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Molecular Profile | APC mutant |
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 mutant | desmoid tumor | predicted - sensitive | Nirogacestat | Phase III | Actionable | In a Phase III trial (DeFi), Ogsiveo (nirogacestat) treatment resulted in improved progression-free survival (HR=0.21, p=0.016) and improved objective response rate (38% (5/13) vs. 13% (2/16)) compared to treatment with placebo in patients with desmoid tumors harboring mutations in APC (J Clin Oncol 42, 2024 (suppl 16; abstr 11558); NCT03785964). | detail... |
PubMed Id | Reference Title | Details |
---|---|---|
Efficacy and safety of nirogacestat in patients with desmoid tumor and adenomatous polyposis coli (APC) mutation: Phase 3 DeFi analyses. | Full reference... |