The FDA approved pimicotinib (Pimjetmi) for adults with symptomatic tenosynovial giant cell tumors (TGCT) where surgery may cause worsening functional limitations or severe morbidity. The decision was based on results from the placebo-controlled, international MANEUVER phase 3 trial. In the study of 94 patients with measurable symptomatic TGCT, pimicotinib achieved an objective response rate of 54% at week 25 versus 3% with placebo, with the primary endpoint assessed by a blinded independent review committee using RECIST v1.1. The safety profile included hepatotoxicity, embryo-fetal toxicity, and increases in serum creatinine. Merck—maker of pimicotinib—said the once-daily oral regimen is designed to address the treatment gap for patients unlikely to benefit from surgery alone, aiming to shrink tumors and improve pain and function.