20
Orphan Designations
3
FDA Approvals
36
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| B-cell chronic lymphocytic leukemia | Brukinsa | Orphan Designation | - |
| Hodgkin's lymphoma, lymphocytic depletion | Brukinsa | Orphan Designation | - |
| MALT lymphoma | Brukinsa | Orphan Designation | - |
| Nodal T-follicular helper cell lymphoma, follicular type | Brukinsa | Orphan Designation | - |
| Waldenstrom macroglobulinemia | orally available BTK-targeting chimeric degradation activation compound designed to degrade wildtype BTK and multiple mutant formsSonrotoclaxBrukinsa | Orphan Designation | - |
| acute myeloid leukemia | Sonrotoclax | Orphan Designation | - |
| adult embryonal tumor with multilayered rosettes, c19mc-altered | Tevimbra | Orphan Designation | - |
| adult neuronal ceroid lipofuscinosis | BrukinsaBRUKINSA | Orphan Designation | - |
| adult pleomorphic rhabdomyosarcoma | BrukinsaBRUKINSA | Orphan Designation | - |
| adult-onset chronic progressive external ophthalmoplegia with mitochondrial myopathy | Tevimbra | Orphan Designation | - |
| adult-onset non-insulinoma persistent hyperinsulinemic hypoglycemia | BrukinsaBRUKINSA | Orphan Designation | - |
| adult-onset proximal spinal muscular atrophy, autosomal dominant | BrukinsaBRUKINSA | Orphan Designation | - |
| atrioventricular block | Brukinsa | Orphan Designation | - |
| cataract 13 with adult I phenotype | Tevimbra | Orphan Designation | - |
| esophageal cancer | Tevimbra | Orphan Designation | - |
| esophageal squamous cell carcinoma | TEVIMBRA | - | FDA Approved |
| extranodal nasal NK/T cell lymphoma | Brukinsa | Orphan Designation | - |
| follicular lymphoma | orally available BTK-targeting chimeric degradation activation compound designed to degrade wildtype BTK and multiple mutant formsBrukinsa | Orphan Designation | - |
| gastric cancer | Tevimbra | Orphan Designation | - |
| gastroesophageal junction adenocarcinoma | TEVIMBRA | - | FDA Approved |
| hepatocellular carcinoma | Tislelizumab | Orphan Designation | - |
| heterotaxy, visceral, 5, autosomal | Brukinsa | Orphan Designation | - |
| mantle cell lymphoma | Sonrotoclax3-tert-butyl-N-{(1R)-1-[4-(6-{6-[4-({1-[4-(2,4-dioxo-1,3-diazinan-1-yl)phenyl]piperidin-4-yl}methyl)piperazin-1-yl]pyridin-3-yl}-7H-pyrrolo[2,3-d]pyrimidin-4-yl)-2-methylphenyl]ethyl}-1,2,4-oxadiazole-5-carboxamideBRUKINSA | Orphan Designation | - |
| membranous glomerulonephritis | zanubrutinib | Orphan Designation | - |
| mycosis fungoides | Brukinsa | Orphan Designation | - |
| myelodysplastic syndrome | sonrotoclax | Orphan Designation | - |
| nasopharyngeal carcinoma | Tislelizumab | Orphan Designation | - |
| nodal marginal zone B-cell lymphoma | Brukinsa | Orphan Designation | - |
| nut midline carcinoma | Tevimbra | Orphan Designation | - |
| plasma cell myeloma | Sonrotoclax | Orphan Designation | - |
| primary adult heart tumor | BrukinsaBRUKINSA | Orphan Designation | - |
| splenic disorder | Brukinsa | Orphan Designation | - |
| splenic flexure cancer | Brukinsa | Orphan Designation | - |
| splenic infarction | Brukinsa | Orphan Designation | - |
| splenic manifestation of hairy cell leukemia | Brukinsa | Orphan Designation | - |
| splenic marginal zone lymphoma | Brukinsa | Orphan Designation | - |