3
Orphan Designations
0
FDA Approvals
6
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| carbon monoxide-induced delayed encephalopathy | a bispecific antibody composed of a Light Chain and a Heavy Chain pair targeting HER2, and a Light Chain and a Heavy Chain pair targeting CD47 | Orphan Designation | - |
| colorectal cancer, hereditary nonpolyposis, type 6 | small molecule that selectively and irreversibly inhibits the KRAS p.G12C mutant protein | Orphan Designation | - |
| colorectal cancer, hereditary nonpolyposis, type 7 | small molecule that selectively and irreversibly inhibits the KRAS p.G12C mutant protein | Orphan Designation | - |
| malignant pancreatic neoplasm | a multiple substituted pyrimidoheterocycle small molecule inhibitor that covalently binds to the Switch II pocket of KRAS p.G12C and locks the oncogenic protein in its GDP-bound inactive form | Orphan Designation | - |
| multisystemic smooth muscle dysfunction syndrome | a bispecific antibody composed of a Light Chain and a Heavy Chain pair targeting HER2, and a Light Chain and a Heavy Chain pair targeting CD47 | Orphan Designation | - |
| natal teeth-intestinal pseudoobstruction-patent ductus syndrome | a bispecific antibody composed of a Light Chain and a Heavy Chain pair targeting HER2, and a Light Chain and a Heavy Chain pair targeting CD47 | Orphan Designation | - |