, Positive, Last updated: 11/11/2022 -
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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: (S)-N-(5-(4-(1-(benzo[d][1,3]dioxol-5-yl)ethyl)piperazin-1-yl)-1,3,4-thiadiazol-2-yl)acetamide, hydrochloride salt for: Treatment of progressive supranuclear palsy
Date of designation: 09/12/2020, Positive, Last updated: 27/06/2023 -
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Orphan designation: (R)-3-(1-(2,3-dichloro-4-(pyrazin-2-yl)phenyl)-2,2,2-trifluoroethyl)-1-methyl-1-(1-methylpiperidin-4-yl)urea fumarate for: Treatment of Prader-Willi syndrome
Date of designation: 09/12/2020, Positive, Last updated: 19/05/2021 -
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Orphan designation: Dabrafenib mesylate for: Treatment of glioma (updated)
Date of designation: 09/12/2020, Positive, Last updated: 15/09/2023