USA TODAY International Edition
Newly OK’d drug ‘is the future’ in cancer fight
Treatment targets gene mutations in any organ
A cutting-edge cancer treatment focusing on genetic biomarkers rather than any specific type of cancer won accelerated approval from the Food and Drug Administration.
The approval this week for Vitrakvi, the brand name for larotrectinib, marks an emerging method for developing cancer drugs that are “tissue-agnostic” – drugs that are not specific to one organ such as the colon or breast.
Vitrakvi, developed by Bayer and Loxo Oncology, is designed to treat solid tumors from TRK fusion cancer wherever it develops in the body. The particular mutation is rare – and the treatment is not cheap.
Just the test for the cancer costs thousands of dollars, and the price tag for the treatment could reach hundreds of thousands of dollars. It's not clear how much of that a patient would pay, but
Bayer said nobody who needs the drug will do without it.
The drug could be a lifesaver for patients with no alternatives.
“There had been no treatment for cancers that frequently express this mutation,” the FDA said in a statement.
Laura Eckwall, a hospital pharmacy director in Chicago, said the genetic approach to fighting conquering cancer is the future.
“The end game should be patenting a process for developing these drugs, not the drugs themselves,” Eckwall told USA TODAY. “Imagine if you could come into the hospital, figure out where your particular cancer was mutated and have a drug developed for it. That is the future, once one of the drug companies figures it out.”
Michael Weiner, pediatric oncologist and vice chair of the Department of Pediatrics at Columbia University, called the genetic approach the “new horizon” in cancer treatment.
“We're moving away from the era of chemotherapy into immunotherapy, which is driven by the ability to detect and target genetic mutations,” Weiner said. “In fact, in children we're finding that as many as 40 to 50 percent of newly diagnosed cancers have some type of mutation that would benefit from precision medicine.”
The early returns for Vitrakvi are encouraging. Seventy-five percent of the drug's recipients responded, and 73 percent of responses lasted at least six months. Almost 40 percent lasted a year or more, the FDA said.
FDA Commissioner Scott Gottlieb called the approval the latest step in “an important shift toward treating cancers based on their tumor genetics.”
The approval reflects advances in the use of genetic biomarkers to guide drug development aimed at more closely targeting the delivery of medicine, he said. He said the drug's development would not have been possible a decade
“The end game should be patenting a process for developing these drugs, not the drugs themselves.” Laura Eckwall, hospital pharmacy director in Chicago
ago.
Vitrakvi received an accelerated approval, which enables the FDA to approve drugs for serious conditions to fill an unmet medical need using clinical trial data that is thought to predict a clinical benefit to patients. Further clinical trials are in the works, the FDA says.
The FDA had granted the drug a Priority Review and Breakthrough Therapy designation. Vitrakvi also received Orphan Drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases.
“We now have the ability to make sure that the right patients get the right treatment at the right time,” Gottlieb said.