12
Orphan Designations
0
FDA Approvals
20
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| B-cell chronic lymphocytic leukemia | brexucabtagene autoleucel | Orphan Designation | - |
| Bicervical bicornuate uterus with patent cervix and vagina | Human autologous CD4+ and CD8+ T cells engineered ex vivo to express a T cell receptor that recognizes amino acids 11-19 of the human papillomavirus (HPV)16 E7 protein when bound to human leukocyte antigen-A*02:01 | Orphan Designation | - |
| MALT lymphoma | axicabtagene ciloleucel | Orphan Designation | - |
| acute lymphoblastic leukemia | Tecartus | Orphan Designation | - |
| acute myeloid leukemia | An autologous chimeric antigen receptor (CAR) T-cell therapy engineered to express an antibody-derived single-chain variable fragment that recognizes C-type lectin-like molecule-1 (CLL-1). | Orphan Designation | - |
| adult neuronal ceroid lipofuscinosis | TECARTUS™YescartaTecartus | Orphan Designation | - |
| adult pleomorphic rhabdomyosarcoma | TECARTUS™YescartaTecartus | Orphan Designation | - |
| adult-onset non-insulinoma persistent hyperinsulinemic hypoglycemia | TECARTUS™YescartaTecartus | Orphan Designation | - |
| adult-onset proximal spinal muscular atrophy, autosomal dominant | TECARTUS™YescartaTecartus | Orphan Designation | - |
| carbon monoxide-induced delayed encephalopathy | Human autologous CD4+ and CD8+ T cells engineered ex vivo to express a T cell receptor that recognizes amino acids 11-19 of the human papillomavirus (HPV)16 E7 protein when bound to human leukocyte antigen-A*02:01 | Orphan Designation | - |
| diffuse large B-cell lymphoma | Yescarta | Orphan Designation | - |
| extranodal nasal NK/T cell lymphoma | axicabtagene ciloleucel | Orphan Designation | - |
| follicular lymphoma | Yescarta | Orphan Designation | - |
| mantle cell lymphoma | TECARTUS™ | Orphan Designation | - |
| mycosis fungoides | axicabtagene ciloleucel | Orphan Designation | - |
| natal teeth-intestinal pseudoobstruction-patent ductus syndrome | Human autologous CD4+ and CD8+ T cells engineered ex vivo to express a T cell receptor that recognizes amino acids 11-19 of the human papillomavirus (HPV)16 E7 protein when bound to human leukocyte antigen-A*02:01 | Orphan Designation | - |
| nodal marginal zone B-cell lymphoma | axicabtagene ciloleucel | Orphan Designation | - |
| plasma cell myeloma | anitocabtagene autoleucelautologous engineered T-cell product that targets B-cell maturation antigen (BCMA) | Orphan Designation | - |
| primary adult heart tumor | TECARTUS™YescartaTecartus | Orphan Designation | - |
| primary mediastinal large B-cell lymphoma | Yescarta | Orphan Designation | - |