The FDA approved nirogacestat (Ogsiveo, SpringWorks Therapeutics) for adult patients with progressing desmoid tumors who require systemic treatment. This is the first approved treatment for desmoid tumors, a rare subtype of soft tissue sarcomas.
Efficacy was evaluated in DeFi (ClinicalTrials.gov Identifier: NCT03785964), an international, multicenter, randomized (1:1), double-blind, placebo-controlled trial in 142 patients with progressing desmoid tumors not amenable to surgery. Patients were
NOVEMBER 28, 2023