The US Food and Drug Administration (FDA) granted regular approval to pembrolizumab for patients with locally advanced or metastatic urothelial carcinoma who have disease progression during or following platinum-containing chemotherapy or within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy.1

Also granted was accelerated approval for first-line use of pembrolizumab in patients with locally advanced or metastatic urothelial carcinoma ineligible for cisplatin-containing chemotherapy.

The approval for the second-line indication was based on results from the phase 3, open-label KEYNOTE 045 trial ( Identifier: NCT02256436). The study randomly assigned 542 patients to pembrolizumab 200 mg or investigator’s choice of paclitaxel, docetaxel, or vinflunine every 3 weeks.

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Median overall survival was 10.3 months in the pembrolizumab arm vs 7.4 months in the chemotherapy arm. Data from a planned survival analysis to be presented at the 2017 American Society of Clinical Oncology (ASCO) annual meeting confirm these findings; at a median follow-up of 18.5 months, the overall survival remained at 10.3 vs 7.4 months, respectively.2

The objective response rate, a secondary end point, was 21% for pembrolizumab and 11% for chemotherapy (P = .002). No significant difference in progression-free survival between the 2 arms was observed.

Accelerated approval for the first-line indication was based on data from the single-arm, open-label KEYNOTE 052 trial ( Identifier: NCT02335424) in 370 patients treated with pembrolizumab 200 mg every 3 weeks.

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At a median follow-up of 7.8 months, the objective response rate was 28.6% and the median response duration was not reached (range 1.4+, 17.8+ months).

According to the FDA, “an additional trial is required to confirm clinical benefit of pembrolizumab for the first-line indication.”


  1. Pebrolizumab (Keytruda): advanced or metastatic urothelial carcinoma [news release]. Silver Spring, MD: US Food and Drug Administration; May 18, 2017. ApprovedDrugs/ucm559300.htm. Accessed May 18, 2017.
  2. Bajorin DF, De Wit R, Vaughn, DJ, et al. Planned survival analysis from KEYNOTE-045: Phase 3, open-label study of pembrolizumab (pembro) versus paclitaxel, docetaxel, or vinflunine in recurrent, advanced urothelial cancer (UC). J Clin Oncol. 2017;35(suppl; abstract 4501).