6
Orphan Designations
1
FDA Approvals
10
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| adult-onset non-insulinoma persistent hyperinsulinemic hypoglycemia | Avmapki Fakzynja Co-Pack | Orphan Designation | - |
| bronchial endocrine tumor | AvutometinibAvmapki Fakzynja Co-Pack | Orphan Designation | - |
| congenital disorder of deglycosylation 1 | AvutometinibAvmapki Fakzynja Co-Pack | Orphan Designation | - |
| low grade vulvar intraepithelial neoplasia | Avmapki Fakzynja Co-Pack | Orphan Designation | - |
| low-grade neuroendocrine tumor of the corpus uteri | Avmapki Fakzynja Co-Pack | Orphan Designation | - |
| malignant mesothelioma | 5-[8-methyl-9-(1-methylethyl)-2-(4-morpholinyl)-9H-purin-6yl]-2-pyrimidinamine | Orphan Designation | - |
| malignant pancreatic neoplasm | avutometinib and defactinib | Orphan Designation | - |
| mesothelioma | N-methyl-4-({4-[({3-methyl(methylsulfonyl)amino]pyrazin-2-yl}methyl)amino]-5-(trifluoromethyl)pyrimidin-2-yl}amino)benzamide hydrochloride | Orphan Designation | - |
| neurodevelopmental disorder with variable familial hypercholanemia | AvutometinibAvmapki Fakzynja Co-Pack | Orphan Designation | - |
| ovarian cancer | defactinib | Orphan Designation | - |