Fibroblast growth factor receptor inhibition for succinate dehydrogenase-deficient gastrointestinal stromal tumors: a phase 2 trial

Nature Medicine, Published online: 26 May 2026; doi:10.1038/s41591-026-04376-9

In a multicenter phase 2 trial, the fibroblast growth factor receptor inhibitor rogaratinib showed encouraging clinical efficacy in patients with succinate dehydrogenase-deficient gastrointestinal stromal tumors, suggesting a potential new treatment option for this patient population and demonstrating that an epigenetic mechanism of oncogene activation can be successfully targeted with a tyrosine kinase inhibitor.

Source: www.nature.com