Nirogacestat is primarily used in the treatment of desmoid tumors, a rare type of non-metastatic soft tissue tumor. It functions as a gamma-secretase inhibitor, which blocks the Notch signaling pathway—this pathway is often overactive in desmoid tumors and contributes to uncontrolled cell growth. By inhibiting this pathway, nirogacestat helps reduce tumor progression and can lead to tumor shrinkage in some patients. It has shown promising results in clinical trials, particularly for patients with progressive, inoperable, or recurrent desmoid tumors who have limited treatment options. Due to its targeted mechanism, it offers a more specific therapeutic approach compared to traditional chemotherapy, potentially reducing side effects and improving quality of life. Nirogacestat is being investigated in advanced clinical development and may become a key option in precision oncology for rare tumors driven by Notch signaling.