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FDA Grants Breakthrough Therapy Designation to Keytruda for Advanced Colorectal Cancer
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The FDA granted breakthrough therapy designation to pembrolizumab for treatment of patients with microsatellite instability high metastatic colorectal cancer.
The agency based its decision on results from a phase 2 study designed to evaluate the activity of pembrolizumab (Keytruda, Merck), an anti–PD-1 monoclonal antibody, based on microsatellite instability status in patients with previously treated advanced colorectal cancers.
The average tumor has dozens of mutations. Tumors with DNA mismatch repair deficiency, however, can harbor thousands of mutations — particularly in regions of repetitive DNA known as microsatellites.
Tumors with mutations in select microsatellite sequences are characterized as DNA mismatch repair-deficient. These tumors are considered microsatellite instability high.
Mismatch repair deficiency is observed in an estimated 15% to 20% of non-hereditary colorectal cancers, as well as in most hereditary colorectal cancers associated with Lynch syndrome.
Merck plans to conduct a phase 3 study of pembrolizumab in treatment-naive patients with microsatellite instability high colorectal cancer.
“We are committed to understanding the full potential of Keytruda to help patients with a broad range of difficult-to-treat cancers,” Roger M. Perlmutter, MD, PhD, president of Merck Research Laboratories, said in a press release. “The data investigating the use of Keytruda in patients with advanced colorectal cancer whose tumors have substantial evidence of mismatch DNA repair defects have been encouraging, and we appreciate the opportunity that this FDA breakthrough therapy designation provides us to accelerate our effort to bring Keytruda to these patients.”
Breakthrough therapy designation is intended to expedite the development and review of agents in development for treatment of serious or life-threatening diseases when preliminary clinical evidence indicates it may provide substantial improvement over existing therapies.
The FDA in October approved pembrolizumab for use in previously treated patients with metastatic non–small cell lung cancer whose tumors express PD-L1. Last year, the FDA granted accelerated approval to pembrolizumab for treatment of certain patients with advanced or unresectable melanoma who progressed on prior therapies.