2
Orphan Designations
0
FDA Approvals
4
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| acute myeloid leukemia | 8-bromo-2-(1-methylpiperidin-4-yl) amino)-4-(4-phenoxyphenyl) amino) pyrido[4,3-d] pyrimidin-5(6H)-one hydrochloride | Orphan Designation | - |
| autoimmune thrombocytopenic purpura | Cevidoplenib | Orphan Designation | - |
| inborn error of immunity | Cevidoplenib | Orphan Designation | - |
| nephrotic syndrome 14 | Cevidoplenib | Orphan Designation | - |