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Orphan designations
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Last updated 14 days ago
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, deutivaca
14 days ago
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 Treatment of cystic fibrosis...
Orphan designation: diflunisal Treatment of ATTR amyloidosis, 21/06/2022 Positive
14 days ago
Orphan designation: diflunisal Treatment of ATTR amyloidosis, 21/06/2022 Positive
Orphan designation: Zanidatamab Treatment of biliary tract cancer, 19/07/2021 Positive
14 days ago
Orphan designation: Zanidatamab Treatment of biliary tract cancer, 19/07/2021 Positive
Orphan designation: (S)-2-amino-6-(2-hydroxypropanoyl)-7,8-dihydropteridin-4(3H)-one Treatment of hyperphenylalaninemia, 20/05/2021 Positive
14 days ago
Orphan designation: (S)-2-amino-6-(2-hydroxypropanoyl)-7,8-dihydropteridin-4(3H)-one Treatment of hyperphenylalaninemia, 20/05/2021 Positive
Orphan designation: Expanded human allogeneic mesenchymal adult stem cells extracted from adipose tissue (darvadstrocel) Treatment of anal fistula, 08/10/2009 Withdrawn
15 days ago
Orphan designation: Expanded human allogeneic mesenchymal adult stem cells extracted from adipose...
Orphan designation: Unesbulin Treatment of soft tissue sarcoma, 10/12/2021 Withdrawn
about 1 month ago
Orphan designation: Unesbulin Treatment of soft tissue sarcoma, 10/12/2021 Withdrawn
Orphan designation: Alrefimotide acetate,riletamotide acetate,tapderimotide acetate Treatment of mesothelioma, 21/03/2024 Withdrawn
about 1 month ago
Orphan designation: Alrefimotide acetate,riletamotide acetate,tapderimotide acetate Treatment of mesothelioma, 21/03/2024 Withdrawn
Orphan designation: Glofitamab Treatment of diffuse large B-cell lymphoma, 15/10/2021 Withdrawn
about 1 month ago
Orphan designation: Glofitamab Treatment of diffuse large B-cell lymphoma, 15/10/2021 Withdrawn
Orphan designation: single guide RNA containing a sequence complementary to human ALB locus gene, intron 1, target region, Ziclumeran Treatment of haemophilia B, 25/03/2025 Positive
about 1 month ago
Orphan designation: single guide RNA containing a sequence complementary to human ALB...
Orphan designation: povetacicept Treatment of primary IgA nephropathy, 25/03/2025 Positive
about 1 month ago
Orphan designation: povetacicept Treatment of primary IgA nephropathy, 25/03/2025 Positive
Orphan designation: extract from Cannabis flower, containing high levels of cannabidiolic acid and <0.3% of tetrahydrocannabinol, Extraction solvent: olive oil, virgin Treatment of Rett syndrome, 25/03/2025 Positive
about 1 month ago
Orphan designation: extract from Cannabis flower, containing high levels of cannabidiolic acid...
Orphan designation: allopurinol Treatment of Marfan syndrome, 25/03/2025 Positive
about 1 month ago
Orphan designation: allopurinol Treatment of Marfan syndrome, 25/03/2025 Positive