23
Orphan Designations
0
FDA Approvals
50
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| AIDS | Hivid | Orphan Designation | - |
| AIDS related complex | Roferon-A | Orphan Designation | - |
| AIDS-related disorder | Roferon-A | Orphan Designation | - |
| Bloom syndrome | Lariam | Orphan Designation | - |
| Down syndrome | Lariam | Orphan Designation | - |
| Kabuki syndrome 1 | Zelboraf | Orphan Designation | - |
| Kaposi's sarcoma | Roferon-A | Orphan Designation | - |
| Plasmodium falciparum malaria | Lariam | Orphan Designation | - |
| acquired polycythemia vera | idasanutlin | Orphan Designation | - |
| acute flaccid myelitis | Humanized anti-tacLariam | Orphan Designation | - |
| acute graft versus host disease | Humanized anti-tac | Orphan Designation | - |
| acute infection of pinna | Vesanoid | Orphan Designation | - |
| acute kidney injury | Zenapax | Orphan Designation | - |
| acute kidney tubular necrosis | Zenapax | Orphan Designation | - |
| acute lymphoblastic leukemia | Humanized anti-tac | Orphan Designation | - |
| acute papillary necrosis | Zenapax | Orphan Designation | - |
| acute promyelocytic leukemia | Vesanoid | Orphan Designation | - |
| adult neuronal ceroid lipofuscinosis | Hivid | Orphan Designation | - |
| adult pleomorphic rhabdomyosarcoma | Hivid | Orphan Designation | - |
| adult-onset non-insulinoma persistent hyperinsulinemic hypoglycemia | Hivid | Orphan Designation | - |
| adult-onset proximal spinal muscular atrophy, autosomal dominant | Hivid | Orphan Designation | - |
| advanced heart failure | Interferon alfa-2a (recombinant)Fluorouracil | Orphan Designation | - |
| advanced sleep phase syndrome | Interferon alfa-2a (recombinant)Fluorouracil | Orphan Designation | - |
| carcinoma of esophagus | FluorouracilInterferon alfa-2a (recombinant) | Orphan Designation | - |
| chronic myelogenous leukemia, BCR-ABL1 positive | Roferon A | Orphan Designation | - |
| clear cell renal carcinoma | Interleukin-2Interferon alfa-2a (recombinant) | Orphan Designation | - |
| colorectal cancer | Interferon alfa-2a (recombinant)Fluorouracil | Orphan Designation | - |
| congenital cataracts-facial dysmorphism-neuropathy syndrome | Lariam | Orphan Designation | - |
| crush syndrome | Zenapax | Orphan Designation | - |
| fragile X syndrome | basimglurant | Orphan Designation | - |
| fungal infection of eye | Vesanoid | Orphan Designation | - |
| fungal infection of the toenail | Vesanoid | Orphan Designation | - |
| gonococcal infection of joint | Vesanoid | Orphan Designation | - |
| hemolytic-uremic syndrome | Zenapax | Orphan Designation | - |
| hereditary hyperekplexia | Clonazepam | Orphan Designation | - |
| hyperekplexia 1 | Clonazepam | Orphan Designation | - |
| hyperekplexia 3 | Clonazepam | Orphan Designation | - |
| hypertensive urgency | Zenapax | Orphan Designation | - |
| metastatic malignant neoplasm in the colon | Interferon alfa-2a (recombinant)Interleukin-2 | Orphan Designation | - |
| metastatic malignant neoplasm in the lymph nodes | Interferon alfa-2a (recombinant) | Orphan Designation | - |
| metastatic melanoma | Interferon alfa-2a (recombinant)Interleukin-2 | Orphan Designation | - |
| mitochondrial DNA depletion syndrome 13 | Zelboraf | Orphan Designation | - |
| mitochondrial DNA depletion syndrome 9 | Zelboraf | Orphan Designation | - |
| multiple congenital anomalies-hypotonia-seizures syndrome 2 | Lariam | Orphan Designation | - |
| neonatal encephalomyopathy-cardiomyopathy-respiratory distress syndrome | Zelboraf | Orphan Designation | - |
| primary adult heart tumor | Hivid | Orphan Designation | - |
| renal cell carcinoma | Interferon alfa-2a (recombinant) | Orphan Designation | - |
| retinitis pigmentosa 12 | Zelboraf | Orphan Designation | - |
| short-rib thoracic dysplasia 9 with or without polydactyly | Zelboraf | Orphan Designation | - |
| viral infection of central nervous system | Vesanoid | Orphan Designation | - |