8
Orphan Designations
0
FDA Approvals
35
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Diamond-Blackfan anemia | Prolastin | Orphan Designation | - |
| Hodgkins lymphoma | Sorafenib | Orphan Designation | - |
| Hughes-Stovin syndrome | Thrombate III | Orphan Designation | - |
| Joubert syndrome 22 | Nexavar | Orphan Designation | - |
| Leber congenital amaurosis 5 | Nexavar | Orphan Designation | - |
| Noonan syndrome 1 | Kogenate | Orphan Designation | - |
| anosmia for isobutyric acid | TrasylolProlastin | Orphan Designation | - |
| bilirubin encephalopathy | Gamimune N | Orphan Designation | - |
| cytomegalovirus pneumonia | Cytomegalovirus immune globulin intravenous (human) | Orphan Designation | - |
| double nail for fifth toe | TrasylolProlastin | Orphan Designation | - |
| enhanced S-cone syndrome | Nexavar | Orphan Designation | - |
| esophageal varices | Kogenate | Orphan Designation | - |
| hemoglobin--variants for which the chain carrying the mutation 1S unknown or uncertain | TrasylolProlastin | Orphan Designation | - |
| hemorrhagic disease of newborn | Kogenate | Orphan Designation | - |
| hepatocellular carcinoma | Nexavar | Orphan Designation | - |
| idiopathic disseminated cytomegalovirus infection | Cytomegalovirus immune globulin intravenous (human) | Orphan Designation | - |
| infection by Trypanosoma gambiense | Gamimune N | Orphan Designation | - |
| infection by Trypanosoma rhodesiense | Gamimune N | Orphan Designation | - |
| infection-related hemolytic uremic syndrome | Gamimune N | Orphan Designation | - |
| modifier, X-linked, for Neurofunctional defects | Prolastin | Orphan Designation | - |
| overhydrated hereditary stomatocytosis | Thrombate III | Orphan Designation | - |
| platelet-type bleeding disorder 10 | Kogenate | Orphan Designation | - |
| platelet-type bleeding disorder 17 | Kogenate | Orphan Designation | - |
| platelet-type bleeding disorder 19 | Kogenate | Orphan Designation | - |
| puerperal infection | Gamimune N | Orphan Designation | - |
| puerperal pulmonary embolism | Thrombate III | Orphan Designation | - |
| pulmonary embolism | Thrombate III | Orphan Designation | - |
| pulmonary embolism and infarction | Thrombate III | Orphan Designation | - |
| retinitis pigmentosa 93 | Nexavar | Orphan Designation | - |
| senior-loken syndrome 3 | Nexavar | Orphan Designation | - |
| spatial visualization, aptitude for | Prolastin | Orphan Designation | - |
| spinal muscular atrophy | Gamimune N | Orphan Designation | - |
| stage II endometrioid carcinoma | Sorafenib | Orphan Designation | - |
| stage IVb bladder cancer | Sorafenib | Orphan Designation | - |
| thrombotic disease | Thrombate III | Orphan Designation | - |