Novartis Announces FDA Approval of First BRAF/MEK Inhibitor Combination for Pediatric Low-Grade Glioma with BRAF V600E Mutation

On March 16, Novartis announced that the FDA had approved the listing application for Tafinlar (dabrafenib) in combination with Mekinist (trametinib) for the treatment of pediatric patients aged 1 year and older with BRAF V600E-mutated low-grade glioma (LGG) who require systemic therapy. At the same time, the FDA also approved the liquid formulation of Tafinlar + Mekinist. According to the Novartis press release, this is the first and only combined targeted therapy approved for the treatment of pediatric patients with BRAF V600E LGG.

This approval is based on the results of the Phase II/III clinical trial TADPOLE for Tafinlar + Mekinist. This is a randomized, open-label Phase II/III study designed to evaluate the efficacy of Tafinlar + Mekinist in pediatric and adolescent patients with BRAF V600-mutant low-grade glioma (LGG) or recurrent/refractory high-grade glioma (HGG), with a total of 110 patients with BRAF V600-mutant LGG enrolled.

The results showed that, compared with standard chemotherapy, the Tafinlar+Mekinist group significantly improved the overall response rate (ORR) in patients with LGG and reduced the risk of disease progression or death.

Specifically, the ORR was 47% in LGG patients treated with Tafinlar + Mekinist and 11% in those randomized to chemotherapy, showing a statistically significant improvement. At a median follow-up of 18.9 months, the median progression-free survival (mPFS) was 20.1 months in the Tafinlar + Mekinist group versus 7.4 months in the chemotherapy group. In terms of safety, compared with chemotherapy, the probability of grade ≥3 adverse events in the Tafinlar + Mekinist group was reduced by 47%, and the likelihood of treatment discontinuation due to adverse reactions decreased by 14%.

Tafinlar is a BRAF inhibitor, and Mekinist is a MEK1/2 inhibitor. The combination of the two can slow tumor growth by blocking signals associated with BRAF and MEK kinases. This FDA approval for the treatment of pediatric patients with BRAF V600E LGG is the sixth indication granted to Tafinlar + Mekinist, which has previously been approved for various BRAF V600 solid tumors, including melanoma, thyroid cancer, and lung cancer.

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