Cancer

[mr40]

FDA approves new drug reflecting cutting-edge, 'tissue-agnostic' effort to beat cancer

A cutting-edge cancer treatment focusing on genetic biomarkers rather than any specific type of cancer has won accelerated approval from the Food and Drug Administration.

The approval this week for Vitrakvi, the brand name for larotrectinib, marks a new method for developing cancer drugs that are “tissue agnostic” – cancer that is not specific to one organ such as colon or breast cancer.

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, 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 says nobody who needs the drug will do without it.

“There had been no treatment for cancers that frequently express this mutation, like mammary analogue secretory carcinoma, cellular or mixed congenital mesoblastic nephroma and infantile fibrosarcoma,” the FDA said in a statement.

More: Her cancer persisted through high school. A new therapy offers her hope

More: High price of precision medicine forces cancer patients to make agonizing choices

The treatment specifically goes after mutations in DNA, and the numbers 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 added that the drug's development would not have been possible a decade ago.

Vitrakvi's accelerated approval enables the FDA to approve drugs for serious conditions to fill an unmet medical need using clinical trial data that appears 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.

https://www.usatoday.com/story/news/nation/2018/11/28/new-cancer-drug-vitrakvi-larotrectinib-targets-genetic-links/2135582002/