4
Orphan Designations
0
FDA Approvals
9
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| 3-hydroxy-3-methylglutaric aciduria | citric acid | Orphan Designation | - |
| adult hepatocellular carcinoma | ezurpimtrostat | Orphan Designation | - |
| cholangiocarcinoma | ezurpimtrostat | Orphan Designation | - |
| cholecystitis | citric acid monohydrate | Orphan Designation | - |
| chronic beryllium disease | citric acid monohydrate | Orphan Designation | - |
| hepatocellular carcinoma | ezurpimtrostat | Orphan Designation | - |
| infantile liver failure syndrome 2 | citric acid | Orphan Designation | - |
| pediatric hepatocellular carcinoma | ezurpimtrostat | Orphan Designation | - |
| respiratory failure | citric acid monohydrate | Orphan Designation | - |