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.
Prasad V, Kaestner V, Mailankody S. Cancer Drugs Approved Based on Biomarkers and Not Tumor Type—FDA Approval of Pembrolizumab for Mismatch Repair-Deficient Solid Cancers. JAMA Oncol. 2018;4(2):157–158. doi:10.1001/jamaoncol.2017.4182
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