Novartis’ Tafinlar/Mekinist combo scores new indication in pediatric brain cancer

Novartis’ Tafinlar (dabrafenib) and Mekinist (trametinib) combo has been approved for another indication by the FDA — this time for treatment of low-grade glioma with a BRAF V600E mutation in kids aged 1 year and up.

This marks the sixth approval for the combination. It’s already approved for use in multiple BRAF V600E solid tumors, including melanoma, thyroid cancer and lung cancer.

Liquid formulations for both drugs were also approved by the FDA, which Novartis said is the first time a BRAF/MEK inhibitor has been developed in a formulation suitable for patients as young as one.

According to Novartis, low-grade glioma is the most common pediatric brain cancer, while BRAF V600 mutations are present in 15% to 20% of pediatric low-grade glioma cases.

Reshema Kemps-Polanco

“This new indication for Tafinlar and Mekinist is a potential new standard of care treatment option for young patients with this form of brain cancer with a BRAF V600E mutation, in formulations specifically designed for them,” Reshema Kemps-Polanco, executive VP of US oncology at Novartis, said in a statement.

The FDA issued its approval based on results from Phase II/III trials that showed patients on the combo had an improvement in overall response rate of 47% compared to 11% of patients on chemotherapy.

Progression-free survival was better for patients on the combination, too. After 18.9 months, median progression-free survival for patients on the combination was 20.1 months compared to 7.4 months just on chemotherapy (p=<0.001).

The drugs brought in $1.7 million in 2022, according to Novartis.

In 2022, Tafinlar and Mekinist broadened their label widely by adding any solid tumor in patients with BRAF V600E mutations whose cancers are unresectable or metastatic. The combo was approved for patients as young as six.

Mekinist first hit the market in 2013 for advanced melanoma. GSK, which owned the drug at the time, then paired Mekinist with Tafinlar for a 2014 approval in the same indication. A few months after that combo approval, GSK sold it (and most of the rest of its oncology portfolio) to Novartis. Novartis paid roughly $16 billion, completing the deal in March 2015.

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