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 |