28
Orphan Designations
0
FDA Approvals
37
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| ELANE-related neutropenia | Sivelestat | Orphan Designation | - |
| Ehlers-Danlos syndrome, vascular type | papaverine | Orphan Designation | - |
| Evans syndrome | Cepharanthine | Orphan Designation | - |
| Kennedy disease | geranylgeranylacetone | Orphan Designation | - |
| acute myeloid leukemia | Ubenimex | Orphan Designation | - |
| acute radiation syndrome | UlinastatinSivelestatgeranylgeranylacetone | Orphan Designation | - |
| anti-neutrophil cytoplasmic antibody-associated vasculitis | Gabexate | Orphan Designation | - |
| autoimmune thrombocytopenic purpura | Cepharanthine | Orphan Designation | - |
| bulbospinal muscular atrophy | geranylgeranylacetone | Orphan Designation | - |
| carcinoma of esophagus | 4-Methylumbelliferone | Orphan Designation | - |
| chronic intestinal vascular insufficiency | papaverine | Orphan Designation | - |
| chronic myelogenous leukemia, BCR-ABL1 positive | Ubenimex | Orphan Designation | - |
| chronic pancreatitis | Proglumide | Orphan Designation | - |
| cyclic hematopoiesis | Sivelestat | Orphan Designation | - |
| dengue disease | Nafamostat | Orphan Designation | - |
| esophageal cancer | nedaplatin | Orphan Designation | - |
| gastric cancer | Proglumidesivelestat | Orphan Designation | - |
| granulomatosis with polyangiitis | Gabexate | Orphan Designation | - |
| heparin-induced thrombocytopenia | papaverine | Orphan Designation | - |
| hepatocellular carcinoma | Ubenimex4-Methylumbelliferone | Orphan Designation | - |
| immune dysregulation-polyendocrinopathy-enteropathy-X-linked syndrome | Cepharanthine | Orphan Designation | - |
| malignant pancreatic neoplasm | Nafamostat4-Methylumbelliferone | Orphan Designation | - |
| membranous glomerulonephritis | Mizoribine | Orphan Designation | - |
| microvillus inclusion disease | racecadotril | Orphan Designation | - |
| mucocutaneous lymph node syndrome | Ulinastatin | Orphan Designation | - |
| myelodysplastic syndrome | Sivelestat | Orphan Designation | - |
| neutropenia, severe congenital, 1, autosomal dominant | Sivelestat | Orphan Designation | - |
| pauci-immune glomerulonephritis without ANCA | Gabexate | Orphan Designation | - |
| post-thrombotic syndrome | Geranylgeranylacetone | Orphan Designation | - |
| post-traumatic stress disorder | Geranylgeranylacetone | Orphan Designation | - |
| post-vaccinal encephalitis | Geranylgeranylacetone | Orphan Designation | - |
| primary biliary cholangitis | combination of p-Tolylmethylcarbinol nicotinic acid ester and a-naphthylacetic acid | Orphan Designation | - |
| small cell lung carcinoma | Nedaplatin | Orphan Designation | - |
| spinal cord disorder | geranylgeranylacetone | Orphan Designation | - |
| spinal cord injury | geranylgeranylacetone | Orphan Designation | - |
| spinal injury | geranylgeranylacetone | Orphan Designation | - |
| spinal muscular atrophy | geranylgeranylacetone | Orphan Designation | - |