9
Orphan Designations
3
FDA Approvals
25
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Dubowitz syndrome | Oncaspar; Asparlas | Orphan Designation | - |
| Gaucher disease type III | Voranigo | Orphan Designation | - |
| Sandhoff disease, adult form | Voranigo | Orphan Designation | - |
| Young syndrome | Oncaspar; Asparlas | Orphan Designation | - |
| acute lymphoblastic leukemia | ONCASPAROncaspar; Asparlas | Orphan Designation | FDA Approved |
| acute myeloid leukemia | Menin InhibitorTIBSOVO | Orphan Designation | - |
| adult neuronal ceroid lipofuscinosis | TibsovoTIBSOVO | Orphan Designation | - |
| adult pleomorphic rhabdomyosarcoma | TibsovoTIBSOVO | Orphan Designation | - |
| adult-onset non-insulinoma persistent hyperinsulinemic hypoglycemia | TibsovoTIBSOVO | Orphan Designation | - |
| adult-onset proximal spinal muscular atrophy, autosomal dominant | TibsovoTIBSOVO | Orphan Designation | - |
| ataxia and polyneuropathy, adult-onset | Voranigo | Orphan Designation | - |
| cholangiocarcinoma | Tibsovo | Orphan Designation | - |
| chondrosarcoma | ivosidenib | Orphan Designation | - |
| deafness, congenital, and adult-onset progressive leukoencephalopathy | Voranigo | Orphan Designation | - |
| fragile X syndrome | small molecule modulator of large conductance, calcium-activated potassium (BK, KCa1.1) channels | Orphan Designation | - |
| glioma | Voranigoivosidenib | Orphan Designation | - |
| glioma susceptibility 3 | Oncaspar; Asparlas | Orphan Designation | - |
| myelodysplastic syndrome | Tibsovo | Orphan Designation | - |
| neuronopathy, distal hereditary motor, autosomal recessive 5 | Oncaspar; Asparlas | Orphan Designation | - |
| neuropathy, hereditary sensory and autonomic, adult-onset, with anosmia | Voranigo | Orphan Designation | - |
| nut midline carcinoma | Oncaspar; Asparlas | Orphan Designation | - |
| primary adult heart tumor | TibsovoTIBSOVO | Orphan Designation | - |
| primary microcephaly-mild intellectual disability-young-onset diabetes syndrome | Oncaspar; Asparlas | Orphan Designation | - |
| thrombocytopenia 5 | Oncaspar; Asparlas | Orphan Designation | - |
| young-onset Parkinson disease | Oncaspar; Asparlas | Orphan Designation | - |