2
Orphan Designations
1
FDA Approvals
10
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| acute myeloid leukemia | a humanized IgG4 monoclonal antibody targeting CD47 | Orphan Designation | - |
| adult-onset autosomal dominant demyelinating leukodystrophy | penpulimab-kcqx | Orphan Designation | - |
| cystic fibrosis | penpulimab-kcqx | Orphan Designation | - |
| dermatomyositis | penpulimab-kcqx | Orphan Designation | - |
| nasopharyngeal carcinoma | penpulimab-kcqx | Orphan Designation | - |
| nasopharyngeal carcinoma, susceptibility to, 1 | PENPULIMAB KCQX | - | FDA Approved |
| nasopharyngeal carcinoma, susceptibility to, 2 | PENPULIMAB KCQX | - | FDA Approved |
| nasopharyngeal carcinoma, susceptibility to, 3 | PENPULIMAB KCQX | - | FDA Approved |
| nut midline carcinoma | penpulimab-kcqx | Orphan Designation | - |
| polycystic kidney disease 2 | penpulimab-kcqx | Orphan Designation | - |