3
Orphan Designations
0
FDA Approvals
8
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| acute myeloid leukemia | cereblon enzyme 3 (E3) ligase modulating agent | Orphan Designation | - |
| juvenile myelomonocytic leukemia | Vidaza | Orphan Designation | - |
| pediatric acute-onset neuropsychiatric syndrome | Vidaza | Orphan Designation | - |
| pediatric hepatocellular carcinoma | Vidaza | Orphan Designation | - |
| pediatric ovarian dysgerminoma | Vidaza | Orphan Designation | - |
| pediatric ovarian germ cell tumor | Vidaza | Orphan Designation | - |
| pediatric-onset Graves disease | Vidaza | Orphan Designation | - |
| plasma cell myeloma | iberdomide | Orphan Designation | - |