6
Orphan Designations
1
FDA Approvals
18
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Angelman syndrome | Hepzato | Orphan Designation | - |
| H syndrome | melphalan hydrochloride | Orphan Designation | - |
| Timothy syndrome | melphalan hydrochloride | Orphan Designation | - |
| Toriello-Lacassie-Droste syndrome | Hepzato | Orphan Designation | - |
| aortic valve stenosis | Hepzato | Orphan Designation | - |
| argininosuccinic aciduria | Hepzato | Orphan Designation | - |
| asthma, nasal polyps, and aspirin intolerance | Hepzato | Orphan Designation | - |
| carbon monoxide-induced delayed encephalopathy | melphalan hydrochlorideHepzato | Orphan Designation | - |
| cholangiocarcinoma | melphalan | Orphan Designation | - |
| congenital muscular dystrophy without intellectual disability | Hepzato | Orphan Designation | - |
| corpus callosum agenesis-intellectual disability-coloboma-micrognathia syndrome | Hepzato | Orphan Designation | - |
| cutaneous neuroendocrine carcinoma | melphalan hydrochloride | Orphan Designation | - |
| hepatocellular carcinoma | doxorubicin | Orphan Designation | - |
| leiomyomatosis | Hepzato | Orphan Designation | - |
| multisystemic smooth muscle dysfunction syndrome | melphalan hydrochloride | Orphan Designation | - |
| natal teeth-intestinal pseudoobstruction-patent ductus syndrome | melphalan hydrochloride | Orphan Designation | - |
| pancreatic neuroendocrine neoplasm | melphalan hydrochloride | Orphan Designation | - |
| pancreatic neuroendocrine tumor | melphalan hydrochloride | Orphan Designation | - |