1
Orphan Designations
0
FDA Approvals
3
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| NTRK fusion positive cancer | (R)-N-(5-(2-(2,5-difluorophenyl)pyrrolidin-1-yl)pyrazolo[1,5-a] pyrimidin-3-yl)-4-((4-methylpiperazin-1-yl)methyl)benzamide sulfate hydrate | Orphan Designation | - |
| hereditary sensory and autonomic neuropathy | (R)-N-(5-(2-(2,5-difluorophenyl)pyrrolidin-1-yl)pyrazolo[1,5-a] pyrimidin-3-yl)-4-((4-methylpiperazin-1-yl)methyl)benzamide sulfate hydrate | Orphan Designation | - |
| obesity, hyperphagia, and developmental delay | (R)-N-(5-(2-(2,5-difluorophenyl)pyrrolidin-1-yl)pyrazolo[1,5-a] pyrimidin-3-yl)-4-((4-methylpiperazin-1-yl)methyl)benzamide sulfate hydrate | Orphan Designation | - |