NEW HAVEN — A new drug to treat bladder and urinary tract cancers, developed by Yale Cancer Center and Smilow Cancer Hospital researchers, has been approved by the U.S. Food & Drug Administration.
Enfortumab vedotin, described as a “smart bomb” by the senior investigator in its clinical trial, was granted accelerated approval for treating adult patients with advanced urothelial or bladder cancer, after a clinical trial at multiple institutions, according to a press release. The approval was granted to Astellas Pharma US Inc., the American subsidiary of a Japanese pharmaceutical company.
“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,” said Dr. Daniel P. Petrylak, professor of medical oncology and urology and co-director of the Cancer Signaling Research Program at the cancer center. Petrylak was the senior investigator in the trial.
“It’s gratifying to know Yale spearheaded the research effort to get this life-saving therapy to patients,” he said.
Data from the trial were presented in June at the American Society of Clinical Oncology’s annual meeting in Chicago. EV combines an antibody that targets a specific protein on the surface of tumor cells with chemotherapy. In the trial, 44 percent of patients with locally advanced or metastatic urothelial cancer who had been previously treated with chemotherapy and checkpoint inhibitors showed a positive response, the press release said.
Among 125 patients who had received both standard chemotherapy plus a checkpoint inhibitor and EV, 9 percent showed no detectable cancer after treatment, with half of patients surviving 11.7 months or more.
One gratifying finding, according to the release, was that 36 percent of patients whose cancer had metastasized to the liver, which has been resistant to both chemotherapy and immune therapy, responded to the treatment.
More than 80,000 Americans are diagnosed with bladder cancer annually, and about 17,000 die from it each year. Since there is no routine screening for the disease, most people do not get tested until they experience symptoms, the press release said. Signs and symptoms 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 said. “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.”