Biomarker Is King In Latest US FDA Cancer Drug Approval

Accelerated approval for patients with microsatellite instability-high or mismatch repair deficient solid tumors is the first time the US regulatory agency has granted an indication that does not specify the location of the tumor – a change that precision-medicine researchers have been eager to make.

FDA's approval of Merck's Keytruda (pembrolizumab) for use in any patient with a solid tumor with a particular genetic mutation is the first of its kind and could be the herald of a new approach to treating cancer.

The agency announced the accelerated approval of the PD-1 inhibitor May 23, noting "this is the first time the agency has approved a cancer treatment based on a common biomarker

The new indication, Keytruda's ninth, is for adult and pediatric patients with unresectable or metastatic solid tumors that have been identified as having microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) and specifically in colorectal cancer patients with the biomarkers that have completed earlier lines of treatment (see box)

More from Approvals

More from Policy & Regulation