This pharmaceutical company is engaged in developing treatments for rare diseases, with 29 FDA-approved drugs and 2 orphan drug designations, focusing on conditions like nephropathic cystinosis and Mycobacterium avium complex disease.
2
Orphan Designations
29
FDA Approvals
11
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| DYRK1A-related intellectual disability syndrome | Sulfamylon solution | Orphan Designation | - |
| Fanconi renotubular syndrome | Cystagon | Orphan Designation | - |
| Mycobacterium avium complex disease | RIFABUTIN | - | FDA Approved |
| anosmia for isobutyric acid | Sulfamylon solution | Orphan Designation | - |
| disseminated | RIFABUTIN | - | FDA Approved |
| early-onset partial cataract | PREGABALIN | - | FDA Approved |
| juvenile nephropathic cystinosis | Cystagon | Orphan Designation | - |
| nephropathic cystinosis | Cystagon | Orphan Designation | - |
| nephropathic infantile cystinosis | Cystagon | Orphan Designation | - |
| neuroacanthocytosis | Sulfamylon solution | Orphan Designation | - |
| ocular cystinosis | Cystagon | Orphan Designation | - |