9
Orphan Designations
0
FDA Approvals
18
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| B-cell chronic lymphocytic leukemia | Breyanzi | Orphan Designation | - |
| Hodgkin's lymphoma, lymphocytic depletion | Breyanzi | Orphan Designation | - |
| MALT lymphoma | Breyanzi | 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 | - |
| diffuse large B-cell lymphoma | Breyanzi | Orphan Designation | - |
| extranodal nasal NK/T cell lymphoma | Breyanzi | Orphan Designation | - |
| follicular lymphoma | Breyanzi | Orphan Designation | - |
| folliculotropic mycosis fungoides | Breyanzi | Orphan Designation | - |
| lymphoma, non-Hodgkin, familial | Breyanzi | Orphan Designation | - |
| mycosis fungoides | Breyanzi | Orphan Designation | - |
| nodal marginal zone B-cell lymphoma | Breyanzi | Orphan Designation | - |
| plasma cell myeloma | Dual targeting chimeric antigen receptor (CAR) T cell therapy directed against B-cell maturation antigen (BCMA) and G-protein coupled receptor family C group 5 member D (GPRC5D)an investigational chimeric antigen receptor (CAR) T-cell product that consists of autologous T cells that have been transduced with a genetically engineered replication-incompetent, third-generation, self-inactivating lentiviral vector encoding a G protein-coupled receptor class C, group 5, member D (GPRC5D)-specific CAR | Orphan Designation | - |
| primary adult heart tumor | Breyanzi | Orphan Designation | - |
| primary mediastinal large B-cell lymphoma | Breyanzi | Orphan Designation | - |
| splenic marginal zone lymphoma | Breyanzi | Orphan Designation | - |