FDA Approves Dabrafenib Plus Trametinib for Anaplastic Thyroid Cancer with BRAF V600E Mutation

Approval is based on results from the BRF117019 study that enrolled patients with rare cancers with BRAF V600E mutation

On 4 May 2018, the US Food and Drug Administration (FDA) approved dabrafenib (TAFINLAR®, Novartis Pharmaceuticals Corp.) and trametinib (MEKINIST®, Novartis Pharmaceuticals Corp.) in combination for the treatment of patients with locally advanced or metastatic anaplastic thyroid cancer with BRAF V600E mutation and with no satisfactory locoregional treatment options. 

Approval was based on a nine-cohort, non-randomised trial, BRF117019 (NCT02034110) enrolling patients with rare cancers with the BRAF V600E mutation, including locally advanced, unresectable, or metastatic anaplastic thyroid cancer with no locoregional treatment options. 

The overall response rate was 61% (95% CI: 39%, 80%) in 23 patients with anaplastic thyroid cancer who were evaluable for response. The complete and partial response rates were 4% and 57%, respectively. Response duration was at least 6 months in 64% of responding patients. 

The adverse reaction profile among all patients in the trial and among patients in the anaplastic thyroid cancer cohort was similar to that observed in other approved indications. 

The recommended doses for anaplastic thyroid cancer are 150 mg of dabrafenib orally twice daily and 2 mg of trametinib orally once daily. Full prescribing information for dabrafenib is available here and for trametinib here

FDA granted this application priority review. FDA also granted breakthrough designation and orphan drug designation for the combination of dabrafenib and trametinib in the anaplastic thyroid cancer with BRAF V600 mutation indication. 

Healthcare professionals should report all serious adverse events suspected to be associated with the use of any medicine and device to FDA’s MedWatch Reporting System.