12
Orphan Designations
0
FDA Approvals
24
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Kifafa seizure disorder | caffeine and sodium benzoate | Orphan Designation | - |
| atypical hemolytic-uremic syndrome | Sodium Thiosulfate | Orphan Designation | - |
| avascular necrosis of femoral head, primary, 1 | Recombinant human platelet derived growth factor BB | Orphan Designation | - |
| colonic pseudo-obstruction | neostigmine | Orphan Designation | - |
| congenital | methylene blue injection | Orphan Designation | - |
| drug-induced methemoglobinemia | methylene blue injection | Orphan Designation | - |
| epilepsy | caffeine and sodium benzoate | Orphan Designation | - |
| familial avascular necrosis of femoral head | Recombinant human platelet derived growth factor BB | Orphan Designation | - |
| gastric adenocarcinoma | DHA-paclitaxel | Orphan Designation | - |
| gastric cardia adenocarcinoma | DHA-paclitaxel | Orphan Designation | - |
| hemolytic-uremic syndrome | Sodium Thiosulfate | Orphan Designation | - |
| hormone receptor-positive breast cancer | docosahexanoic acid-paclitaxel | Orphan Designation | - |
| hormone-resistant breast carcinoma | docosahexanoic acid-paclitaxel | Orphan Designation | - |
| hormone-resistant prostate carcinoma | docosahexanoic acid-paclitaxel | Orphan Designation | - |
| malignant pancreatic neoplasm | DHA-paclitaxel | Orphan Designation | - |
| metastatic melanoma | DHA-paclitaxel | Orphan Designation | - |
| mucinous gastric adenocarcinoma | DHA-paclitaxel | Orphan Designation | - |
| myasthenia gravis | neostigmine methylsulfate | Orphan Designation | - |
| neonatal-onset encephalopathy with rigidity and seizures | caffeine and sodium benzoate | Orphan Designation | - |
| non-Zellweger spectrum disorder | Sodium Thiosulfate | Orphan Designation | - |
| osteonecrosis of the jaw | Recombinant human platelet derived growth factor BB | Orphan Designation | - |
| tetralogy of fallot | phenylephrine | Orphan Designation | - |
| trigeminal neuralgia | dehydrated alcohol | Orphan Designation | - |
| uremic neuropathy | Sodium Thiosulfate | Orphan Designation | - |