9
Orphan Designations
0
FDA Approvals
13
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Friedreich ataxia | coenzyme Q10 and d-alpha-tocopherol | Orphan Designation | - |
| acute myeloid leukemia | menatetrenone | Orphan Designation | - |
| acute promyelocytic leukemia | menatetrenone | Orphan Designation | - |
| adult hepatocellular carcinoma | menatetrenone | Orphan Designation | - |
| hepatocellular carcinoma | menatetrenone | Orphan Designation | - |
| inborn mitochondrial metabolism disorder | cytochrome C, flavin mononucleotide and thiamin diphosphate | Orphan Designation | - |
| malignant pancreatic neoplasm | met-enkephalin; Met-N | Orphan Designation | - |
| mitochondrial substrate carrier disorder | cytochrome C, flavin mononucleotide and thiamin diphosphate | Orphan Designation | - |
| myelodysplastic syndrome | menatetrenone and Vitamin D3menatetrenone | Orphan Designation | - |
| pediatric hepatocellular carcinoma | menatetrenone | Orphan Designation | - |
| pyridoxal phosphate-responsive seizures | pyridoxine; vitamin B6 | Orphan Designation | - |
| pyridoxine-dependent epilepsy | pyridoxine; vitamin B6 | Orphan Designation | - |
| pyridoxine-responsive sideroblastic anemia | pyridoxine; vitamin B6 | Orphan Designation | - |