4
Orphan Designations
1
FDA Approvals
21
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Grant syndrome | Emapalumab | Orphan Designation | - |
| autoimmune thrombocytopenic purpura | avatrombopag maleate | Orphan Designation | - |
| cellular phase chronic idiopathic myelofibrosis | Vonjo | Orphan Designation | - |
| chemotherapy-induced alopecia | avatrombopag maleate | Orphan Designation | - |
| chemotherapy-induced hypertension | avatrombopag maleate | Orphan Designation | - |
| chemotherapy-induced toxicity | avatrombopag maleate | Orphan Designation | - |
| dermatomyositis | Vonjo | Orphan Designation | - |
| essential thrombocythemia | Vonjo | Orphan Designation | - |
| graft versus host disease | Emapalumab | Orphan Designation | - |
| platelet-type bleeding disorder 20 | avatrombopag maleate | Orphan Designation | - |
| post-thrombotic syndrome | Vonjo | Orphan Designation | - |
| post-traumatic epilepsy | Vonjo | Orphan Designation | - |
| post-traumatic stress disorder | Vonjo | Orphan Designation | - |
| post-vaccinal encephalitis | Vonjo | Orphan Designation | - |
| primary biliary cholangitis | Vonjo | Orphan Designation | - |
| primary cutaneous lymphoma | Vonjo | Orphan Designation | - |
| primary myelofibrosis | Vonjo | Orphan Designation | - |
| radioulnar synostosis with amegakaryocytic thrombocytopenia 1 | avatrombopag maleate | Orphan Designation | - |
| radioulnar synostosis with amegakaryocytic thrombocytopenia 2 | avatrombopag maleate | Orphan Designation | - |
| thrombocytopenia 4 | avatrombopag maleate | Orphan Designation | - |
| thrombocytopenia 5 | avatrombopag maleate | Orphan Designation | - |