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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
Date of designation: 24/03/2020, Positive, Last updated: 07/04/2021 -
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Orphan designation: Fosgemcitabine palabenamide for: Treatment of biliary tract cancer
Date of designation: 24/03/2020, Positive, Last updated: 12/08/2020 -
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Orphan designation: Losmapimod for: treatment of facioscapulohumeral muscular dystrophy
Date of designation: 24/03/2020, Positive, Last updated: 12/08/2020 -
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Orphan designation: Trifarotene for: Treatment of autosomal recessive congenital ichthyosis
Date of designation: 24/03/2020, Withdrawn, Last updated: 07/06/2022 -
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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Date of designation: 24/03/2020, Withdrawn, Last updated: 16/02/2023 -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: Asciminib for: Treatment of chronic myeloid leukaemia
Date of designation: 24/03/2020, Positive, Last updated: 07/09/2022