1
Orphan Designations
0
FDA Approvals
3
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Carney-Stratakis syndrome | Small-molecule oral KIT inhibitor targeting activating mutations and resistance mutations | Orphan Designation | - |
| colorectal gastrointestinal stromal tumor | Small-molecule oral KIT inhibitor targeting activating mutations and resistance mutations | Orphan Designation | - |
| gastrointestinal stromal tumor | Small-molecule oral KIT inhibitor targeting activating mutations and resistance mutations | Orphan Designation | - |