8
Orphan Designations
3
FDA Approvals
14
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Alagille syndrome | Livmarli | Orphan Designation | - |
| arthrogryposis, renal dysfunction, and cholestasis 2 | Livmarli | Orphan Designation | - |
| benign recurrent intrahepatic cholestasis | Livmarli | Orphan Designation | - |
| biliary atresia | maralixibat | Orphan Designation | - |
| cerebrotendinous xanthomatosis | Ctexli | Orphan Designation | - |
| congenital bile acid synthesis defect 3 | Cholbam | Orphan Designation | - |
| inborn disorder of amino acid metabolism | Cholbam | Orphan Designation | - |
| inborn disorder of bile acid synthesis | Cholbam | Orphan Designation | - |
| inborn disorder of purine or pyrimidine metabolism | Cholbam | Orphan Designation | - |
| inborn error of immunity | Cholbam | Orphan Designation | - |
| inborn errors of metabolism | Cholbam | Orphan Designation | - |
| primary biliary cholangitis | volixibat(4R,5R)-1-[[4-[[4-[3,3-Dibutyl-7-(dimethylamino)-2,3,4,5-tetrahydro-4-hydroxy-1,1-dioxido-1-benzothiepin-5-yl]phenoxy]methyl]phenyl]methyl]-4-aza-l-azoniabicyclo[2.2.2]octane Chloride | Orphan Designation | - |
| primary sclerosing cholangitis | (4R,5R)-1-[[4-[[4-[3,3-Dibutyl-7-(dimethylamino)-2,3,4,5-tetrahydro-4-hydroxy-1,1-dioxido-1-benzothiepin-5-yl]phenoxy]methyl]phenyl]methyl]-4-aza-l-azoniabicyclo[2.2.2]octane Chloride | Orphan Designation | - |
| progressive familial intrahepatic cholestasis | Livmarli | Orphan Designation | - |