FGFR Inhibition Shuts Down Tumor Growth in SDHd GIST, Offering a New Therapeutic Avenue

In preclinical models and a Phase II trial of rogaratinib in advanced SDHd GIST, FGFR inhibitors demonstrated significant tumor growth suppression and promising clinical activity with a 41.7% partial response rate and a median PFS of 31.1 months, regardless of SDH subunit alteration, alongside manageable toxicity profiles.

Targeting aberrant FGFR signaling driven by epigenetic dysregulation in SDHd GIST may provide a powerful, class-wide treatment strategy, opening new therapeutic options for patients with these challenging tumors.