4
Orphan Designations
1
FDA Approvals
9
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Diamond-Blackfan anemia 9 | Aqvesme | Orphan Designation | - |
| adult Refsum disease | Aqvesme | Orphan Designation | - |
| glutathione synthetase deficiency without 5-oxoprolinuria | Pyrukynd | Orphan Designation | - |
| megaloblastic anemia, folate-responsive | AqvesmePyrukynd | Orphan Designation | - |
| myelodysplastic syndrome | 6-((6-aminopyridin-2-yl)methyl)-4-methyl-2-(1H-pyrazol-3-ylmethyl)-4,6-dihydro-5H-[1,3]thiazolo[5',4':4,5]pyrrolo[2,3-d] pyridazin-5-one | Orphan Designation | - |
| pyruvate kinase deficiency of red cells | Pyrukynd | Orphan Designation | - |
| refractory anemia with excess blasts in transformation | Aqvesme | Orphan Designation | - |
| sickle cell disease | mitapivat | Orphan Designation | - |
| thalassemia | Aqvesme | Orphan Designation | - |