6
Orphan Designations
2
FDA Approvals
23
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| CIC-rearranged sarcoma | GAVRETO™ | Orphan Designation | - |
| Diamond-Blackfan anemia 14 with mandibulofacial dysostosis | Gavreto | Orphan Designation | - |
| FOXG1 disorder | Gavreto | Orphan Designation | - |
| RET fusion positive cancer | Gavreto | Orphan Designation | - |
| acrodysostosis 2 with or without hormone resistance | Gavreto | Orphan Designation | - |
| acute myeloid leukemia | Rezlidhia | Orphan Designation | - |
| adult neuronal ceroid lipofuscinosis | GAVRETO™Rezlidhia | Orphan Designation | - |
| adult pleomorphic rhabdomyosarcoma | GAVRETO™Rezlidhia | Orphan Designation | - |
| adult-onset non-insulinoma persistent hyperinsulinemic hypoglycemia | GAVRETO™Rezlidhia | Orphan Designation | - |
| adult-onset proximal spinal muscular atrophy, autosomal dominant | GAVRETO™Rezlidhia | Orphan Designation | - |
| autoimmune hemolytic anemia | fostamatinib disodium | Orphan Designation | - |
| autoimmune thrombocytopenic purpura | fostamatinib disodium hexahydrate | Orphan Designation | - |
| epilepsy, familial adult myoclonic, 1 | Gavreto | Orphan Designation | - |
| mixed-type autoimmune hemolytic anemia | fostamatinib disodium | Orphan Designation | - |
| myelodysplastic syndrome | selective dual small molecule inhibitor of IRAK1 and IRAK4 | Orphan Designation | - |
| non-autoimmune hemolytic anemia | fostamatinib disodium | Orphan Designation | - |
| optic atrophy 11 | Gavreto | Orphan Designation | - |
| platelet-type bleeding disorder 20 | fostamatinib disodium hexahydrate | Orphan Designation | - |
| primary adult heart tumor | GAVRETO™Rezlidhia | Orphan Designation | - |
| radioulnar synostosis with amegakaryocytic thrombocytopenia 1 | fostamatinib disodium hexahydrate | Orphan Designation | - |
| radioulnar synostosis with amegakaryocytic thrombocytopenia 2 | fostamatinib disodium hexahydrate | Orphan Designation | - |
| thrombocytopenia 4 | fostamatinib disodium hexahydrate | Orphan Designation | - |
| thrombocytopenia 5 | fostamatinib disodium hexahydrate | Orphan Designation | - |