4
Orphan Designations
1
FDA Approvals
6
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Alstrom syndrome | hexasodium salt of myo-inositol hexaphosphate (IP6, phytate) hexasodium phytate | Orphan Designation | - |
| Bardet-Biedl syndrome 2 | hexasodium salt of myo-inositol hexaphosphate (IP6, phytate) hexasodium phytate | Orphan Designation | - |
| Williams syndrome | hexasodium salt of myo-inositol hexaphosphate (IP6, phytate) hexasodium phytate | Orphan Designation | - |
| beta-thalassemia HBB/LCRB | 2-(2-{[2-(1H-benzimidazol-2-yl)ethyl]amino}ethyl)-N-[(3-fluoropyridin-2-yl)methyl]-1,3-oxazole-4-carboxamide trihydrochloride | Orphan Designation | - |
| calciphylaxis | hexasodium phytate | Orphan Designation | - |
| sickle cell disease | ferroportin inhibitor | Orphan Designation | - |