Novartis' Targeted Therapy Tafinlar + Mekinist Reduces Risk of Disease Progression by 69% in Pediatric Low-Grade Glioma, Poised to Redefine Standard of Care

Recently, Novartis announced the phase 2/3 clinical trial results of the combination therapy consisting of the BRAF inhibitor Tafinlar and the MEK inhibitor Mekinist for treating pediatric low-grade glioma (LGG) with BRAF V600 mutations at the ASCO Annual Meeting. The trial results showed that Tafinlar/Mekinist significantly improved the overall response rate compared to standard chemotherapy and reduced the risk of disease progression or death in patients. The press release noted that if approved, the Tafinlar/Mekinist combination could become a new standard of care for these pediatric brain tumor patients, offering an oral treatment option that may improve quality of life.

Low-grade gliomas are the most common pediatric brain tumors, with BRAF V600 mutations occurring in 15-20% of pediatric low-grade gliomas. The current standard systemic treatment is chemotherapy, which is associated with poor prognosis and high treatment burden.

Tafinlar/Mekinist combination slows tumor growth by inhibiting signaling associated with BRAF and MEK kinases. This combination has been used in more than 20 clinical trials to treat over 6,000 BRAF-positive patients.

The results of this Phase 2/3 open-label clinical trial showed:

The overall response rate was 47% in the Tafinlar/Mekinist group and 11% in the chemotherapy group (p<0.001).

Moreover, at a median follow-up time of 18.9 months, the median progression-free survival (PFS) was 20.1 months in the Tafinlar/Mekinist group and 7.4 months in the chemotherapy group (HR=0.31, p<0.001).

Tafinlar/Mekinist group: 86% of patients had tumor shrinkage or stable disease, compared to 46% in the chemotherapy group.

"BRAF V600 low-grade glioma carries a risk of neurological deficits, and the current standard treatment requires intravenous infusion, which means patients need to frequently visit cancer clinics or hospitals for treatment. These young patients and their families endure a heavy treatment burden," said Mr. Jeff Legos, Global Head of Oncology and Hematology Development at Novartis. "The Tafinlar/Mekinist combination has demonstrated unprecedented efficacy, and we will work with health authorities to bring these patients a more effective and easier-to-administer liquid oral treatment option as soon as possible."

References:

[1] Novartis Tafinlar® (dabrafenib) + Mekinist® (trametinib) demonstrates unprecedented efficacy in pediatric patients with BRAF V600 low-grade gliomas in Phase II/III study. Retrieved June 7, 2022, from https://www.novartis.com/news/media-releases/novartis-tafinlar-dabrafenib-mekinist-trametinib-demonstrates-unprecedented-efficacy-pediatric-patients-braf-v600-low-grade-gliomas-phase-iiiii-study

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