8
Orphan Designations
0
FDA Approvals
8
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| acute lymphoblastic leukemia | 3-[5-[(1R)-1-[bis(aziridin-1-yl(phosphoryloxy]ethyl]-2-nitrophenoxy]-N,N-dimethylbenzamide | Orphan Designation | - |
| carbon monoxide-induced delayed encephalopathy | antibody-drug conjugate (ADC) consisting of a humanized anti-trophoblast cell surface antigen-2 (TROP2) antibody (R4702) and a cathepsin B protease-cleavable linker-payload N-PM-0017 (MCCa-PEG24-VA-PAB-exatecan) | Orphan Designation | - |
| cholangiocarcinoma | antibody-drug conjugate (ADC) consisting of a humanized anti-trophoblast cell surface antigen-2 (TROP2) antibody (R4702) and a protease-cleavable linker-payload N-PM-0022 (BCN-GGVA-PAB(PEG24)-exatecan) | Orphan Designation | - |
| gastric cancer | antibody-drug conjugate (ADC) consisting of a humanized anti-trophoblast cell surface antigen-2 (TROP2) antibody and a protease-cleavable linker-payload N-PM-0022 (BCN-GGVA-PAB(PEG24)-exatecan)Humanized Anti-Globo H Monoclonal Antibody Monomethyl Auristatin E Conjugate | Orphan Designation | - |
| hepatocellular carcinoma | 3-[5-[(1R)-1-[bis(aziridin-1-yl(phosphoryloxy]ethyl]-2-nitrophenoxy]-N,N-dimethylbenzamide | Orphan Designation | - |
| malignant pancreatic neoplasm | Humanized Anti-Globo H Monoclonal Antibody Monomethyl Auristatin E Conjugatehumanized anti-Globo H monoclonal antibody | Orphan Designation | - |
| multisystemic smooth muscle dysfunction syndrome | antibody-drug conjugate (ADC) consisting of a humanized anti-trophoblast cell surface antigen-2 (TROP2) antibody (R4702) and a cathepsin B protease-cleavable linker-payload N-PM-0017 (MCCa-PEG24-VA-PAB-exatecan) | Orphan Designation | - |
| natal teeth-intestinal pseudoobstruction-patent ductus syndrome | antibody-drug conjugate (ADC) consisting of a humanized anti-trophoblast cell surface antigen-2 (TROP2) antibody (R4702) and a cathepsin B protease-cleavable linker-payload N-PM-0017 (MCCa-PEG24-VA-PAB-exatecan) | Orphan Designation | - |