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February 2018

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

Author Affiliations
  • 1Division of Hematology Oncology, Knight Cancer Institute, Oregon Health & Science University, Portland, Oregon
  • 2Department of Public Health and Preventive Medicine, Oregon Health & Science University, Portland, Oregon
  • 3Myeloma Service, Division of Hematologic Oncology, Department of Medicine, Memorial Sloan Kettering Cancer Center, New York, New York
JAMA Oncol. 2018;4(2):157-158. doi:10.1001/jamaoncol.2017.4182

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.

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