8
Orphan Designations
1
FDA Approvals
20
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Gaucher disease type III | tazemetostat | Orphan Designation | - |
| Sandhoff disease, adult form | tazemetostat | Orphan Designation | - |
| acute myeloid leukemia | small molecule inhibitor of histone methyltransferase DOT1L | Orphan Designation | - |
| adult acute lymphoblastic leukemia | small molecule inhibitor of histone methyltransferase DOT1L | Orphan Designation | - |
| adult neuronal ceroid lipofuscinosis | Tazverik | Orphan Designation | - |
| adult pleomorphic rhabdomyosarcoma | Tazverik | Orphan Designation | - |
| adult-onset non-insulinoma persistent hyperinsulinemic hypoglycemia | Tazverik | Orphan Designation | - |
| adult-onset proximal spinal muscular atrophy, autosomal dominant | Tazverik | Orphan Designation | - |
| chordoma | tazemetostat | Orphan Designation | - |
| deafness, congenital, and adult-onset progressive leukoencephalopathy | tazemetostat | Orphan Designation | - |
| follicular lymphoma | Tazverik | Orphan Designation | - |
| mesothelioma | tazemetostat | Orphan Designation | - |
| multisystem inflammatory syndrome in children and adults | tazemetostat | Orphan Designation | - |
| nut midline carcinoma | tazemetostat | Orphan Designation | - |
| plasma cell myeloma | Small molecule inhibitor of the enzymatic activity of Su(var), Enhancer-of-zeste, Trithorax domain containing 2 (SETD2) | Orphan Designation | - |
| primary adult heart tumor | Tazverik | Orphan Designation | - |
| rhabdoid tumor | tazemetostat | Orphan Designation | - |
| rhabdoid tumor of the kidney | tazemetostat | Orphan Designation | - |
| rhabdoid tumor predisposition syndrome 1 | tazemetostat | Orphan Designation | - |
| soft tissue sarcoma | tazemetostat | Orphan Designation | - |