5
Orphan Designations
0
FDA Approvals
15
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| B-lymphoblastic leukemia/lymphoma with t(9;22)(q34.1;q11.2) | N-[3-(4',5'-bipyrimidin-2-ylamino)-4-methylphenyl]-4-{[(3S)-3-(dimethylamino)pyrrolidin-1-yl]methyl}-3-(trifluoromethyl)benzamide | Orphan Designation | - |
| Philadelphia-positive myelogenous leukemia | N-[3-(4',5'-bipyrimidin-2-ylamino)-4-methylphenyl]-4-{[(3S)-3-(dimethylamino)pyrrolidin-1-yl]methyl}-3-(trifluoromethyl)benzamide | Orphan Designation | - |
| X-linked intellectual disability-psychosis-macroorchidism syndrome | Poloxamer 188 | Orphan Designation | - |
| acute flaccid myelitis | Tamibarotene | Orphan Designation | - |
| acute lymphoblastic leukemia | Tamibarotene | Orphan Designation | - |
| acute promyelocytic leukemia | Tamibarotene | Orphan Designation | - |
| chronic myelogenous leukemia, BCR-ABL1 positive | N-[3-(4',5'-bipyrimidin-2-ylamino)-4-methylphenyl]-4-{[(3S)-3-(dimethylamino)pyrrolidin-1-yl]methyl}-3-(trifluoromethyl)benzamide | Orphan Designation | - |
| coccidioidomycosis | Poloxamer 188 | Orphan Designation | - |
| coronary vasospasm | Poloxamer 188 | Orphan Designation | - |
| early yaws | Poloxamer 331 | Orphan Designation | - |
| intimal sarcoma | Poloxamer 331 | Orphan Designation | - |
| intracranial vasospasm | Poloxamer 188 | Orphan Designation | - |
| orofaciodigital syndrome IV | Poloxamer 188 | Orphan Designation | - |
| pulmonary hypertension, primary, 1 | Poloxamer 331 | Orphan Designation | - |
| severe Canavan disease | Poloxamer 188 | Orphan Designation | - |