The Medical LetterIn Brief: A New Indication for Dabrafenib (Tafinlar) and Trametinib (Mekinist) Combination Therapy

Based on prior studies in patients with melanoma and lung cancer as well as overall response and duration of response rates in open-label, multiple cohort trials involving 131 adults and 36 children, the combination was approved for the new indication (treatment of unresectable or metastatic solid tumors with a BRAF V600E mutation in patients ≥6 years old who have progressed following prior treatment and have no satisfactory alternative treatment options). Adults with BRAF V600E mutation-positive solid tumors, such as low-grade glioma (LGG), anaplastic thyroid cancer, biliary tract cancer, small intestine cancer, and high-grade glioma (HGG), were enrolled in the new trials, along with 36 children with BRAF V600 refractory or recurrent LGG or HGG. The overall response rate (ORR) served as the trial’s main measure of success, and out of the 131 adults who participated, 54 (41%) had an objective reaction. Among the most prevalent tumor types in the clinical trials, the ORR for biliary tract cancer was 46%, HGG was 33%, and LGG was 50%. Children’s ORR was 25%.