FDA Approves Immunotherapy Pembrolizumab to Treat Patients with BCG-Unresponsive, Non-Muscle Invasive Bladder Cancer

San Francisco, CA (UroToday.com) -- Pembrolizumab (Keytruda®) was approved January 8, 2020, by the U.S. Food and Drug Administration (FDA) to treat patients with high-risk, non-muscle invasive bladder cancer (NMIBC) unresponsive to the treatment Bacillus Calmette-Guérin (BCG). It is the first new treatment for this type of bladder cancer in 20 years (valrubicin, 1998).

“For patients with high-risk, non-muscle invasive bladder cancer that has become unresponsive to BCG treatment, the most effective treatment option is a radical cystectomy, which includes removing the entire bladder and a prostatectomy for men or total hysterectomy in women,” says Arjun V. Balar, MD, associate professor of medicine and director of Genitourinary Medical Oncology at NYU Langone Health’s Perlmutter Cancer Center. 

“This surgery carries a substantial risk for morbidity and mortality and can negatively impact quality of life. In fact, many patients are not medically eligible for a radical cystectomy.  This approval provides a new approach for treating patients with BCG unresponsive high-risk non-muscle invasive bladder cancer, and moreover highlights the continued impact that systemic immunotherapies have had in the management of this disease.”
FDA approval was based on results from the Phase 2 KEYNOTE-057 trial. Dr. Balar was the lead investigator for this study. The main finding was 40 percent of patients had a complete response (clinically meaningful); half of those (20 percent) still had a complete response after one year. “The study demonstrates local activity in the bladder from a systemically administered treatment,” Dr. Balar notes. “This is promising since systemic immune activation is what ultimately leads to long-term durable remissions of cancer.”

Gary D. Steinberg, MD, a member of the faculty of NYU Langone urology and director of Perlmutter Cancer Center’s Urology Bladder Cancer Program, joined NYU Langone in 2019 to complement ongoing efforts in the care of patients with bladder cancer. Dr. Steinberg was a scientific advisor for Merck on the KEYNOTE-057 trial and is principal investigator for the KEYNOTE-676 Phase 3 study of BCG with or without pembrolizumab for high-risk NMIBC that is persistent or recurrent following BCG induction.

“The approval of pembrolizumab is paradigm-changing and offers patients a possible therapy to preserve their native bladder function while eliminating their cancer,” says Dr. Steinberg. “This a breakthrough for some patients and most likely will lead to new combinations of therapy for this aggressive form of localized bladder cancer that will hopefully benefit additional patients. This will lead to further research and innovative treatments for the bladder cancer community and clearly is a significant advance.”

Estimates are about 80,000 people are diagnosed with bladder cancer each year in the US. Seventy-five percent of these diagnosed bladder cancer cases are NMIBC.

WATCH Arjun Balar, MD Discuss the Data and Clinical Implications Supporting the FDA Approval for patients with NMIBC

Read: ASCO GU 2019: Updated Results of Keynote 057: Pembrolizumab for Patients with High-risk Nonmuscle Invasive Bladder Cancer Unresponsive to BCG

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