6
Orphan Designations
6
FDA Approvals
13
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| drug- or toxin-induced pulmonary arterial hypertension | Tyvaso | Orphan Designation | - |
| familial pulmonary arterial hypertension leucopenia and atrial septal defect | Tyvaso | Orphan Designation | - |
| heritable pulmonary arterial hypertension | Tyvaso | Orphan Designation | - |
| idiopathic pulmonary arterial hypertension | Tyvaso | Orphan Designation | - |
| idiopathic pulmonary fibrosis | Treprostinil | Orphan Designation | - |
| neuroblastoma | Unituxinmonoclonal antibody 3F8 | Orphan Designation | - |
| pediatric acute-onset neuropsychiatric syndrome | Unituxin | Orphan Designation | - |
| pediatric hepatocellular carcinoma | Unituxin | Orphan Designation | - |
| pediatric ovarian dysgerminoma | Unituxin | Orphan Designation | - |
| pediatric ovarian germ cell tumor | Unituxin | Orphan Designation | - |
| pediatric-onset Graves disease | Unituxin | Orphan Designation | - |
| pulmonary arterial hypertension | ORENITRAMralinepagTyvasoRemodulin;Orenitram | Orphan Designation | FDA Approved |
| pulmonary fibrosis | TYVASO DPI | - | FDA Approved |