10
Orphan Designations
4
FDA Approvals
29
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| HER2 positive breast carcinoma | Tukysa | Orphan Designation | - |
| Her2-receptor negative breast cancer | Tukysa | Orphan Designation | - |
| Hodgkins lymphoma | Adcetris | Orphan Designation | - |
| adult neuronal ceroid lipofuscinosis | tucatinibAdcetris | Orphan Designation | - |
| adult pleomorphic rhabdomyosarcoma | tucatinibAdcetris | Orphan Designation | - |
| adult-onset non-insulinoma persistent hyperinsulinemic hypoglycemia | TukysatucatinibAdcetris | Orphan Designation | - |
| adult-onset proximal spinal muscular atrophy, autosomal dominant | tucatinibAdcetris | Orphan Designation | - |
| breast cancer | tucatinib | Orphan Designation | - |
| cervical cancer | TIVDAK | - | FDA Approved |
| congenital heart disease | Adcetris | Orphan Designation | - |
| congenital lethal erythroderma | Adcetris | Orphan Designation | - |
| esophageal cancer | humanized immunoglobulin G1 anti-integrin beta-6 monoclonal antibody conjugated to monomethyl auristatin E | Orphan Designation | - |
| gastric cancer | disitamab vedotin | Orphan Designation | - |
| hormone receptor-positive breast cancer | Tukysa | Orphan Designation | - |
| malignant carotid body paraganglioma | Adcetris | Orphan Designation | - |
| mature T-cell and NK-cell non-Hodgkin lymphoma | Adcetris | Orphan Designation | - |
| metachromatic leukodystrophy, adult form | Adcetris | Orphan Designation | - |
| mycosis fungoides | Adcetris | Orphan Designation | - |
| plasma cell myeloma | Nonfucosylated humanized immunoglobulin G1 (IgG1) monoclonal antibody targeting B-cell maturation antigen (SEA-BCMA) | Orphan Designation | - |
| post 5-alpha-reductase inhibitors treatment syndrome | Adcetris | Orphan Designation | - |
| post-treatment Lyme disease syndrome | Adcetris | Orphan Designation | - |
| primary adult heart tumor | TukysatucatinibAdcetris | Orphan Designation | - |
| primary cutaneous CD30+ T-cell lymphoproliferative disease | Adcetris | Orphan Designation | - |
| primary cutaneous T-cell non-Hodgkin lymphoma | Brentuximab vedotin | Orphan Designation | - |
| primary cutaneous anaplastic large cell lymphoma | Adcetris | Orphan Designation | - |
| primary cutaneous follicle center lymphoma | Adcetris | Orphan Designation | - |
| primary cutaneous lymphoma | Adcetris | Orphan Designation | - |
| treatment-refractory schizophrenia | Adcetris | Orphan Designation | - |
| triple-positive breast carcinoma | Tukysa | Orphan Designation | - |