Tafinlar-Mekinist Combination Approved for Metastatic Thyroid Cancer with BRAF V600E Mutation

August 2018, Vol 8, No 8 - FDA Approvals, News & Updates


On May 4, 2018, the FDA expedited its approval of the combination of dabrafenib and trametinib (Tafinlar and Mekinist; Novartis) for the treatment of patients with locally advanced or metastatic anaplastic thyroid cancer and BRAF V600E mutation who have no appropriate locoregional therapy options.

“This is the first FDA-approved treatment for patients with this aggressive form of thyroid cancer, and the third cancer with this specific gene mutation that this drug combination has been approved to treat,” said Richard Pazdur, MD, Director of the FDA’s Oncology Center of Excellence. This drug combination is already approved for melanoma or non–small-cell lung cancer associated with specific BRAF V600E or V600K mutations.

This new indication was based on a multicenter, nonrandomized, open-label clinical trial involving patients with rare cancers and BRAF V600E mutation. Of 23 patients with locally advanced, unresectable, or metastatic anaplastic thyroid cancer who had no appropriate locoregional treatment options and were evaluable for response, 14 patients had response to treatment with the combination, including 57% partial responses and 4% complete responses. The overall response rate was 61%, and the responses lasted ≥6 months in 64% of patients. The side effects were similar to those seen in other tumors.