9
Orphan Designations
0
FDA Approvals
18
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Ewing sarcoma | padnarsertib | Orphan Designation | - |
| acute myeloid leukemia | selinexor | Orphan Designation | - |
| adult glioblastoma | Selinexor | Orphan Designation | - |
| adult neuronal ceroid lipofuscinosis | Xpovio | Orphan Designation | - |
| adult pleomorphic rhabdomyosarcoma | Xpovio | Orphan Designation | - |
| adult-onset non-insulinoma persistent hyperinsulinemic hypoglycemia | Xpovio | Orphan Designation | - |
| adult-onset proximal spinal muscular atrophy, autosomal dominant | Xpovio | Orphan Designation | - |
| alveolar rhabdomyosarcoma | padnarsertib | Orphan Designation | - |
| diffuse large B-cell lymphoma | Xpovio | Orphan Designation | - |
| glioblastoma | Selinexor | Orphan Designation | - |
| mesenchymal glioblastoma | Selinexor | Orphan Designation | - |
| myelodysplastic syndrome | Eltanexor | Orphan Designation | - |
| myelofibrosis | Selinexor | Orphan Designation | - |
| plasma cell myeloma | Xpovio | Orphan Designation | - |
| primary adult heart tumor | Xpovio | Orphan Designation | - |
| rhabdomyosarcoma | padnarsertib | Orphan Designation | - |
| rhabdomyosarcoma of the cervix uteri | padnarsertib | Orphan Designation | - |
| soft tissue sarcoma | selinexor | Orphan Designation | - |