6
Orphan Designations
0
FDA Approvals
15
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Gaucher disease type III | Rezurock | Orphan Designation | - |
| Grant syndrome | Rezurock | Orphan Designation | - |
| Roberts-SC phocomelia syndrome | Tesevatinib | Orphan Designation | - |
| acute graft versus host disease | Rezurock | Orphan Designation | - |
| autosomal dominant polycystic kidney disease | tesevatinib | Orphan Designation | - |
| chronic graft versus host disease | Rezurock | Orphan Designation | - |
| deafness, congenital, and adult-onset progressive leukoencephalopathy | Rezurock | Orphan Designation | - |
| graft versus host disease | Rezurock | Orphan Designation | - |
| lung cancer | tesevatinib | Orphan Designation | - |
| malignant pancreatic neoplasm | salirasib | Orphan Designation | - |
| microcephalic osteodysplastic primordial dwarfism type I | Tesevatinib | Orphan Designation | - |
| non-small cell lung carcinoma | tesevatinib | Orphan Designation | - |
| non-small cell squamous lung carcinoma | tesevatinib | Orphan Designation | - |
| odontochondrodysplasia 1 | Tesevatinib | Orphan Designation | - |
| systemic sclerosis | Belumosudil | Orphan Designation | - |