6
Orphan Designations
1
FDA Approvals
17
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Alstrom syndrome | setmelanotide | Orphan Designation | - |
| Bardet-Biedl syndrome | Imcivree | Orphan Designation | - |
| PR interval, variation in | Imcivree | Orphan Designation | - |
| PRC-2 complex-related overgrowth spectrum | Imcivree | Orphan Designation | - |
| PrP systemic amyloidosis | Imcivree | Orphan Designation | - |
| chronic atrial and intestinal dysrhythmia | Imcivree | Orphan Designation | - |
| chronic beryllium disease | Imcivree | Orphan Designation | - |
| chronic graft versus host disease | Imcivree | Orphan Designation | - |
| chronic hiccup | Imcivree | Orphan Designation | - |
| hemolytic poikilocytic anemia due to reduced ankyrin binding sites | Imcivree | Orphan Designation | - |
| long QT syndrome 2 | Imcivree | Orphan Designation | - |
| malignant carotid body paraganglioma | oral melanocortin 4 receptor agonistSmall molecule melanocortin 4 receptor agonist | Orphan Designation | - |
| obesity due to leptin receptor gene deficiency | Imcivree | Orphan Designation | - |
| obesity due to pro-opiomelanocortin deficiency | Imcivree | Orphan Designation | - |
| post-treatment Lyme disease syndrome | oral melanocortin 4 receptor agonistSmall molecule melanocortin 4 receptor agonist | Orphan Designation | - |
| pre-malignant neoplasm | Imcivree | Orphan Designation | - |
| treatment-refractory schizophrenia | oral melanocortin 4 receptor agonistSmall molecule melanocortin 4 receptor agonist | Orphan Designation | - |