FDA approves dabrafenib plus trametinib and liquid formulations to treat BRAF V600E low-grade glioma


Pediatric patients with BRAF V600E low-grade glioma as young as 1 year can now be treated with dabrafenib plus trametinib following its FDA approval.

FDA Approval | Image Credit: © Calin - © Calin - stock.adobe.com.

FDA Approval | Image Credit: © Calin - © Calin - stock.adobe.com.

The FDA recently approved dabrafenib plus trametinib (Tafinlar plus Mekinist; Novartis) for the treatment of low-grade glioma (LGG)with a BRAF V600E mutation that requires systemic therapy in pediatric patients aged 1 year and older, according to a press release from Novartis.

In addition, the FDA also approved dabrafenib plus trametinib for liquid formations, easing the administration process across approved indications. According to the release, this is the first time a BRAF/MEK inhibitor has been developed in a formulation suitable for patients as young as 1 year. The approvals make dabrafenib + trametinib the first and only approved combination targeted therapy to treat pediatric patients with BRAF V600E LGG, according to Novartis.

By blocking signals associated with the BRAF and MEK kinases that are implicated in the growth of various cancers, dabrafenib plus trametinib can help slow tumor growth. Dabrafenib plus trametinib has been studied in over 6000 BRAF-positive patients in more than 20 ongoing and completed trials and has been prescribed to over 200,000 patients worldwide.

The approval is based on the phase 2 and 3 TADPOLE trial (NCT02684058), which saw an overall response rate (ORR) of 47% (CI: 35 to 59%) and median progression-free survival (mPFS) of 20.1 months (CI: 12.8 months–not estimable) for dabrafenib plus trametinib compared to 11% ORR (CI: 3 to 25%) and 7.4 months mPFS with chemotherapy (CI: 3.6 to 11.8 months, hazard ratio=0.31 [CI: 0.17 to 0.55] [P < 0.001]).

“Pediatric cancer research is vital to uncover new treatment methods for a population,” said Eric Bouffet, MD, FRCPC, principal investigator of the TADPOLE trial, and associate scientist emeritus at The Hospital for Sick Children (SickKids). “Developing targeted therapies based on the unique genetic features of a patient’s tumor is the future of pediatric cancer care.”

The safety profile for dabrafenib plus trametinib observed in its study was consistent with other approved indications’ known safety profiles. The most common adverse reactions (≥ 15%) included pyrexia (68%), rash (51%), headache (47%), vomiting (34%), musculoskeletal pain (34%), fatigue (33%), diarrhea (29%), dry skin (26%), nausea (25%), hemorrhage (25%), abdominal pain (25%), dermatitis acneiform (22%), dizziness (15%), upper respiratory tract infection (15%), and an increase in weight (15%).

Dabrafenib plus trametinib is now FDA-approved across 6 indications for multiple BRAF V600E solid tumors, including melanoma, thyroid cancer, and lung cancer.


Novartis Tafinlar + Mekinist approved by FDA for pediatric patients with BRAF V600E low-grade glioma, the most common pediatric brain cancer. Novartis. March 17, 2023. Accessed March 24, 2023. https://www.novartis.com/news/media-releases/novartis-tafinlar-mekinist-approved-fda-pediatric-patients-braf-v600e-low-grade-glioma-most-common-pediatric-brain-cancer

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