3
Orphan Designations
0
FDA Approvals
8
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| B-cell chronic lymphocytic leukemia | Calquence | Orphan Designation | - |
| Waldenstrom macroglobulinemia | acalabrutinib | Orphan Designation | - |
| adult neuronal ceroid lipofuscinosis | CALQUENCECalquence | Orphan Designation | - |
| adult pleomorphic rhabdomyosarcoma | CALQUENCECalquence | Orphan Designation | - |
| adult-onset non-insulinoma persistent hyperinsulinemic hypoglycemia | CALQUENCECalquence | Orphan Designation | - |
| adult-onset proximal spinal muscular atrophy, autosomal dominant | CALQUENCECalquence | Orphan Designation | - |
| mantle cell lymphoma | CALQUENCE | Orphan Designation | - |
| primary adult heart tumor | CALQUENCECalquence | Orphan Designation | - |