Bioanalysis Zone

FDA approval for first cancer treatment targeting solid tumors based on genetic characteristics


Merck’s blockbuster drug Keytruda (pembrolizumab) has been approved by the FDA as the first cancer treatment for any solid tumors on the basis of genetic characteristics, regardless of the specific type or originating location.

Keytruda, a PD-1 inhibitor, received an accelerated approval from the FDA for the treatment of solid tumors with microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) biomarkers. The presence of these biomarkers is characteristic of cancer cells being unable to repair DNA errors.

In the press release from Merck, Roger M. Perlmutter, president of Merck Research Laboratories (NJ, USA), stated: “The FDA’s approval of this first-of-its-kind, tumor-agnostic, indication is further evidence of Merck’s commitment to helping people with difficult-to-treat cancers and to advancing the use of biomarkers to guide clinical decision-making.”

The use of Keytruda will include any solid tumors with MSI-H or dMMR biomarkers that are no longer responding to any alternative treatment. Although the most commonly found tumors with MSI-H or dMMR are associated with colorectal, endometrial and gastrointestinal cancers, these biomarkers have also been observed in other types of cancer, for example, breast cancer.

Speaking on the significance of this landmark decision, Luis A. Diaz (Memorial Sloan Kettering Cancer Center; NY, USA) commented: “This is the first time in the history of oncology that a cancer medicine has been approved by the FDA using a pan-tumor predictive biomarker rather than a tumor-specific approach.”

He added: “This approval for KEYTRUDA is a transformational milestone in our progress toward personalized immunotherapy, offering certain patients with difficult-to-treat cancers a medicine based on the genetic makeup of the tumor – regardless of tumor type.”







Leave A Comment