Long-term treatment with nirogacestat resulted in an objective response rate of 45.7%. Safety findings showed a decrease in the incidence and severity of treatment-emergent adverse events over time.
Study
|
Phase III placebo-controlled trial [DeFi; NCT03785964] |
| Adults with progressing desmoid tumors |
| Nirogacestat 150 mg twice daily continuing on open-label extension (n=39)
|
Efficacy
|
ORR: 45.7% among pts who continued for 4 yrs
|
Safety
|
Grade >=3 AEs: Diarrhea (Not reported after year 3), Ovarian toxicity (3 events after 3 years in patients with previously resolved OT), Nonmelanoma skin cancers: 2 events reported after primary analysis.
|
J Clin Oncol. Published online October 20, 2025
Schöffski PF, Ratan R, Kasper B New Reference: Nirogacestat for Desmoid Tumors
http://doi.org/10.1200/JCO-25-00582
Reviewed by Ulas D. Bayraktar, MD on Nov 13, 2025
