4
Orphan Designations
2
FDA Approvals
18
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Char syndrome | CABOMETYX | Orphan Designation | - |
| VIPoma | Cabometyx | Orphan Designation | - |
| carbon monoxide-induced delayed encephalopathy | CABOMETYX | Orphan Designation | - |
| deafness, congenital, and adult-onset progressive leukoencephalopathy | CabometyxCometriq | Orphan Designation | - |
| ectopic Cushing syndrome | Cabometyx | Orphan Designation | - |
| follicular bronchiolits | Cometriq | Orphan Designation | - |
| follicular dendritic cell sarcoma | Cometriq | Orphan Designation | - |
| follicular lymphoma | Cometriq | Orphan Designation | - |
| hepatocellular carcinoma | CABOMETYX | Orphan Designation | - |
| multisystemic smooth muscle dysfunction syndrome | CABOMETYX | Orphan Designation | - |
| myasthenic syndrome, congenital, 22 | recombinant humanized monoclonal antibody targeting the anti-müllerian hormone receptor II | Orphan Designation | - |
| natal teeth-intestinal pseudoobstruction-patent ductus syndrome | CABOMETYX | Orphan Designation | - |
| non-functional pancreatic neuroendocrine tumor | Cabometyx | Orphan Designation | - |
| pancreatic neuroendocrine neoplasm | Cabometyx | Orphan Designation | - |
| pancreatic neuroendocrine tumor | Cabometyx | Orphan Designation | - |
| thyroid cancer, nonmedullary, 2 | Cometriq | Orphan Designation | - |
| thyroid gland mixed medullary and follicular cell-derived carcinoma | Cometriq | Orphan Designation | - |
| thyroid hormone resistance, generalized, autosomal recessive | recombinant humanized monoclonal antibody targeting the anti-müllerian hormone receptor II | Orphan Designation | - |