5
Orphan Designations
1
FDA Approvals
4
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Becker muscular dystrophy | VILTEPSO | Orphan Designation | - |
| Duchenne muscular dystrophy | VILTEPSOall-P-ambo- [2',3'-Azanediyl-P,2',3'-trideoxy-P-(dimethylamino)-2',3'-seco](2'-N 5')(TCGGTAAGTTCTGAGATGGCATTTC)Brogidirsen | Orphan Designation | FDA Approved |
| eosinophilic granulomatosis with polyangiitis | small molecule, selective Janus kinase (JAK) 1 inhibitor | Orphan Designation | - |
| myelofibrosis | N-[(1S)-1-(4- fluorophenyl)ethyl]-4-(1-methyl-1H-pyrazol-4-yl)-N'-(pyrazin-2-yl)pyridine-2,6- diamine maleate | Orphan Designation | - |