Milestone Precision Medicine Decision: FDA Approves the First Treatment with an Initial Tumor-Agnostic Indication


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The US Food and Drug Administration (FDA) has approved the drug larotrectinib for cancers caused by a genetic mutation called a TRK fusion. Today’s decision marks a transformation in the field of precision medicine as this is the first time that an entirely new treatment has received a tumor-agnostic indication at its initial approval, meaning that the drug was approved based on mutation type rather than on where in the body the tumor originated.

The landmark data that led to this approval support the foundation of precision medicine by creating a treatment option for a genetically defined cancer while continuing to validate the concept that comprehensive molecular profiling should be strongly considered in people of all ages with advanced solid tumors. This approval also opens the door to a unique and innovative approach to treating both adults and children with cancer. People between the ages of one month and 80 years have been treated with this drug through clinical trials. David Hyman, Chief of the Early Drug Development Service at Memorial Sloan Kettering Cancer Center (MSK), is the global principal investigator of the larotrectinib NAVIGATE trial.

Precision Medicine at MSK

MSK has paved the way in the field of precision medicine, beginning with the landmark publication of the first basket trial in the New England Journal of Medicine in August 2015. Data from that trial led to the first FDA approval of a targeted therapy based on a basket trial in November 2017. (“Basket trials” refer to tumor-agnostic clinical trials that explore responses to drugs based on the specific mutations in tumors rather than where the cancer originated.)

Throughout this journey from bench to bedside, the MSK team has continued to pioneer tumor-agnostic treatment approaches and next-generation sequencing (through MSK-IMPACT™), publishing a seminal review of the rapidly evolving field of precision oncology in Cell in February 2017. Additionally, Dr. Hyman presented larotrectinib data as part of the press program for the American Society of Clinical Oncology’s annual meeting in 2017, and he published updated data in the New England Journal of Medicine in February 2018.

Investigator Comments

“Based on today’s approval, TRK fusion-positive cancers may be the first type of cancer that is more appropriate to define based on a shared genetic mutation than by the organ in which it arises. This distinction marks a new chapter for precision medicine,” explained Dr. Hyman. “While the concept of targeted therapy seemed wildly futuristic just two decades ago, we’ve seen firsthand — through a combination of advances in basic science, clinical practice, and genetic sequencing and an evolving design of clinical trials — how this treatment approach can benefit select individuals.”

David Hyman has received grants from Loxo Oncology.