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December 02, 2019
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FDA grants priority review to Keytruda for certain patients with high-risk bladder cancer

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The FDA granted priority review to a supplmemental biologics license application that seeks approval of pembrolizumab for treatment of certain patients with high-risk, nonmuscle-invasive bladder cancer.

The approval would apply to use of the agent as monotherapy for patients with Bacillus Calmette-Guerin (BCG)-unresponsive disease with carcinoma in situ, with or without papillary tumors, who are ineligible for or have decided not to undergo cystectomy.

The FDA based the designation on results of the phase 2 KEYNOTE-057 trial, which included 102 patients with this type of bladder cancer.

Patients enrolled in the multicenter, open-label, single-arm trial received the anti-PD-1 therapy pembrolizumab (Keytruda, Merck) dosed at 200 mg every 3 weeks. Treatment continued for up to 24 months or until progressive disease, unacceptable toxicity, or detection of persistent or recurrent high-risk nonmuscle-invasive bladder cancer.

Complete response and duration of response served as major efficacy outcome measures.

The FDA’s Oncologic Drugs Advisory Committee will review this application at its Dec. 17 meeting. The FDA — which often follows the advisory committee’s recommendation but is not required to do so — is expected to make a decision on the application in January.

“Patients with high-risk, nonmuscle-invasive bladder cancer sometimes make an informed decision to decline — or are medically ineligible for — radical cystectomy, and there are currently limited nonsurgical treatment options approved by the FDA for these patients who are BCG-unresponsive,” Roy Baynes, MD, PhD, senior vice president, head of global clinical development and chief medical officer at Merck Research Laboratories, said in a company-issued press release. “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 Keytruda.”

The FDA has approved pembrolizumab for treatment of specific patients with melanoma, Merkel cell carcinoma, lung cancer, hepatocellular carcinoma, head and neck cancer, Hodgkin lymphoma, primary mediastinal large B-cell lymphoma, urothelial carcinoma, gastric cancer, esophageal cancer, cervical cancer, renal cell carcinoma and microsatellite instability-high cancer.