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| Molecular Profile | APC inact mut |
| Therapy | Nirogacestat |
| Indication/Tumor Type | desmoid tumor |
| Response Type | predicted - sensitive |
| Molecular Profile | Indication/Tumor Type | Response Type | Therapy Name | Approval Status | Evidence Type | Efficacy Evidence | References |
|---|---|---|---|---|---|---|---|
| APC inact mut | desmoid tumor | predicted - sensitive | Nirogacestat | Case Reports/Case Series | Actionable | In a clinical case study, Ogsiveo (nirogacestat) resulted in stable disease lasting 9 months in a patient with a patient with a desmoid tumor harboring an inactivating APC mutation (PMID: 32762028). | 32762028 |
| PubMed Id | Reference Title | Details |
|---|---|---|
| (32762028) | Safety and efficacy of gamma-secretase inhibitor nirogacestat (PF-03084014) in desmoid tumor: Report of four pediatric/young adult cases. | Full reference... |