STAMFORD, Conn., Oct. 21, 2025 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc., a healthcare company of Merck KGaA, Darmstadt, Germany, announced today that long-term efficacy and safety data from the Phase 3 DeFi trial of OGSIVEO® (nirogacestat), an oral gamma secretase inhibitor for the treatment of adults with progressing desmoid tumors who require systemic treatment, were published in the Journal of Clinical Oncology (JCO). The long-term follow-up data from DeFi, which was a global, randomized, multi ...

Core Insights - Nirogacestat is poised to become the first and only approved therapy in the EU for desmoid tumors if the European Commission grants marketing authorization, with a decision expected in Q3 2025 [1][2] Company Overview - SpringWorks Therapeutics, Inc. is a biopharmaceutical company focused on severe rare diseases and cancer, currently advancing nirogacestat for desmoid tumors [2][10] - The company has previously received Orphan Drug designation for nirogacestat from the European Commission [3] Clinical Trial Results - The positive opinion from the EMA's CHMP is based on the Phase 3 DeFi trial, which demonstrated a 71% lower risk of disease progression for nirogacestat compared to placebo [3][6] - The DeFi trial involved 142 patients, with nirogacestat showing significant improvements in progression-free survival, objective response rate, and patient-reported outcomes [6] Safety Profile - Nirogacestat has a manageable safety and tolerability profile, with common adverse reactions including diarrhea, nausea, and fatigue [4] Disease Context - Desmoid tumors are rare, aggressive tumors with an estimated 1,300-2,300 new cases diagnosed annually in the EU, predominantly affecting individuals aged 20 to 44 [7][8] - Despite not metastasizing, desmoid tumors have high recurrence rates, leading to a shift in treatment guidelines recommending systemic therapies as first-line interventions [8]