4
Orphan Designations
0
FDA Approvals
18
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Down syndrome | Anascorp | Orphan Designation | - |
| acute disseminated encephalomyelitis | Hydroxocobalamin | Orphan Designation | - |
| acute flaccid myelitis | Hydroxocobalamin | Orphan Designation | - |
| acute lymphoblastic leukemia | Hydroxocobalamin | Orphan Designation | - |
| adult pleomorphic rhabdomyosarcoma | Anavip | Orphan Designation | - |
| black widow spider envenomation | Anavip | Orphan Designation | - |
| endogenous depression | Anascorp | Orphan Designation | - |
| extramedullary plasmacytoma | Anascorp | Orphan Designation | - |
| hypoparathyroidism | Synthetic Human Parathyroid Hormone 1-34 | Orphan Designation | - |
| intellectual disability, autosomal recessive 61 | Anascorp | Orphan Designation | - |
| pediatric acute-onset neuropsychiatric syndrome | Anavip | Orphan Designation | - |
| pediatric hepatocellular carcinoma | Anavip | Orphan Designation | - |
| pediatric ovarian dysgerminoma | Anavip | Orphan Designation | - |
| pediatric ovarian germ cell tumor | Anavip | Orphan Designation | - |
| pediatric-onset Graves disease | Anavip | Orphan Designation | - |
| refractory malignant neoplasm | Anascorp | Orphan Designation | - |
| scorpion envenomation | AnavipAnascorp | Orphan Designation | - |
| snakebite envenomation | Anavip | Orphan Designation | - |