8
Orphan Designations
5
FDA Approvals
21
Rare Diseases
1
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Netherton syndrome | a fusion protein comprised of genetically engineered human serine peptidase inhibitor and human immunoglobulin G1 Fc | Orphan Designation | - |
| acute myeloid leukemia | Vanflyta | Orphan Designation | - |
| adult neuronal ceroid lipofuscinosis | EnhertuVanflyta | Orphan Designation | - |
| adult pleomorphic rhabdomyosarcoma | EnhertuVanflyta | Orphan Designation | - |
| adult-onset non-insulinoma persistent hyperinsulinemic hypoglycemia | EnhertuTURALIOVanflyta | Orphan Designation | - |
| adult-onset proximal spinal muscular atrophy, autosomal dominant | EnhertuVanflyta | Orphan Designation | - |
| autosomal recessive inherited pseudoxanthoma elasticum | a small molecule tissue-nonspecific alkaline phosphatase inhibitor | Orphan Designation | - |
| combined small cell lung carcinoma | Ifinatamab deruxtecan | Orphan Designation | - |
| enteropathy-associated T-cell lymphoma | Valemetostat tosylate | Orphan Designation | - |
| esophageal cancer | ifinatamab deruxtecan | Orphan Designation | - |
| gastric cancer | Enhertu | Orphan Designation | - |
| mature T-cell and NK-cell non-Hodgkin lymphoma | Valemetostat tosylate | Orphan Designation | - |
| non-small cell squamous lung carcinoma | Ifinatamab deruxtecan | Orphan Designation | - |
| peripheral T-cell lymphoma, not otherwise specified | Valemetostat tosylate | Orphan Designation | - |
| pigmented dermatofibrosarcoma protuberans | TURALIO | Orphan Designation | - |
| pigmented nodular adrenocortical disease, primary, 1 | TURALIO | Orphan Designation | - |
| pigmented nodular adrenocortical disease, primary, 3 | TURALIO | Orphan Designation | - |
| primary adult heart tumor | EnhertuVanflyta | Orphan Designation | - |
| skin pigmented basal cell carcinoma | TURALIO | Orphan Designation | - |
| small cell lung carcinoma | Ifinatamab deruxtecan | Orphan Designation | - |
| tenosynovial giant cell tumor, diffuse type | TURALIO | Orphan Designation | - |