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

Table 1 Examples of FDA orphan drug designations

#	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

ISSN: 2041-1480