Targeted Therapy Approved for Younger Pediatric Patients 

The FDA has approved the inhibitor entrectinib for some patients 1 month of age or older with solid tumors harboring a certain genetic mutation. 

The U.S. Food and Drug Administration (FDA) has granted accelerated approval to entrectinib (Rozlytrek) for the treatment of certain pediatric patients older than 1 month who have solid tumors that are metastatic or cannot be safely removed, harbor a gene fusion involving the NTRK gene, and have progressed on prior treatment or have no other satisfactory treatment options. The approval also included a new formulation of the drug that can be delivered as an oral pellet or suspension rather than in capsule form. 

A pediatric cancer patient with a NTRK-mutated solid tumor high-fiving their doctor.

Entrectinib is a targeted therapy that blocks the activity of the kinase NTRK. Part of the NTRK gene can sometimes fuse with part of another gene, which can increase the expression or activity of NTRK to drive cancer growth. In this approval, entrectinib is intended to treat tumors with NTRK fusions but without other NTRK mutations that may make the tumor resistant to NTRK blockade. 

The FDA previously granted accelerated approval to entrectinib for the same indication in adults and children aged 12 and older. The current approval allows patients as young as 1 month to receive treatment. 

The approval was based on results from two multicenter, single-arm clinical trials: the phase I/II STARTRK-NG trial and the phase II TAPISTRY trial. Between the two studies, 33 patients with NTRK fusion-positive tumors received entrectinib, with an overall response rate of 70% and a median duration of response of 25.4 months. 

Entrectinib is one of only six targeted therapy regimens approved to treat tumors based on their molecular characteristics rather than their site of origin. While NTRK fusions occur in less than 1% of all solid tumors, they are more common in rare tumor types that may not have as many treatment options. 

The FDA rendered its decision on October 20, 2023. Accelerated approval means that continued approval may be contingent upon a confirmatory trial.