Opinion|Videos|December 9, 2025

Examining a Novel FDA-approved Therapy in Desmoid Tumors

FDA approval of Nirogacistat for desmoid tumors highlights improved survival rates, quality of life, and manageable side effects for patients.

Panelists discuss the questions below in this video.

  • What was the key data that led to the FDA approval of nirogacestat in patients with desmoid tumors? How does its mechanism of action differ from other therapies? Can you describe the data from the long-term study of nirogacestat? Why is this study valuable to clinicians? From your clinical experience, how has patient satisfaction been with nirogacestat? What has long-term data demonstrated? What was found in studies that evaluated nirogacestat in patient-reported outcomes and pain control? Ovarian toxicity was seen in the clinical trials in women treated with nirogacestat. What was the outcome in these patients and is any intervention necessary?

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