Nirogacestat Beneficial for Progressing Desmoid Tumors

Significant progression-free survival benefit seen over placebo; more patients receiving nirogacestat have objective response
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Medically Reviewed By:
Mark Arredondo, M.D.

THURSDAY, March 9, 2023 (HealthDay News) -- For adults with progressing desmoid tumors, the oral γ-secretase inhibitor nirogacestat is associated with significant benefits, according to a study published in the March 9 issue of the New England Journal of Medicine.

Mrinal Gounder, M.D., from the Memorial Sloan Kettering Cancer Center in New York City, and colleagues conducted a phase 3 trial involving adults with progressing desmoid tumors according to the Response Evaluation Criteria in Solid Tumors, version 1.1. Participants were randomly assigned to either nirogacestat or placebo twice daily (70 and 72 patients, respectively).

The researchers found that compared with placebo, nirogacestat had a significant progression-free survival benefit over placebo (hazard ratio for disease progression or death, 0.29); the likelihood of being event-free at two years was 76 and 44 percent, respectively, for nirogacestat and placebo. Across prespecified subgroups, the between-group differences in progression-free survival were consistent. The percentage of patients with an objective response was significantly higher with nirogacestat (41 versus 8 percent), with a median of 5.6 and 11.1 months, respectively, to response; a complete response occurred in 7 and 0 percent of patients, respectively. In secondary patient-reported outcomes, including pain, symptom burden, physical or role functioning, and health-related quality of life, significant between-group differences were observed.

"Nirogacestat showed significant benefits with respect to progression-free survival, objective response, pain, disease-specific symptom burden, physical functioning, role functioning, and health-related quality of life," the authors write.

The study was funded by SpringWorks Therapeutics, the developer of nirogacestat.

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