Rahway: Merck, known as MSD outside of the United States and Canada, has announced that the U.S. Food and Drug Administration (FDA) has approved KEYTRUDA, Merck’s anti-PD-1 therapy, in combination with gemcitabine and cisplatin, for the treatment of patients with locally advanced unresectable or metastatic biliary tract cancer (BTC).
The approval was based on results from the Phase 3 KEYNOTE-966 trial, in which KEYTRUDA plus chemotherapy demonstrated a statistically significant improvement in the study’s primary endpoint of overall survival (OS), reducing the risk of death by 17% (HR=0.83 [95% CI, 0.72-0.95]; one-sided p=0.0034) compared to chemotherapy alone at the trial’s pre-specified final analysis for OS. Median OS was 12.7 months (95% CI, 11.5-13.6) for KEYTRUDA plus chemotherapy versus 10.9 months (95% CI, 9.9-11.6) for chemotherapy alone. This approval marks the sixth indication for KEYTRUDA in gastrointestinal cancers.

Biliary tract cancer (BTC) is a group of rare and highly aggressive cancers in the liver, gallbladder and bile ducts. Biliary tract cancer is the second most common type of primary liver cancer after hepatocellular carcinoma (HCC), accounting for approximately 15% of all liver cancers. In the U.S., there are about 20,000 patients diagnosed with BTC each year. Biliary tract cancer is most frequently diagnosed in patients between 50 and 70 years old, and approximately 70% of BTC patients are diagnosed at an advanced stage. Patients diagnosed with BTC face a very poor prognosis, with a five-year relative survival rate of 2-3% for those diagnosed with advanced disease and 9-11% for those diagnosed across all stages.
KEYTRUDA is an anti-programmed death receptor-1 (PD-1) therapy that works by increasing the ability of the body’s immune system to help detect and fight tumor cells. KEYTRUDA is a humanized monoclonal antibody that blocks the interaction between PD-1 and its ligands, PD- L1 and PD-L2, thereby activating T lymphocytes which may affect both tumor cells and healthy cells.
“Cancers of the biliary tract can be highly aggressive tumors, underscoring the need for additional treatment options for the growing number of patients facing this challenging disease,” said Dr. Robin Kate Kelley, professor of clinical medicine in the division of hematology/oncology, University of California, San Francisco. “The approval of pembrolizumab in combination with chemotherapy offers patients with locally advanced unresectable or metastatic biliary tract cancer a new immunotherapy regimen that has demonstrated the potential to help these patients live longer.”
“Many patients with biliary tract cancer are diagnosed with locally advanced or metastatic disease, at which point they are not eligible for surgery and face poor survival outcomes with limited treatment options,” said Dr. Marjorie Green, senior vice president and head of late-stage oncology, global clinical development, Merck Research Laboratories. “With this approval, Merck is proud to offer a new treatment option to certain patients with locally advanced unresectable or metastatic biliary tract cancer, and their healthcare providers, that has shown an overall survival benefit compared to chemotherapy alone.”
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