A biopharmaceutical company focused on developing and commercializing treatments for rare diseases, with 7 FDA-approved drugs and active involvement in 12 rare conditions, including pediatric cancers and syndromes.
2
Orphan Designations
7
FDA Approvals
12
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Adams-Oliver syndrome 1 | Xifaxan | Orphan Designation | - |
| Diamond-Blackfan anemia 11 | Xifaxan | Orphan Designation | - |
| IgE responsiveness, atopic | Xifaxan | Orphan Designation | - |
| cleidocranial dysplasia 1 | Colazal | Orphan Designation | - |
| discrimination, Two-point, reduction 1N | Xifaxan | Orphan Designation | - |
| fibrous dysplasia | Xifaxan | Orphan Designation | - |
| hepatic encephalopathy | Xifaxan | Orphan Designation | - |
| pediatric acute-onset neuropsychiatric syndrome | Colazal | Orphan Designation | - |
| pediatric hepatocellular carcinoma | Colazal | Orphan Designation | - |
| pediatric ovarian dysgerminoma | Colazal | Orphan Designation | - |
| pediatric ovarian germ cell tumor | Colazal | Orphan Designation | - |
| pediatric-onset Graves disease | Colazal | Orphan Designation | - |