2
Orphan Designations
1
FDA Approvals
7
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| T-cell non-Hodgkin lymphoma | tipifarnib | Orphan Designation | - |
| acute myeloid leukemia | Komzifti | Orphan Designation | - |
| adult neuronal ceroid lipofuscinosis | Komzifti | Orphan Designation | - |
| adult pleomorphic rhabdomyosarcoma | Komzifti | Orphan Designation | - |
| adult-onset non-insulinoma persistent hyperinsulinemic hypoglycemia | Komzifti | Orphan Designation | - |
| adult-onset proximal spinal muscular atrophy, autosomal dominant | Komzifti | Orphan Designation | - |
| primary adult heart tumor | Komzifti | Orphan Designation | - |