FDA Approves Keytruda for High-Risk, Non-Muscle Invasive Bladder Cancer


The Food and Drug Administration approved Keytruda for the treatment of patients with Bacillus Calmette-Guerin–unresponsive, high-risk, non-muscle invasive bladder cancer.

The Food and Drug Administration (FDA) approved Keytruda (pembrolizumab) for the treatment of patients with Bacillus Calmette-Guerin—unresponsive, high-risk, non-muscle invasive bladder cancer (NMIBC) with carcinoma in-situ (CIS) with or without papillary tumors who are ineligible for or chose to not undergo cystectomy.

The agency’s decision follows a favorable vote (9 to 4) from the FDA’s Oncologic Drugs Advisory Committee (ODAC) last month.

The new drug application for Keytruda in this patient population was based on findings from the phase 2 KEYNOTE-057 trial — designed to evaluate the agent in 96 patients, who elicited a 41.2% complete response rate and a median duration of complete response of 16.2 months. Nineteen (48%) of the 40 responding patients maintained their response for one year or more.

The most common side effects noted in the study were fatigue, diarrhea, rash, pruritis, musculoskeletal pain, hematuria, cough, arthralgia, nausea, constipation, urinary tract infection, peripheral edema, hypothyroidism, and nasopharyngitis.

“The drug is safe and it does seem to be effective for these patients. I also have a lot of confidence in the physicians counseling these patients to present this as an option, and to present it in the context of what’s known and not known, and for the patients and the doctors to make good decisions about this treatment,” Dr. Christian S. Hinrichs, from the Experimental Transplantation and Immunology Branch at the National Cancer Institute, said when explaining his “yes” vote during the ODAC meeting.

This population historically is treated with radical cystectomy, which means surgical removal of the bladder. However, BCG has been a treatment option for those with non-invasive bladder cancer for more than 40 years.

The FDA recommended a dose of 200 of Keytruda mg every three weeks.

Check back later on what you need to know about this approval.

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