4 May 2018 - The U.S. FDA approved Tafinlar (dabrafenib) and Mekinist (trametinib), administered together, for the treatment of anaplastic thyroid cancer that cannot be removed by surgery or has spread to other parts of the body (metastatic), and has a type of abnormal gene, BRAF V600E (BRAF V600E mutation-positive).

The efficacy of Tafinlar and Mekinist in treating anaplastic thyroid cancer was shown in an open-label clinical trial of patients with rare cancers with the BRAF V600E mutation. Data from trials in BRAF V600E mutation-positive, metastatic melanoma or lung cancer and results in other BRAF V600E mutation-positive rare cancers provided confidence in the results seen in patients with ATC. The trial measured the percent of patients with a complete or partial reduction in tumour size (overall response rate). Of 23 evaluable patients, 57% experienced a partial response and 4% experienced a complete response; in nine (64%) of the 14 patients with responses, there were no significant tumour growths for six months or longer.

The FDA granted priority review and breakthrough therapy designation for this indication. Orphan drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases, was also granted for this indication.

Read FDA press release