2
Orphan Designations
0
FDA Approvals
11
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| adult neuronal ceroid lipofuscinosis | Abecma | Orphan Designation | - |
| adult pleomorphic rhabdomyosarcoma | Abecma | Orphan Designation | - |
| adult-onset non-insulinoma persistent hyperinsulinemic hypoglycemia | Abecma | Orphan Designation | - |
| adult-onset proximal spinal muscular atrophy, autosomal dominant | Abecma | Orphan Designation | - |
| anemia | Reblozyl | Orphan Designation | - |
| anemia, congenital hypoplastic, with multiple congenital anomalies/intellectual disability syndrome | Reblozyl | Orphan Designation | - |
| anemia, hypochromic microcytic with iron overload | Reblozyl | Orphan Designation | - |
| myelodysplastic syndrome | Reblozyl | Orphan Designation | - |
| pancreatic insulin-producing neuroendocrine tumor | Reblozyl | Orphan Designation | - |
| plasma cell myeloma | Abecma | Orphan Designation | - |
| primary adult heart tumor | Abecma | Orphan Designation | - |