6
Orphan Designations
4
FDA Approvals
18
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Huntington disease | Ingrezza | Orphan Designation | - |
| Lennox-Gastaut syndrome | (S)-4-((1-Benzylpyrrolidin-3-yl)(methyl)amino)-2-fluoro-5-methyl-N-(thiazol-4-yl)benzenesulfonamide | Orphan Designation | - |
| TMEM63B-related developmental and epileptic encephalopathy with anemia | N-[1-(5-Cyano-pyridin-2-ylmethyl)-1H-pyrazol-3-yl]-2-[4-(1-trifluoromethyl-cyclopropyl-phenyl]-acetamide | Orphan Designation | - |
| classic congenital adrenal hyperplasia | 2,5-dimethyl-3-[2-methyl-4-(methyloxy)phenyl]-N-[(1S)-1-(3-methyl-1,2,4-oxadiazol-5- | Orphan Designation | - |
| congenital adrenal hyperplasia | Crenessity2,5-dimethyl-3-[2-methyl-4-(methyloxy)phenyl]-N-[(1S)-1-(3-methyl-1,2,4-oxadiazol-5- | Orphan Designation | - |
| congenital sodium diarrhea | (S)-4-((1-Benzylpyrrolidin-3-yl)(methyl)amino)-2-fluoro-5-methyl-N-(thiazol-4-yl)benzenesulfonamide | Orphan Designation | - |
| continuous spikes and waves during sleep | N-[1-(5-Cyano-pyridin-2-ylmethyl)-1H-pyrazol-3-yl]-2-[4-(1-trifluoromethyl-cyclopropyl-phenyl]-acetamide | Orphan Designation | - |
| developmental and epileptic encephalopathy 103 | N-[1-(5-Cyano-pyridin-2-ylmethyl)-1H-pyrazol-3-yl]-2-[4-(1-trifluoromethyl-cyclopropyl-phenyl]-acetamide | Orphan Designation | - |
| early-infantile DEE | (S)-4-((1-Benzylpyrrolidin-3-yl)(methyl)amino)-2-fluoro-5-methyl-N-(thiazol-4-yl)benzenesulfonamide | Orphan Designation | - |
| hyperkalemic periodic paralysis | (S)-4-((1-Benzylpyrrolidin-3-yl)(methyl)amino)-2-fluoro-5-methyl-N-(thiazol-4-yl)benzenesulfonamide | Orphan Designation | - |
| neuroferritinopathy | Ingrezza | Orphan Designation | - |
| non-classic congenital adrenal hyperplasia | 2,5-dimethyl-3-[2-methyl-4-(methyloxy)phenyl]-N-[(1S)-1-(3-methyl-1,2,4-oxadiazol-5- | Orphan Designation | - |
| nut midline carcinoma | Ingrezza | Orphan Designation | - |
| pediatric hepatocellular carcinoma | valbenazine | Orphan Designation | - |
| pediatric ovarian dysgerminoma | valbenazine | Orphan Designation | - |
| pediatric-onset Graves disease | valbenazine | Orphan Designation | - |
| potassium-aggravated myotonia | (S)-4-((1-Benzylpyrrolidin-3-yl)(methyl)amino)-2-fluoro-5-methyl-N-(thiazol-4-yl)benzenesulfonamide | Orphan Designation | - |
| sodium-potassium-ATPase activity of red cell | (S)-4-((1-Benzylpyrrolidin-3-yl)(methyl)amino)-2-fluoro-5-methyl-N-(thiazol-4-yl)benzenesulfonamide | Orphan Designation | - |