5
Orphan Designations
1
FDA Approvals
11
Rare Diseases
1
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Matthew-Wood syndrome | Korlym | Orphan Designation | - |
| amyotrophic lateral sclerosis | dazucorilant | Orphan Designation | - |
| anophthalmia/microphthalmia-esophageal atresia syndrome | Korlym | Orphan Designation | - |
| endogenous Cushing syndrome | relacorilant | Orphan Designation | - |
| isolated microphthalmia 2 | Korlym | Orphan Designation | - |
| leukodystrophy, hypomyelinating, 27 | Korlym | Orphan Designation | - |
| malignant pancreatic neoplasm | relacorilant | Orphan Designation | - |
| microphthalmia with brain and digit anomalies | Korlym | Orphan Designation | - |
| ovarian cancer | mifepristone | Orphan Designation | - |
| teeth, odd shapes of | Korlym | Orphan Designation | - |
| type 2 diabetes mellitus | MIFEPRISTONE | - | FDA Approved |