5
Orphan Designations
0
FDA Approvals
10
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| adult-onset non-insulinoma persistent hyperinsulinemic hypoglycemia | Macrelin | Orphan Designation | - |
| isolated congenital growth hormone deficiency | Macrelin | Orphan Designation | - |
| isolated growth hormone deficiency type IA | Macrelin | Orphan Designation | - |
| isolated growth hormone deficiency type IB | Macrelin | Orphan Designation | - |
| isolated growth hormone deficiency type II | Macrelin | Orphan Designation | - |
| neuroblastoma | perifosine | Orphan Designation | - |
| ovarian cancer | AEZS-108 (LHRH-agonist linked to doxorubicin) | Orphan Designation | - |
| plasma cell myeloma | perifosine | Orphan Designation | - |
| primary adult heart tumor | Macrelin | Orphan Designation | - |
| renal cell carcinoma | antiangiogenic components extracted from marine cartilage | Orphan Designation | - |