11
Orphan Designations
0
FDA Approvals
28
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Duchenne muscular dystrophy | idebenone | Orphan Designation | - |
| Friedreich ataxia | idebenone | Orphan Designation | - |
| LIPE-related familial partial lipodystrophy | metreleptin | Orphan Designation | - |
| Leber hereditary optic neuropathy | idebenone | Orphan Designation | - |
| PLIN1-related familial partial lipodystrophy | metreleptin | Orphan Designation | - |
| abetalipoproteinemia | Juxtapid | Orphan Designation | - |
| acute opioid poisoning | buprenorphine | Orphan Designation | - |
| botulism | Filsuvez | Orphan Designation | - |
| cystic fibrosis | (R)-2-(5-cyano-2-(6-(methoxycarbonyl)-7-methyl-3-oxo-8-(3-(trifluoromethyl)phenyl)-2,3,5,8-tetrahydro-[1,2,4]triazolo[4,3-a]pyrimidin-5-yl)phenyl)-N,N,N-trimethylethanaminium methanesulfonate dehydrate | Orphan Designation | - |
| epidermolysis bullosa | Filsuvez | Orphan Designation | - |
| familial chylomicronemia syndrome | lomitapide | Orphan Designation | - |
| familial partial lipodystrophy | metreleptin | Orphan Designation | - |
| familial partial lipodystrophy, Dunnigan type | metreleptin | Orphan Designation | - |
| familial partial lipodystrophy, Kobberling type | metreleptin | Orphan Designation | - |
| homozygous 11P15-p14 deletion syndrome | Juxtapid | Orphan Designation | - |
| homozygous familial hypercholesterolemia | Juxtapid | Orphan Designation | - |
| hypotonia-cystinuria syndrome | Juxtapid | Orphan Designation | - |
| idiopathic pulmonary fibrosis | zampilimab | Orphan Designation | - |
| inborn carbohydrate metabolic disorder | MYALEPT | Orphan Designation | - |
| inborn errors of metabolism | MYALEPT | Orphan Designation | - |
| inborn vitamin metabolic disorder | MYALEPT | Orphan Designation | - |
| injury | Filsuvez | Orphan Designation | - |
| mannose-binding lectin deficiency | metreleptin | Orphan Designation | - |
| opioid abuse | buprenorphine | Orphan Designation | - |
| opioid-induced constipation | buprenorphine | Orphan Designation | - |
| steroid inherited metabolic disorder | MYALEPT | Orphan Designation | - |
| wound botulism | Filsuvez | Orphan Designation | - |
| wound myiasis | Filsuvez | Orphan Designation | - |