Nirogacestat Shows Statistically Significant Improvements in Desmoid Tumors

Mrinal M. Gounder, MD, of Memorial Sloan Kettering Cancer Center in New York, New York, delves into the data supporting the recent FDA approval of nirogacestat (Ogsiveo) for the treatment of adult patients with desmoid tumors.

On November 27, 2023, the FDA granted this approval to nirogacestat based on findings from the phase 3 DeFi trial (NCT03785964). In the study, nirogacestat reduced the risk of disease progression or death by 71% vs placebo among patients with desmoid tumors (HR, 0.29; 95% CI, 0.15-0.55; P < .001). Among the 70 patients treated with nirogacestat, the Kaplan-Meier-estimated median progression-free survival (PFS) was not estimated vs 15.1 months among the 72 patients treated with placebo (95% CI, 8.4-not estimable).

For safety, grade 3 or greater adverse events were observed in 55% vs 17% of patients in the nirogacestat vs placebo arms, respectively. According to Goudner, patients given nirogacestat reported that they felt better on an objective patient reported outcomes questionnaire.

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0:10 | The best data is a global, randomized phase 3, placebo controlled study of nirogacestat vs placebo in [patients with] desmoid tumors who have progressive disease. This data has previously been presented last year at ESMO in 2022, Bernd Kasper, MD, PhD, and published in the New England Journal of Medicine earlier this year in 2023.

0:37 | The data essentially shows that first, the study's primary end point was progression-free survival, and this showed that the treatment with nirogacestat had a significantly higher improvement of median progression-free survival compared with placebo. Beyond the median progression-free survival, there were also significantly higher rates of objective response noted by tumor shrinkage.

1:08 | Lastly, patients who are on nirogacestat reported that they felt better on an objective patient reported outcomes questionnaire. So these 3 different ways of looking at these tumor responses, stopping the tumors from getting bigger, as well as the patient's themselves feeling better on this drug was captured in the publication and the presentation.