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May 25, 2017

FDA approves first-ever cancer drug based on tumor genetics

Daily Briefing

FDA on Tuesday granted Merck's cancer drug Keytruda an accelerated approval to treat nonsurgical or metastatic solid tumors linked to the genetic defect known as microsatellite instability.

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Keytruda is the first drug that FDA has approved as a cancer treatment that can be used based on the presence of a certain biomarker in a tumor, rather than on the type of tissue from which a tumor originated.

According to Reuters, the approval represents a major step in treating cancer with a precision medicine approach, which centers on determining courses of treatment based on genetic biomarkers rather than specific types of cancer. Researchers hope that with precision medicine, they eventually will be able to use genetic information to determine the best treatment options for individual patients.

Keytruda details

FDA first approved Keytruda, which is designed to strengthen the immune system to help fight tumors, in 2014 to treat advanced melanoma. Keytruda was the first approved therapy that inhibits a protein that prevents the immune system from fighting off cancerous tumors.

FDA grants accelerated approval for Keytruda to treat other types of cancer

FDA on Tuesday granted accelerated approval for Keytruda to treat other forms of cancer.

Under the approval, Keytruda can be used to treat cancer in children and adults with solid tumor cancers that progressed despite prior treatment, as well as in those who do not have suitable options for alternative treatments, such as surgery. According to MedPage Today, such tumors most commonly occur in colorectal, endometrial, and gastrointestinal cancers, but also can appear in bladder, breast, and prostate cancers.

Keytruda now is approved to treat:

  • Bladder cancer;
  • Metastatic melanoma;
  • Metastatic non-small cell lung cancer;
  • Recurrent/metastatic head and neck cancer;
  • Refractory classical Hodgkin lymphoma; and
  • Urothelial carcinoma.

According to the Wall Street Journal, Keytruda costs about $13,000 per month per patient. 


Richard Pazdur of FDA's Office of Hematology and Oncology Products in a statement called Keytruda's latest approval an "important first for the cancer community" because of the treatment's novel approach to treating tumors.

Bert Vogelstein, co-director of the Ludwig Center at the Johns Hopkins Kimmel Cancer Center, said Keytruda's treatment approach, which is different than "the classical way of treating patients," has the potential to improve the treatment of tumors with less-common molecular traits.

Drew Pardoll—director of the Johns Hopkins Bloomberg-Kimmel Institute for Cancer Immunology, which led and designed the clinical trial FDA used to approve Keytruda for its latest indication—called the new approval "a marriage of cancer genetics and cancer immunology." Pardoll said about 4 percent of advanced cancers—or between 15,000 to 20,000 cases annually in the United States—have the biomarker Keytruda targets (Bankhead, MedPage Today, 5/23; Berkrot, Reuters, 5/23; Loftus, Wall Street Journal, 5/23).

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