• Long-term treatment with nirogacestat (Ogsiveo) demonstrated improved objective response rates reaching 45.7% at 4 years compared to 34.3% at 1 year in patients with desmoid tumors, according to updated DeFi trial data.
• Patients experienced significant quality-of-life benefits with nirogacestat, including rapid pain reduction within two weeks of treatment initiation, allowing many to discontinue opioid medications despite minimal initial changes in tumor imaging.
• While 75% of female patients experienced reversible ovarian dysfunction with nirogacestat, the study confirmed that ovarian function returned in all patients who discontinued treatment, providing important reassurance for the predominantly young female patient population.