7
Orphan Designations
0
FDA Approvals
23
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Guillain-Barre syndrome | immune globulin (human) | Orphan Designation | - |
| Langerhans cell histiocytosis | Wilate | Orphan Designation | - |
| amyopathic dermatomyositis | Octagam 10% | Orphan Designation | - |
| autosomal dominant Ehlers-Danlos syndrome, vascular type | Wilate | Orphan Designation | - |
| classical dermatomyositis | Octagam 10% | Orphan Designation | - |
| dermatomyositis | Octagam 10% | Orphan Designation | - |
| hypertensive urgency | Balfaxar | Orphan Designation | - |
| immune dysregulation, autoimmunity, and autoinflammation | human coagulation factor VIII | Orphan Designation | - |
| immune dysregulation-polyendocrinopathy-enteropathy-X-linked syndrome | human coagulation factor VIII | Orphan Designation | - |
| immune-mediated scleritis | human coagulation factor VIII | Orphan Designation | - |
| juvenile dermatomyositis | Octagam 10% | Orphan Designation | - |
| neonatal dermatomyositis | Octagam 10% | Orphan Designation | - |
| serous adenofibroma | Balfaxar | Orphan Designation | - |
| serous cystadenofibroma | Balfaxar | Orphan Designation | - |
| serous or mucinous cystadenoma of childhood | Balfaxar | Orphan Designation | - |
| serous otitis media | Balfaxar | Orphan Designation | - |
| serous surface papilloma | Balfaxar | Orphan Designation | - |
| stiff-person syndrome | Intravenous immune globulin (human) 10% | Orphan Designation | - |
| thrombocytopenia 12 with or without myopathy | Wilate | Orphan Designation | - |
| thrombotic thrombocytopenic purpura | solvent/detergent treated non-blood-group specific human coagulation active plasma | Orphan Designation | - |
| von Willebrand disease (hereditary or acquired) | Wilate | Orphan Designation | - |
| von Willebrand disease 2 | Wilate | Orphan Designation | - |
| von Willebrand disease 3 | Wilate | Orphan Designation | - |