Specializing in treatments for rare diseases, the company has FDA-approved drugs for conditions like Gaucher disease and pulmonary arterial hypertension, with one orphan drug designation.
1
Orphan Designations
8
FDA Approvals
12
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Gaucher disease type I | ZAVESCA | - | FDA Approved |
| Gaucher disease type II | ZAVESCA | - | FDA Approved |
| Gaucher disease type III | ZAVESCA | - | FDA Approved |
| adult embryonal tumor with multilayered rosettes, c19mc-altered | Opsynvi | Orphan Designation | - |
| cystic fibrosis | Opsynvi | Orphan Designation | - |
| dermatomyositis | Opsynvi | Orphan Designation | - |
| grade II glioma | Opsynvi | Orphan Designation | - |
| grade II meningioma | Opsynvi | Orphan Designation | - |
| grade III glioma | Opsynvi | Orphan Designation | - |
| grade III meningioma | Opsynvi | Orphan Designation | - |
| nut midline carcinoma | Opsynvi | Orphan Designation | - |
| pulmonary arterial hypertension | Opsynvi | Orphan Designation | - |