1
Orphan Designations
3
FDA Approvals
4
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| X-linked cerebral adrenoleukodystrophy | Skysona | - | FDA Approved |
| beta-thalassemia major | Zynteglo | - | FDA Approved |
| plasma cell myeloma | autologous T lymphocyte enriched population cultured in the rpesence of phosphatidylinositol 3-kinase inhibitor (bb007) that contains cells transduced with an anti-human B cell maturation antigen (BCMA02) chimeric antigen receptor (CAR) lentiviral vector | Orphan Designation | - |
| sickle cell disease | Lyfgenia | - | FDA Approved |