5
Orphan Designations
0
FDA Approvals
13
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| acute lymphoblastic leukemia | autologous CD4+/CD8+ T cells expressing a CD19 chimeric antigen receptor and a truncated epidermal growth factor receptorautologous CD4+/CD8+ T cells transduced with lentiviral vector containing a chimeric antigen receptor directed against CD19 | Orphan Designation | - |
| acute lymphoblastic leukemia congenital sporadic aniridia | autologous CD4+/CD8+ T cells transduced with lentiviral vector containing a chimeric antigen receptor directed against CD19 | Orphan Designation | - |
| adult acute lymphoblastic leukemia | Autologous CD3+ T cells transduced with retroviral vector containing a chimeric antigen receptor directed against CD19 (Autologous CD3+ T cells containing CD19 chimeric antigen receptor) | Orphan Designation | - |
| adult neuronal ceroid lipofuscinosis | Breyanzi | Orphan Designation | - |
| adult pleomorphic rhabdomyosarcoma | Breyanzi | Orphan Designation | - |
| adult-onset non-insulinoma persistent hyperinsulinemic hypoglycemia | Breyanzi | Orphan Designation | - |
| adult-onset proximal spinal muscular atrophy, autosomal dominant | Breyanzi | Orphan Designation | - |
| childhood acute lymphoblastic leukemia | autologous CD4+/CD8+ T cells transduced with lentiviral vector containing a chimeric antigen receptor directed against CD19 | Orphan Designation | - |
| indolent plasma cell myeloma | Autologous CD4+/CD8+ T cells expressing a BCMA-specific chimeric antigen receptor (CAR) | Orphan Designation | - |
| mantle cell lymphoma | Breyanzi | Orphan Designation | - |
| osteosclerotic plasma cell myeloma | Autologous CD4+/CD8+ T cells expressing a BCMA-specific chimeric antigen receptor (CAR) | Orphan Designation | - |
| plasma cell myeloma | Autologous CD4+/CD8+ T cells expressing a BCMA-specific chimeric antigen receptor (CAR) | Orphan Designation | - |
| primary adult heart tumor | Breyanzi | Orphan Designation | - |