9
Orphan Designations
1
FDA Approvals
29
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Adams-Oliver syndrome 1 | Enbrel | Orphan Designation | - |
| Bloom syndrome | Interleukin-1 alpha, human recombinant | Orphan Designation | - |
| Diamond-Blackfan anemia 11 | Enbrel | Orphan Designation | - |
| IgE responsiveness, atopic | Enbrel | Orphan Designation | - |
| Wolf-Hirschhorn syndrome | Interleukin-1 alpha, human recombinant | Orphan Designation | - |
| acute intermittent porphyria | Enbrel | Orphan Designation | - |
| acute myeloid leukemia | Leukine | Orphan Designation | - |
| anosmia for isobutyric acid | Interleukin-1 alpha, human recombinant | Orphan Designation | - |
| autosomal recessive osteopetrosis 2 | Interleukin-1 alpha, human recombinant | Orphan Designation | - |
| carbon monoxide-induced delayed encephalopathy | Leucovorin calcium | Orphan Designation | - |
| ceroid lipofuscinosis, neuronal, 6B (Kufs type) | Enbrel | Orphan Designation | - |
| congenital afibrinogenemia | Leukine | Orphan Designation | - |
| crush syndrome | Leukine | Orphan Designation | - |
| discrimination, Two-point, reduction 1N | Enbrel | Orphan Designation | - |
| epidermolysis bullosa simplex 1C, localized | Enbrel | Orphan Designation | - |
| fibrous dysplasia | Enbrel | Orphan Designation | - |
| granulomatosis with polyangiitis | Etanercept | Orphan Designation | - |
| hereditary coproporphyria | Enbrel | Orphan Designation | - |
| hydrops-lactic acidosis-sideroblastic anemia-multisystemic failure syndrome | Interleukin-1 alpha, human recombinant | Orphan Designation | - |
| metastatic malignant neoplasm in the colon | Leucovorin calcium | Orphan Designation | - |
| metastatic melanoma | Leucovorin calcium | Orphan Designation | - |
| metastatic neoplasm | Leucovorin calcium | Orphan Designation | - |
| metastatic squamous cell carcinoma | Leucovorin calcium | Orphan Designation | - |
| natal teeth-intestinal pseudoobstruction-patent ductus syndrome | Leucovorin calcium | Orphan Designation | - |
| neutropenia | Leukine | Orphan Designation | - |
| osteosarcoma | Leucovorin calcium | Orphan Designation | - |
| post-vaccinal encephalitis | Leukine | Orphan Designation | - |
| pulmonary valve insufficiency | Leukine | Orphan Designation | - |
| reducing body myopathy | Enbrel | Orphan Designation | - |