Significance and implications of FDA approval of pembrolizumab for biomarker-defined disease

Michael M. Boyiadzis, John M. Kirkwood, John L. Marshall, Colin C. Pritchard, Nilofer S. Azad, James L. Gulley

Research output: Contribution to journalReview articlepeer-review

84 Scopus citations


The U.S. Food and Drug Administration (FDA) recently approved pembrolizumab, an anti- programmed cell death protein 1 cancer immunotherapeutic, for use in advanced solid tumors in patients with the microsatellite-high/DNA mismatch repair-deficient biomarker. This is the first example of a tissue-agnostic FDA approval of a treatment based on a patient's tumor biomarker status, rather than on tumor histology. Here we discuss key issues and implications arising from the biomarker-based disease classification implied by this historic approval.

Original languageEnglish (US)
Article number35
JournalJournal for immunotherapy of cancer
Issue number1
StatePublished - May 14 2018


  • Biomarker
  • DNA mismatch repair
  • Immunotherapy
  • Microsatellite instability
  • Pembrolizumab

ASJC Scopus subject areas

  • Immunology and Allergy
  • Immunology
  • Molecular Medicine
  • Oncology
  • Pharmacology
  • Cancer Research


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