Cancer Drugs Approved Based on Biomarkers and Not Tumor Type | Cancer Biomarkers | JAMA Oncology | The JAMA Network

Cancer Drugs Approved Based on Biomarkers and Not Tumor Type | Cancer Biomarkers | JAMA Oncology | The JAMA Network

December 28, 2017

Cancer Drugs Approved Based on Biomarkers and Not Tumor Type—FDA Approval of Pembrolizumab for Mismatch Repair-Deficient Solid Cancers

Vinay Prasad, MD, MPH^1,2; Victoria Kaestner, BS¹; Sham Mailankody, MB, BS³

Author Affiliations

JAMA Oncol. Published online December 28, 2017. doi:10.1001/jamaoncol.2017.4182

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On May 23, 2017, the US Food and Drug Administration (FDA) granted accelerated approval to pembrolizumab (Keytruda, Merck & Co) for patients with unresectable or metastatic, microsatellite instable-high (MSI-H) or mismatch repair-deficient (dMMR) solid tumors that have progressed on prior therapy and have no satisfactory treatment options. This approval is, to date, the first drug authorized for use based on a molecular biomarker rather than a traditional histopathologic diagnosis. The approval bridges several themes in modern oncologic drug development: the rapid translation of high-quality basic science, tailoring therapy based on genetic subgroups, and a flexibility on the part of the FDA to authorize drugs based on early, favorable data.