5
Orphan Designations
0
FDA Approvals
8
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| T-cell non-Hodgkin lymphoma | 1-{6-[(4-fluorophenyl)methyl]-5-(hydroxymethyl)-3,3-dimethyl-1H,2H,3H-pyrrolo[3,2-b]pyridin-1-yl}-2-[(2R,5R)-5-methyl-2-{[(3R)-3-methylmorpholin-4-yl]methyl}piperazin-1-yl]ethan-1-one, L-(+)-lactic acid salt | Orphan Designation | - |
| acute myeloid leukemia | guadecitabine1-Cyclopropyl-3-[3-(5-morpholin-4-ylmethyl-1H-benzoimidazol-2-yl)-1H-pyrazol-4-yl]-urea | Orphan Designation | - |
| acute myeloid leukemia and myelodysplastic syndromes related to radiation | Inqovi | Orphan Designation | - |
| adult pleomorphic rhabdomyosarcoma | Inqovi | Orphan Designation | - |
| adult-onset non-insulinoma persistent hyperinsulinemic hypoglycemia | Inqovi | Orphan Designation | - |
| myelodysplastic syndrome | guadecitabine SCInqovi | Orphan Designation | - |
| myelodysplastic/myeloproliferative neoplasm | Inqovi | Orphan Designation | - |
| primary adult heart tumor | Inqovi | Orphan Designation | - |