1
Orphan Designations
0
FDA Approvals
6
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| congenital lethal erythroderma | Hematopoietic stem cells and blood progenitors umbilical cord-derived expanded with (1r, 4r)-N1-(2-benzyl-7-(2-methyl-2H-tetrazol-5-yl)-9H-pyrimido[4,5-b]indol-4-yl)cyclohexane-1,4-diamine dihydrobromide dihydrate | Orphan Designation | - |
| immune dysregulation-polyendocrinopathy-enteropathy-X-linked syndrome | Hematopoietic stem cells and blood progenitors umbilical cord-derived expanded with (1r, 4r)-N1-(2-benzyl-7-(2-methyl-2H-tetrazol-5-yl)-9H-pyrimido[4,5-b]indol-4-yl)cyclohexane-1,4-diamine dihydrobromide dihydrate | Orphan Designation | - |
| immune reconstitution inflammatory syndrome | Hematopoietic stem cells and blood progenitors umbilical cord-derived expanded with (1r, 4r)-N1-(2-benzyl-7-(2-methyl-2H-tetrazol-5-yl)-9H-pyrimido[4,5-b]indol-4-yl)cyclohexane-1,4-diamine dihydrobromide dihydrate | Orphan Designation | - |
| major depressive disorder | Hematopoietic stem cells and blood progenitors umbilical cord-derived expanded with (1r, 4r)-N1-(2-benzyl-7-(2-methyl-2H-tetrazol-5-yl)-9H-pyrimido[4,5-b]indol-4-yl)cyclohexane-1,4-diamine dihydrobromide dihydrate | Orphan Designation | - |
| post-treatment Lyme disease syndrome | Hematopoietic stem cells and blood progenitors umbilical cord-derived expanded with (1r, 4r)-N1-(2-benzyl-7-(2-methyl-2H-tetrazol-5-yl)-9H-pyrimido[4,5-b]indol-4-yl)cyclohexane-1,4-diamine dihydrobromide dihydrate | Orphan Designation | - |
| treatment-refractory schizophrenia | Hematopoietic stem cells and blood progenitors umbilical cord-derived expanded with (1r, 4r)-N1-(2-benzyl-7-(2-methyl-2H-tetrazol-5-yl)-9H-pyrimido[4,5-b]indol-4-yl)cyclohexane-1,4-diamine dihydrobromide dihydrate | Orphan Designation | - |