205P Nirogacestat pediatric compassionate use program: Cross-sectional data review

Desmoid tumors (DT) are clinically impactful and rare, especially in the pediatric population; there are currently no published guidelines nor approved systemic treatments for pediatric DT. Nirogacestat (niro), an oral, targeted gamma secretase inhibitor and the only US FDA- and European Commission-approved treatment for adults with progressing DT who require systemic treatment, is under investigation in pediatric patients (pts) in a phase 2 clinical trial (NCT04195399). Meanwhile, eligible pediatric pts may obtain access to niro through the compassionate use program (CUP) if they cannot be treated satisfactorily with, or have exhausted, all other treatment options.