Nirogacestat is primarily used in the treatment of desmoid tumors, which are rare, non-metastatic but locally aggressive tumors that arise in connective tissues. It functions as a gamma-secretase inhibitor, blocking the Notch signaling pathway that is often dysregulated in these tumors. By inhibiting this pathway, nirogacestat helps reduce tumor growth and can lead to tumor shrinkage in some patients.

It has shown clinical benefit in reducing tumor volume and alleviating pain associated with the disease, potentially avoiding the need for surgery or radiation. Nirogacestat is especially valuable for patients with progressive, unresectable, or recurrent desmoid tumors where treatment options have been limited. Its development represents a targeted therapy approach for a condition with high unmet medical need.