11
Orphan Designations
1
FDA Approvals
24
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| B-cell chronic lymphocytic leukemia | Ricin (blocked) conjugated murine MCA (anti-B4) | Orphan Designation | - |
| B-cell neoplasm | Ricin (blocked) conjugated murine MCA (anti-B4) | Orphan Designation | - |
| B-cell prolymphocytic leukemia | Ricin (blocked) conjugated murine MCA (anti-B4) | Orphan Designation | - |
| Bloom syndrome | Ricin (blocked) conjugated murine monoclonal antibody (CD6) | Orphan Designation | - |
| Wolf-Hirschhorn syndrome | Ricin (blocked) conjugated murine mca (anti-b4) | Orphan Designation | - |
| acute myeloid leukemia | Indolino-benzodiazepine compound (sulfonated DGN462) conjugated to the humanized anti-CD33 monoclonal antibody (Z4681A) via the sulfo-SPDB linker | Orphan Designation | - |
| adult neuronal ceroid lipofuscinosis | Elahere | Orphan Designation | - |
| adult pleomorphic rhabdomyosarcoma | Elahere | Orphan Designation | - |
| adult-onset non-insulinoma persistent hyperinsulinemic hypoglycemia | Elahere | Orphan Designation | - |
| adult-onset proximal spinal muscular atrophy, autosomal dominant | Elahere | Orphan Designation | - |
| distal myopathy with vocal cord weakness | Ricin (blocked) conjugated murine monoclonal antibody (CD6) | Orphan Designation | - |
| double nail for fifth toe | Ricin (blocked) conjugated murine mca (anti-b4) | Orphan Designation | - |
| hemoglobin--variants for which the chain carrying the mutation 1S unknown or uncertain | Ricin (blocked) conjugated murine mca (anti-b4) | Orphan Designation | - |
| indolent primary cutaneous T-cell lymphoma | Ricin (blocked) conjugated murine monoclonal antibody (CD6) | Orphan Designation | - |
| mismatch repair cancer syndrome 1 | Ricin (blocked) conjugated murine monoclonal antibody (CD6) | Orphan Designation | - |
| myeloid leukemia | Ricin (blocked) conjugated murine MCA (anti-my9) | Orphan Designation | - |
| ovarian cancer | Elahere | Orphan Designation | - |
| plasma cell myeloma | maytansinoid DM1-conjugated humanized monoclonal antibody N901 | Orphan Designation | - |
| primary adult heart tumor | Elahere | Orphan Designation | - |
| primary cutaneous T-cell lymphoma | Ricin (blocked) conjugated murine monoclonal antibody (CD6) | Orphan Designation | - |
| primary cutaneous T-cell non-Hodgkin lymphoma | Ricin (blocked) conjugated murine monoclonal antibody (CD6) | Orphan Designation | - |
| small cell lung carcinoma | Maytansinoid DM-1 Conjugated Humanized Monoclonal Antibody N901Ricin (blocked) conjugated murine MCA (n901) | Orphan Designation | - |
| trabecular adenocarcinoma | maytansinoid DM1-conjugated humanized monoclonal antibody N901 | Orphan Designation | - |
| urethral obstruction sequence | Ricin (blocked) conjugated murine MCA (anti-my9) | Orphan Designation | - |