Last week, the Food and Drug Administration (FDA) for the first time approved a cancer drug, larotrectinib (Vitrakvi), based on a genetic mutation rather than type of cancer. The FDA’s approval drew on the findings of three clinical trials comprised of 55 patients, the results of which were compiled and reported in the New England Journal of Medicine by MSK’s Dr. David Hyman, Dr. Alexander Drilon, and collaborators.
The NTRK gene fusion mutation can only be identified through broad genetic tumor testing. While the mutation occurs in less than one percent of solid tumors, it has been identified in dozens of often rare tumor types in adults and children. In the three trials, patients given the drug had a 75% overall response rate.