BMS's Augtyro sNDA Accepted by US FDA for NTRK+ Tumors

Bristol Myers Squibb announced that the US Food and Drug Administration (FDA) had accepted the supplemental New Drug Application (sNDA) for Augtyro (repotrectinib) for the treatment of adult and pediatric patients 12 years of age and older with solid tumors that have a neurotrophic tyrosine receptor kinase (NTRK) gene fusion and are locally advanced or metastatic or where surgical resection is likely to result in severe morbidity.

The filing acceptance is based on results from the registrational phase 1/2 TRIDENT-1 trial (adult patients with NTRK-positive solid tumors) and CARE study (pediatric patients with NTRK-positive solid tumors). The FDA granted the application Priority Review status and assigned a Prescription Drug User Fee Act (PDUFA) goal date of June 15, 2024.

“While great advancements have been made over the last decade, patients with NTRK-positive locally advanced or metastatic solid tumors still experience significant unmet needs. New and effective treatment options that may improve the durability of response and address resistance to existing tyrosine kinase inhibitors are critical to helping patients with these aggressive tumors,” said Joseph Fiore, vice president, global program lead, Augtyro, Bristol Myers Squibb. "We look forward to working with the FDA to review Augtyro's application for non-oncology indications and provide new and sustainable treatment options for patients with NTRK-positive disease."

This work is based on the TRIDENT-1 and CARE trial results. In the TRIDENT-1 study, Augtyro demonstrated significant clinical response rates in patients with locally advanced or metastatic solid tumors. The duration of the response was robust, and intracranial responses were observed, even in patients with normal obstructive changes. Augtyro is well tolerated and shows a manageable safety profile. Studies are ongoing to evaluate long-term outcomes and other effects. The TRIDENT-1 results were supported by data from the CARE study, which evaluated Augtyro in pediatric and adolescent patients with locally advanced solid tumors and metastatic solid tumors with ALK, ROS1, or NTRK1-3 gene mutations. In addition, in November 2023, the US Food and Drug Administration (FDA) approved Augtyro for treating adult patients with ROS1-positive and metastatic ROS1-positive non-small cell lung cancer (NSCLC).