3
Orphan Designations
0
FDA Approvals
7
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Waldenstrom macroglobulinemia | oprozomib | Orphan Designation | - |
| adult neuronal ceroid lipofuscinosis | Kyprolis | Orphan Designation | - |
| adult pleomorphic rhabdomyosarcoma | Kyprolis | Orphan Designation | - |
| adult-onset non-insulinoma persistent hyperinsulinemic hypoglycemia | Kyprolis | Orphan Designation | - |
| adult-onset proximal spinal muscular atrophy, autosomal dominant | Kyprolis | Orphan Designation | - |
| plasma cell myeloma | oprozomibKyprolis | Orphan Designation | - |
| primary adult heart tumor | Kyprolis | Orphan Designation | - |