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14 results
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Orphan designation: Refusal of orphan designation for the treatment of fibromyalgia
Date of refusal of designation: 07/11/2020, Negative, Last updated: 24/02/2020 -
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Orphan designation: (R)‑tetrahydrofuran‑3‑yl 4‑(6‑(5‑(4‑ethoxy‑1‑isopropylpiperidin‑4‑yl)pyridin-2-yl)pyrrolo[1,2-b]pyridazin-4-yl)piperazine-1-carboxylate sesquisuccinate for: Treatment of fibrodysplasia ossificans progressiva
Date of designation: 13/11/2020, Positive, Last updated: 15/02/2021 -
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Orphan designation: Zanidatamab for: Treatment of gastric cancer
Date of designation: 13/11/2020, Positive, Last updated: 13/10/2021 -
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Orphan designation: Alisitol, retinol palmitate, zinc gluconate for: Treatment of microvillus inclusion disease
Date of designation: 13/11/2020, Positive, Last updated: 15/02/2021 -
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Orphan designation: Anti-(pancreatic adenocarcinoma upregulated factor) IgG1 humanised monoclonal antibody for: Treatment of pancreatic cancer
Date of designation: 13/11/2020, Positive, Last updated: 15/02/2021 -
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Orphan designation: Autologous CD34+ cells transduced ex vivo with a lentiviral vector containing a modified gamma-globin gene for: Treatment of sickle cell disease
Date of designation: 13/11/2020, Withdrawn, Last updated: 16/09/2022 -
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Orphan designation: Tislelizumab for: Treatment of oesophageal cancer
Date of designation: 13/11/2020, Positive, Last updated: 14/12/2021 -
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Orphan designation: L-pyroglutamyl-L-asparaginyl-L-prolyl-D-tyrosyl-D-tryptophan amide for: Treatment of amyotrophic lateral sclerosis
Date of designation: 13/11/2020, Positive, Last updated: 15/02/2021 -
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Orphan designation: Adeno-associated viral vector serotype 9 expressing codon-optimized human GRN gene for: Treatment of frontotemporal dementia
Date of designation: 13/11/2020, Positive, Last updated: 15/02/2021 -
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Orphan designation: Triheptanoin for: Treatment of carnitine palmitoyltransferase I deficiency
Date of designation: 13/11/2020, Positive, Last updated: 15/02/2021 -
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Orphan designation: Perflubron for: Treatment of congenital pulmonary hypoplasia
Date of designation: 13/11/2020, Positive, Last updated: 16/09/2021 -
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Orphan designation: Autologous bone marrow derived CD34+ cells transduced ex vivo with a self-inactivating lentiviral vector containing a normal version of the coding region of the IL2RG gene for: Treatment of X-linked severe combined immunodeficiency
Date of designation: 13/11/2020, Positive, Last updated: 15/02/2021 -
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Orphan designation: 3,5-diamino-6-chloro-N-(N-(4-(4-(2-(hexyl((2S,3R,4R,5R)-2,3,4,5,6-pentahydroxyhexyl)amino)ethoxy)phenyl)butyl)-carbamimidoyl)pyrazine-2-carboxamide, Sodium chloride solution 4.2% (w/v) for: Treatment of primary ciliary dyskinesia
Date of designation: 16/11/2020, Positive, Last updated: 07/03/2023 -
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Orphan designation: DNA plasmid encoding human transferrin gene for: Treatment of retinitis pigmentosa
Date of designation: 13/11/2020, Positive, Last updated: 15/02/2021