10
Orphan Designations
0
FDA Approvals
16
Rare Diseases
1
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| T-cell non-Hodgkin lymphoma | Zolinza | Orphan Designation | - |
| X-linked reticulate pigmentary disorder | Zolinza | Orphan Designation | - |
| acute myeloid leukemia | bomedemstat | Orphan Designation | - |
| advanced heart failure | Temozolomide | Orphan Designation | - |
| advanced sleep phase syndrome | Temozolomide | Orphan Designation | - |
| advanced sleep phase syndrome 3 | Temozolomide | Orphan Designation | - |
| cutaneous larva migrans | Zolinza | Orphan Designation | - |
| cutaneous leishmaniasis | Zolinza | Orphan Designation | - |
| essential thrombocythemia | bomedemstat | Orphan Designation | - |
| juvenile idiopathic arthritis | rofecoxib | Orphan Designation | - |
| mesothelioma | vorinostat | Orphan Designation | - |
| myelofibrosis | bomedemstat | Orphan Designation | - |
| plasma cell myeloma | Trispecific T cell activating construct targeting BCMA, HSA, and CD3 | Orphan Designation | - |
| primary cutaneous lymphoma | Zolinza | Orphan Designation | - |
| progressive supranuclear palsy | competetive small molecule inhibitor of the enzyme O-GlcNAcase | Orphan Designation | - |
| skin disorder | Zolinza | Orphan Designation | - |