2
Orphan Designations
0
FDA Approvals
7
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| acute myeloid leukemia | Autologous CD3+ T cells genetically modified using a non-viral system to express a CD33 CAR, membrane bound Interleukin-15, and a truncated form of human HER1t (kill switch) on the cell surface. | Orphan Designation | - |
| adult-onset autosomal dominant demyelinating leukodystrophy | Papzimeos | Orphan Designation | - |
| cystic fibrosis | Papzimeos | Orphan Designation | - |
| dermatomyositis | Papzimeos | Orphan Designation | - |
| nut midline carcinoma | Papzimeos | Orphan Designation | - |
| polycystic kidney disease 2 | Papzimeos | Orphan Designation | - |
| recurrent respiratory papillomatosis | Papzimeos | Orphan Designation | - |