FDA Grants Priority Review for Pembrolizumab in Patients with High-Risk, Non-Muscle Invasive Bladder Cancer


The application is based on results from the phase II KEYNOTE-057 trial, which found that pembrolizumab has encouraging activity in patients with high-risk, BCG-unresponsive CIS with or without papillary tumors.

The FDA has accepted and granted priority review for a new supplemental Biologics License Application for pembrolizumab (Keytruda, Merck), an anti-PD-1 therapy.

The application seeks approval of pembrolizumab monotherapy for the treatment of patients with Bacillus Calmette-Guerin (BCG)-unresponsive, high-risk, non-muscle invasive bladder cancer (NMIBC) with carcinoma in-situ (CIS) with or without papillary tumors who are ineligible for or have elected not to undergo cystectomy. This application is based on results from the phase II KEYNOTE-057 trial.

KEYNOTE-057 is a single-arm phase 2 study for patients with BCG-unresponsive NMIBC who were unwilling or unable to undergo radical cystectomy. A total of 102 patients met the study’s criteria, a median age of 73 years. The 3-month complete response rate was 40.2%. Among 41 patients who achieved a complete response at 3 months, 58.5% maintained a complete response at last follow-up, or a median of 16.7 months. Only 13 patients suffered Grade 3/4 treatment-related adverse events, and 1 death was considered treatment-related.

It is estimated that more than 80,000 new cases of bladder cancer are diagnosed with NMIBC. For high-risk NMIBC patients who are BCG-unresponsive with persistent or recurrent disease, treatment guidelines recommend radical cystectomy.

KEYNOTE-057 found that pembrolizumab has encouraging activity in patients with high-risk, BCG-unresponsive CIS with or without papillary tumors.

“Patients with high-risk, non-muscle invasive bladder cancer sometimes make an informed decision to decline, or are medically ineligible for radical cystectomy, and there are currently limited non-surgical treatment options approved by the FDA for these patients who are BCG-unresponsive,” said Roy Baynes, MD, chief medical officer of Merck Research Laboratories. “We look forward to participating in the advisory committee meeting and to continuing to work with the FDA as they review this supplemental application for [pembrolizumab].”

Data from this trial were first presented at the European Society for Medical Oncology 2019 Congress. A target action date is expected in January 2020, based on priority review.


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  • FDA Grants Priority Review to Merck’s Supplemental Biologics License Application (sBLA) for KEYTRUDA® (pembrolizumab) in Certain Patients with High-Risk, Non-Muscle Invasive Bladder Cancer (NMIBC) [press release]. Merck website. Published December 2, 2019. https://www.mrknewsroom.com/news-release/oncology/fda-grants-priority-review-mercks-supplemental-biologics-license-application-s. Accessed December 2, 2019.
  • Balar A, Kulkarni G, Uchio E, et al. Keynote 057: Phase II trial of Pembrolizumab (pembro) for patients (pts) with high-risk (HR) nonmuscle invasive bladder cancer (NMIBC) unresponsive to bacillus calmette-guérin (BCG). Published February 26, 2019. DOI: 10.1200/JCO.2019.37.7_suppl.350 Journal of Clinical Oncology 37, no. 7_suppl (March 01, 2019) 350-350. Accessed December 2, 2019.

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