Keytruda Indicated for BCG-Unresponsive High-Risk, Non–Muscle Invasive Bladder Cancer

February 2020 Vol 13, Special Issue: Payers' Perspectives in Oncology - Drug Updates, FDA Approvals

On January 8, 2020, the FDA accelerated the approval of a new indi­cation for pembrolizumab (Keytruda; Merck & Co) for the treatment of Bacillus Calmette-Guérin (BCG)-unresponsive, high-risk, non–muscle invasive bladder cancer and carcinoma in situ, with or without papillary tumors, in patients who are ineligible for or who have elected not to undergo cystectomy. Pembrolizumab has been previously approved for many indications.

The approval was based on efficacy results from the KEYNOTE-057 clinical trial, a multicenter, single-arm study involving 148 patients with high-risk non–muscle invasive bladder cancer, of whom 96 had BCG-­unresponsive carcinoma in situ with or without papillary tumors. Patients received pembrolizumab 200 mg every 3 weeks until unacceptable toxicity, persistent or recurrent high-risk disease, or progressive disease, or up to 24 months of therapy without disease progression.

The major efficacy outcomes were complete response, urine cytology, computed tomography urography, and duration of response. The complete response rate in the 96 patients with high-risk BCG-unresponsive disease with carcinoma in situ was 41% (95% confidence interval, 31-51), and the median response duration was 16.2 months. Overall, 46% of responding patients had a complete response lasting ≥12 months.

The most common (≥10%) adverse reactions with pembrolizumab in this study were fatigue, diarrhea, rash, pruritis, musculoskeletal pain, hematuria, cough, arthralgia, nausea, constipation, urinary tract infection, peripheral edema, hypothyroidism, and nasopharyngitis.

Related Items
Nubeqa Now Indicated for Metastatic Hormone-Sensitive Prostate Cancer
Web Exclusives published on December 14, 2022 in FDA Approvals
Imfinzi Receives New FDA Indication for Advanced or Metastatic Biliary Tract Cancer
Web Exclusives published on December 7, 2022 in FDA Approvals
Retevmo Receives New FDA Indication for Advanced Solid Tumors with RET Fusion
Web Exclusives published on November 30, 2022 in FDA Approvals
Imbruvica Now Also Indicated for Children with Chronic Graft-versus-Host Disease
Web Exclusives published on November 17, 2022 in FDA Approvals
Pemazyre Receives New Indication for FGFR1-­Positive Myeloid/Lymphoid Neoplasms
Web Exclusives published on November 17, 2022 in FDA Approvals
Last modified: August 30, 2021
Copyright © The Lynx Group, LLC. All rights reserved.