24 May 2023

New drug improved survival of patients with desmoid tumors

Nirogacestat showed high efficacy in adult patients with desmoid tumors. Two years after starting the drug, there was no progression in 76% of the study participants.  

The selective low molecular weight gamma-secretase inhibitor nirogacestat showed promising results in phase III clinical trials for its use in the treatment of advanced desmoid tumors. The results are published on the U.S. National Cancer Institute (NCI) website.

After 16 months of follow-up, the nirogacestat group was 71% less likely to die or deteriorate than the placebo group. Two years after starting therapy, there were no signs of progression in 76% of patients in the nirogacestat group and in 44% of participants who received placebo.

The size of the neoplasms decreased in 41% of patients who received nirogacestat, compared to 8% in the control group. Complete remission was found in 7% of participants in the nirogacestat group. No such cases were found in the placebo group. Finally, patients taking nirogacestat reported decreased pain, improved physical activity, and improved quality of life.

The side effects of the drug were moderate and included mostly diarrhea and rash. Ovarian dysfunction was observed in 75% of women of childbearing age, which disappeared after discontinuation of therapy.

The study enrolled 142 adult patients, aged 18 to 76 years, who were divided into two groups. Participants in the first received oral nirogacestat twice daily, while the second received a placebo. 

The manufacturer has submitted an application for approval of the drug for the treatment of adults with desmoid tumors to the U.S. Food and Drug Administration (FDA). A response is expected in the summer.

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