FDA approves new drug for advanced bladder cancer

Dec. 30, 2019

The U.S. Food and Drug Administration (FDA) granted Astellas Pharma US Inc. accelerated approval of the drug enfortumab vedotin (EV) to treat adult patients with advanced urothelial or bladder cancer. The approval is the direct result of a multi-institutional clinical trial led by Yale Cancer Center (YCC) and Smilow Cancer Hospital researchers demonstrating EV as an effective treatment for this deadly disease. The FDA granted the approval of EV to Astellas Pharma US Inc.

“This is very exciting progress as we haven’t had another therapy option for patients whose urothelial or bladder cancer has progressed after chemotherapy or immunotherapy. To my knowledge, this is most active single drug in urothelial cancer” said Daniel P. Petrylak, M.D., professor of medicine (medical oncology) and urology and co-director of the Cancer Signaling Research Program at YCC. Petrylak was the senior investigator of a phase II clinical trial that studied the efficacy of the EV for patients. “It’s gratifying to know Yale spearheaded the research effort to get this life-saving therapy to patients.”

Persuasive data from the trial were presented last June at the American Society of Clinical Oncology (ASCO) annual meeting. EV, described by Petrylak as a “smart bomb”, is an antibody-drug conjugate, a type of therapy that combines an antibody that targets a specific protein on the surface of tumor cells with a payload of powerful chemotherapy. The trial showed the drug produced responses in 44% of patients with locally advanced or metastatic urothelial cancer who had been previously treated with chemotherapy and checkpoint inhibitors. Among 125 patients who had received both standard chemotherapy plus a checkpoint inhibitor and EV, 9% had a complete response with no detectable sign of cancer. Median overall survival was 11.7 months. Notably, 36% of people whose cancer had metastasized to the liver responded to the treatment, a site which has been resistant to both standard chemotherapy and immune therapy.

More than 80,000 people are diagnosed with bladder cancer each year in the U.S., with an estimated 17,000 people dying from the disease. There is no routine screening recommended, so most people do not undergo any tests to detect bladder cancer unless they experience symptoms. Signs and symptoms of the disease include blood in the urine, painful or frequent urination, urinary incontinence and pain in the abdominal area. 

“Our work continues as there are still many patients who need other treatment options,” Petrylak added. “We have proceeded with a phase I trial to examine the drug’s benefits for people who are newly diagnosed with advanced urothelial cancer but are ineligible for platinum chemotherapy. Additionally, another phase I trial is looking at treating advanced or metastatic disease by combining EV with the checkpoint inhibitor pembrolizumab.”

Yale Cancer Center has the announcement