3
Orphan Designations
0
FDA Approvals
7
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| achondroplasia | Infigratinib | Orphan Designation | - |
| adult embryonal tumor with multilayered rosettes, c19mc-altered | Truseltiq | Orphan Designation | - |
| adult-onset chronic progressive external ophthalmoplegia with mitochondrial myopathy | Truseltiq | Orphan Designation | - |
| cataract 13 with adult I phenotype | Truseltiq | Orphan Designation | - |
| cholangiocarcinoma | Truseltiq | Orphan Designation | - |
| hypochondroplasia | infigratinib | Orphan Designation | - |
| nut midline carcinoma | Truseltiq | Orphan Designation | - |