Core Insights - Long-term treatment with OGSIVEO (nirogacestat) for up to 4 years demonstrated further tumor size reductions, increased objective response rates, sustained improvement in desmoid tumor symptoms, and a consistent safety profile [1][3][5] Efficacy and Safety - The Phase 3 DeFi trial showed significant improvements in progression-free survival (PFS), objective response rate (ORR), and patient-reported outcomes (PRO) in adult patients with progressing desmoid tumors [2][6] - The median duration of OGSIVEO treatment was 33.6 months, with ORR increasing from 34.3% in year 1 to 45.7% for patients treated for up to 4 years [3][6] - The median best percent reduction in target tumor size was −32.3% at year one and −75.8% for patients completing at least four years of treatment [3] Patient-Reported Outcomes - Improvements in PROs related to pain, symptom severity, physical functioning, global health status, and role functioning were observed early and sustained through treatment [3] Adverse Events - The incidence and severity of treatment-emergent adverse events (TEAEs) decreased over the treatment duration, with common adverse events including diarrhea, ovarian toxicity, rash, and nausea [4][21] - Serious adverse reactions occurred in ≥2% of patients, with ovarian toxicity being the most notable [21] Background on Desmoid Tumors - Desmoid tumors are rare, locally aggressive tumors that can cause significant pain and functional impairment, with a prevalence of up to 1650 new cases diagnosed annually in the U.S. [7][8] - Systemic therapies are now recommended as first-line interventions for most desmoid tumor locations requiring treatment [8] About OGSIVEO - OGSIVEO is an oral gamma secretase inhibitor approved for the treatment of adult patients with progressing desmoid tumors requiring systemic treatment, with Orphan Drug designation granted by the FDA and EMA [9]

Core Insights - The European Commission has granted marketing authorization for OGSIVEO (nirogacestat), making it the first and only approved therapy in the EU for treating desmoid tumors [1][2][3] Company Overview - SpringWorks Therapeutics, a healthcare company of Merck KGaA, is focused on developing treatments for rare tumors and has successfully launched OGSIVEO as a systemic therapy for desmoid tumors in both the U.S. and Europe [1][3][15] - The company aims to improve patient outcomes and quality of life for those affected by rare tumors, highlighting its commitment to the rare tumor patient community [3][15] Industry Context - Desmoid tumors are rare, locally aggressive tumors with an annual diagnosis rate of approximately 1,300 to 2,300 cases in the EU, causing significant pain and functional limitations for patients [2][7] - The approval of OGSIVEO is seen as a significant advancement in the management of desmoid tumors, which previously had no approved treatments in Europe [2][3] Clinical Trial Insights - The approval of OGSIVEO is based on the Phase 3 DeFi trial, which demonstrated a 71% reduction in the risk of disease progression compared to placebo, with a confirmed objective response rate of 41% [3][5][6] - The trial included 142 adult patients and met its primary endpoint of improving progression-free survival [3][5] Treatment Efficacy - OGSIVEO has shown significant improvements in patient-reported outcomes, including pain reduction and overall health-related quality of life [3][4][6] - The therapy is positioned as a first-line intervention for desmoid tumors, shifting the treatment paradigm away from surgical options [2][8]