7
Orphan Designations
0
FDA Approvals
19
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| acute myeloid leukemia | co-packaged combination of azacitidine and cedazuridinefixed dose combination of decitabine and cedazuridine | Orphan Designation | - |
| adult neuronal ceroid lipofuscinosis | LONSURF | Orphan Designation | - |
| adult pleomorphic rhabdomyosarcoma | LONSURF | Orphan Designation | - |
| adult-onset non-insulinoma persistent hyperinsulinemic hypoglycemia | LytgobiLONSURF | Orphan Designation | - |
| adult-onset proximal spinal muscular atrophy, autosomal dominant | LONSURF | Orphan Designation | - |
| childhood myelodysplastic syndrome | co-packaged combination of azacitidine and cedazuridine | Orphan Designation | - |
| cholangiocarcinoma | Lytgobi | Orphan Designation | - |
| chronic myelomonocytic leukemia | co-packaged combination of azacitidine and cedazuridine | Orphan Designation | - |
| gastric cancer | LONSURFTegafur/gimeracil/oteracil | Orphan Designation | - |
| hilar cholangiocarcinoma | Lytgobi | Orphan Designation | - |
| hyperprolactinemia | LONSURF | Orphan Designation | - |
| inappropriate ADH syndrome | LONSURF | Orphan Designation | - |
| intrahepatic cholangiocarcinoma | Lytgobi | Orphan Designation | - |
| myelodysplastic syndrome | co-packaged combination of azacitidine and cedazuridine | Orphan Designation | - |
| myelodysplastic syndrome with excess blasts-2 | co-packaged combination of azacitidine and cedazuridine | Orphan Designation | - |
| nephrogenic syndrome of inappropriate antidiuresis | LONSURF | Orphan Designation | - |
| perihilar intrahepatic cholangiocarcinoma | Lytgobi | Orphan Designation | - |
| primary adult heart tumor | LONSURF | Orphan Designation | - |
| sarcomatous intrahepatic cholangiocarcinoma | Lytgobi | Orphan Designation | - |