8
Orphan Designations
0
FDA Approvals
10
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| AL amyloidosis | 2,2'-{2-[1R)-1-({[(2,5-dichlorobenzoyl)amino]acetyl}amino)-3-methylbutyl]-5-oxo-1,3,2-dioxaborolane-4,4-diyl}diacetic acid (ixazomib citrate) | Orphan Designation | - |
| UV-sensitive syndrome 1 | 2,2'-{2-[1R)-1-({[(2,5-dichlorobenzoyl)amino]acetyl}amino)-3-methylbutyl]-5-oxo-1,3,2-dioxaborolane-4,4-diyl}diacetic acid (ixazomib citrate) | Orphan Designation | - |
| acute lymphoblastic leukemia | bortezomib | Orphan Designation | - |
| follicular lymphoma | bortezomib | Orphan Designation | - |
| gastric cancer | antibody drug conjugate consisting of fully human anti-guanylyl cyclase C monoclonal antibody linked to the cytotoxic drug monomethyl auristatin E | Orphan Designation | - |
| malignant pancreatic neoplasm | monomethyl auristatin E conjugated to a monoclonal antibody which targets guanylyl cyclase C | Orphan Designation | - |
| mature T-cell and NK-cell non-Hodgkin lymphoma | alisertib | Orphan Designation | - |
| ovarian cancer | sodium 4-{ [9-chloro-7-(2-fluoro-6-methoxyphenyl)-5H-pyrimido [5,4-d] [2] benzazepin-2-yl]-2-methoxybenzoate | Orphan Designation | - |
| primary systemic amyloidosis | 2,2'-{2-[1R)-1-({[(2,5-dichlorobenzoyl)amino]acetyl}amino)-3-methylbutyl]-5-oxo-1,3,2-dioxaborolane-4,4-diyl}diacetic acid (ixazomib citrate) | Orphan Designation | - |
| small cell lung carcinoma | alisertib | Orphan Designation | - |