3
Orphan Designations
1
FDA Approvals
8
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| B-cell chronic lymphocytic leukemia | COPIKTRA | Orphan Designation | - |
| T-cell non-Hodgkin lymphoma | duvelisib | Orphan Designation | - |
| adult neuronal ceroid lipofuscinosis | COPIKTRA | Orphan Designation | - |
| adult pleomorphic rhabdomyosarcoma | COPIKTRA | Orphan Designation | - |
| adult-onset non-insulinoma persistent hyperinsulinemic hypoglycemia | COPIKTRA | Orphan Designation | - |
| adult-onset proximal spinal muscular atrophy, autosomal dominant | COPIKTRA | Orphan Designation | - |
| follicular lymphoma | COPIKTRA | Orphan Designation | - |
| primary adult heart tumor | COPIKTRA | Orphan Designation | - |