The US Food and Drug Administration approved nirogacestat (Ogsiveo, SpringWorks Therapeutics, Inc) to treat progressive, unresectable, recurrent, or refractory desmoid tumors in adults.
Nirogacestat, an oral gamma secretase inhibitor, is the first medication approved to treat desmoid tumors. These rare nonmetastatic but locally invasive soft tissue tumors are most common in premenopausal women and associated with pain and functional impairment. Standard treatment options include radiation, chemotherapy, targeted therapies, and surgery, although recurrence rates are high after surgery.
Approval was based on the DeFi trial from nirogacestat maker SpringWorks.
"Nirogacestat provided clinically meaningful improvements" for patients, company executive Jim Cassidy, MD, PhD, said in a press release announcing the results. "We look forward to the opportunity to bring this important new medicine to the desmoid tumor community."
DeFi randomly assigned 70 patients with desmoid tumors to nirogacestat 150 mg twice daily and 72 to placebo. To be eligible, tumors had to have grown at least 20% in the previous year.
At a median follow-up of 15.9 months, progression-free survival was 83% with nirogacestat vs 49% with placebo: a 71% lower risk for progression or death (hazard ratio, 0.29; P < .001)
The likelihood of being event-free at 2 years was 76% with nirogacestat vs 44% with placebo; the objective response rate was 41% in the nirogacestat arm vs 8% in the placebo group.
