FDA Grants Priority Review to Keytruda for Patients with High-Risk, Non–Muscle-Invasive Bladder Cancer

Yvette Florio Lane

Bladder Cancer, FDA Approvals, News & Updates, Immunotherapy


On December 2, 2019, Merck announced that the FDA has granted priority review for its supplemental biologics license application for the anti–PD-1 agent pembrolizumab (Keytruda). If approved, the drug would be indicated as monotherapy to treat patients with Bacillus Calmette-Guerin–unresponsive, high-risk, non–muscle-invasive bladder cancer who are ineligible for or have decided not to undergo cystectomy.

The filing was based on data from the multicenter, open-label, single-arm, phase 2 KEYNOTE-057 clinical trial of 102 patients (median age, 73 years) with Bacillus Calmette-Guerin–unresponsive, high-risk, non–muscle-invasive bladder cancer with carcinoma in situ with or without papillary tumors who were ineligible for or had decided not to undergo cystectomy.

Major efficacy outcome measures were complete response and duration of response. Patients received 200 mg of pembrolizumab every 3 weeks for up to 24 months or until unacceptable toxicity, progressive disease, or detection of persistent or recurrent high-risk non–muscle-invasive bladder cancer.

It is estimated that >80,000 new cases of bladder cancer will be diagnosed in 2019 in the United States. Approximately 75% of these patients are diagnosed with non–muscle-invasive bladder cancer, which means that the cancer cells have not grown into the main muscle layer of the bladder. Patients with high-grade non–muscle-invasive bladder cancer are at high risk for developing muscle-invasive and metastatic disease.

Keytruda was previously approved as a single agent or in combination with other agents for several other types of cancer, including 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, and renal-cell carcinoma.

In a press release, Merck stated that its application will be reviewed by the FDA’s Oncologic Drugs Advisory Committee on December 17, 2019, and that it anticipates a Prescription Drug User Fee Act, or target action date, in January 2020.