4
Orphan Designations
0
FDA Approvals
11
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Noonan syndrome 1 | recombinant human fibrinogen | Orphan Designation | - |
| activated PI3K-delta syndrome | Joenja | Orphan Designation | - |
| arthrogryposis, renal dysfunction, and cholestasis 1 | recombinant human fibrinogen | Orphan Designation | - |
| hemorrhagic disease of newborn | recombinant human fibrinogen | Orphan Designation | - |
| maternally-inherited Leigh syndrome | 2-isopropyl-3H-naphtho[1,2-d]imidazole-4,5-dione | Orphan Designation | - |
| maternally-inherited diabetes and deafness | 2-isopropyl-3H-naphtho[1,2-d]imidazole-4,5-dione | Orphan Designation | - |
| maternally-inherited mitochondrial dystonia | 2-isopropyl-3H-naphtho[1,2-d]imidazole-4,5-dione | Orphan Designation | - |
| medulloblastoma SHH activated | Joenja | Orphan Designation | - |
| medulloblastoma SHH activated and TP53 mutant | Joenja | Orphan Designation | - |
| medulloblastoma WNT activated | Joenja | Orphan Designation | - |
| mitochondrial disease | Methyl 4-{[2-(acetamino)ethyl]sulfanyl}-4-oxobutanoate | Orphan Designation | - |