Author Correction: The type II RAF inhibitor tovorafenib in relapsed/refractory pediatric low-grade glioma: the phase 2 FIREFLY-1 trial

BRAF genomic alterations are the most common oncogenic drivers in pediatric low-grade glioma (pLGG). Arm 1 (n = 77) of the ongoing phase 2 FIREFLY-1 (PNOC026) trial evaluated the efficacy of the oral, selective, central nervous system-penetrant type II RAF inhibitor tovorafenib (420 mg/m² once weekly; 600 mg maximum dose) in patients with BRAF-altered, relapsed/refractory pLGG. Arm 2 (n = 60) served as an extension cohort, providing treatment access for patients with RAF-altered pLGG following the closure of Arm 1. Based on independent review using the Response Assessment in Neuro-Oncology High-Grade Glioma (RANO-HGG) criteria, the overall response rate (ORR) for Arm 1 was 67%, meeting the prespecified primary endpoint. The median duration of response (DOR) was 16.6 months, and the median time to response (TTR) was 3.0 months (secondary endpoints). Other key secondary endpoints for Arm 1 included ORR, DOR, and TTR as assessed by the Response Assessment in Pediatric Neuro-Oncology Low-Grade Glioma (RAPNO) criteria, as well as safety (assessed in all treated patients, including those in Arm 2, n = 137). According to RAPNO criteria (including minor responses), the ORR was 51%, with a median DOR of 13.8 months and median TTR of 5.3 months. The most common treatment-related adverse events (TRAEs) included hair color changes (76%), elevated creatine phosphokinase (56%), and anemia (49%). Grade ≥3 TRAEs occurred in 42% of patients, and 9 (7%) patients experienced TRAEs that led to discontinuation of tovorafenib. These findings suggest that tovorafenib may offer MLN2480 an effective treatment option for patients with BRAF-altered, relapsed/refractory pLGG.