2
Orphan Designations
2
FDA Approvals
7
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| adult neuronal ceroid lipofuscinosis | Jaypirca | Orphan Designation | - |
| adult pleomorphic rhabdomyosarcoma | Jaypirca | Orphan Designation | - |
| adult-onset non-insulinoma persistent hyperinsulinemic hypoglycemia | Jaypirca | Orphan Designation | - |
| adult-onset proximal spinal muscular atrophy, autosomal dominant | Jaypirca | Orphan Designation | - |
| malignant pancreatic neoplasm | 6-(2-hydroxy-2-methylpropoxy)-4-(6-(6-((6-methoxypyridin-3-yl)methyl)-3,6-diazabicyclo[3.1.1]heptan-3-yl)pyridin-3-yl)pyrazolo[1,5-a]pyridine-3-carbonitrile | Orphan Designation | - |
| mantle cell lymphoma | Jaypirca | Orphan Designation | - |
| primary adult heart tumor | Jaypirca | Orphan Designation | - |