FDA Approves Pembrolizumab Plus Gemcitabine-Cisplatin for Unresectable, Metastatic BTC

The US Food and Drug Administration (FDA) has granted approval to KEYTRUDA (pembrolizumab) in combination with gemcitabine and cisplatin for the treatment of patients with locally advanced unresectable or metastatic biliary tract cancer (BTC).

“Many patients with BTC 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, at Merck Research Laboratories, in a press release. “With this approval, Merck is proud to offer a new treatment option to certain patients with locally advanced unresectable or metastatic BTC, and their health care providers, that has shown an overall survival (OS) benefit compared [with] chemotherapy alone.”

The approval is based on results of the KEYTNOTE-966 trial—a multicenter, double-blind, randomized, placebo-controlled, phase 3 investigation of pembrolizumab plus gemcitabine-cisplatin for patients with locally advanced unresectable or metastatic BTC who had not received prior systemic therapy in the advanced disease setting. For the 1069 patients enrolled, pembrolizumab plus chemotherapy demonstrated a statistically significant improvement in OS; the median OS was 12.7 months (95% CI, 11.5-13.6) for patients receiving pembrolizumab compared with 10.9 months (95% CI, 9.9-11.6) for those who received chemotherapy alone.

Additionally, the risk of death was reduced by 17% (hazard ratio, 0.83; 95% CI, 0.72-0.95; P=.0034) for patients receiving pembrolizumab at the trial’s prespecified final analysis for OS.

“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 Robin Kate Kelley, MD, the lead investigator of KEYNOTE-966. “Today’s approval of pembrolizumab in combination with chemotherapy offers patients with locally advanced unresectable or metastatic BTC a new immunotherapy regimen that has demonstrated the potential to help these patients live longer.”

The latest approval marks the sixth indication for pembrolizumab in gastrointestinal cancers.