17 April 2020

To date, no company has gained approval for an immuno-oncology drug based on investigational biomarker tumour mutational burden. However, data from the phase 2 Keynote-158 trial, haveĀ persuaded the FDA to grant a priority review for the use of pembrolizumab in previously treated patients with high tumour mutational burden (TMB), regardless of where these tumours are in the body.

FDA has previously approved anti-cancer therapeutics based on biomarker targets irrespective of the type of cancer: in 2017 to pembrolizumab for tumours with microsatellite instability-high or mismatch repair deficient tumours, and to larotrectinib and entrectinib for NTRK gene fusion tumours in 2018 and 2019, respectively.

The ASCO Post