FDA Sets Goal Date for Augtyro for Solid Tumors with NTRK Gene Fusions

The FDA has accepted for priority review Bristol Myers Squibb’s supplemental new drug application for Augtyro (repotrectinib) and assigned a Prescription Drug User Fee Act (PDUFA) goal date of June 15, 2024. Augtyro is being reviewed as a treatment for adult and pediatric patients 12 years of age and older with solid tumors that have a neurotrophic tyrosine receptor kinase (NTRK) gene fusion. If approved, it would be indicated for patients whose cancer is locally advanced or metastatic.

NTRK gene fusions can play a role in the development of cancer. They are rare in patients with solid tumors with less than 1% of patients testing positive, though may be more frequent in patients with other cancers.

Augtyro is an oral tyrosine kinase inhibitor (TKI) that is designed to target ROS1-positive or NTRK-positive locally advanced or metastatic solid tumors, including non-small cell lung cancer. It was developed by Turning Point Therapeutic, which Bristol Myers Squibb acquired in 2022.

Related: New Lung Cancer Drug Augtyro is Now Available

In November 2023, the FDA approved Augtyro to treat adult patients with locally advanced or metastatic ROS1-positive non-small cell lung cancer NSCLC. The wholesale acquisition cost (WAC) for an adult is $29,000 based on 30-day supply of 160 mg twice a day.

The supplemental filing is based on data from the TRIDENT-1 and CARE trials. In the TRIDENT-1 phase 1/2 study, Augtyro was studied in patients with advanced solid tumors, including non-small cell lung cancer and included patients with ROS1 or NTRK fusions.

Augtyro demonstrated clinically meaningful response rates in patients with NTRK-positive locally advanced or metastatic solid tumors. Durability of response was robust, including among patients whose tumors harbor common resistance mutations, and intracranial responses were observed. Augtyro showed a safety profile that was well tolerated and generally manageable.

The CARE phase 1/2 open-label study evaluated Augtyro in pediatric and young adult patients with locally advanced or metastatic solid tumors harboring ALK, ROS1 or NTRK1-3 gene alterations.

Augtyro is also under review in the Europe Union for patients with locally advanced or metastatic ROS1-positive non-small cell lung cancer and patients with NTRK-positive solid tumors.