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Table 1 Examples of FDA orphan drug designations

From: An integrative knowledge graph for rare diseases, derived from the Genetic and Rare Diseases Information Center (GARD)

# Generic Name Orphan Designation Designation Date Designation Status
1 ((1r, 4r)-N1-(2-benzyl-7-(2-methyl-2H-tetrazol-5-yl)-9H-pyrimido[4,5-b]indol-4-yl)cyclohexane-1,4-diamine dihydrobromide dihydrate)-Expanded cord blood Prevention of Graft-versus-Host-Disease 12/13/2018 Designated
2 ((4-(3-benzyl-4-hydroxybenzyl)-3,5-dimethylphenoxy)methyl)phosphonic acid Treatment of X-linked adrenoleukodystrophy 12/05/2016 Designated
3 (+)-(2S)-2-(4-chloro-2-methoxyphenyl)-2-{[3-methoxy-5-(methylsulfonyl)phenyl]amino}-1-[5-(trifluoromethoxy)-1H-indol-3-yl]ethanone Treatment of dengue virus infection 12/26/2017 Designated