Bristol Myers Squibb (NYSE: BMY) announced that the U.S. Food and Drug Administration (FDA) has 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 harboring a neurotrophic tyrosine receptor kinase (NTRK) gene fusion. This includes cases where the tumors are locally advanced or metastatic, or where surgical resection poses significant risks. The FDA has granted Priority Review status to the application, with a target action date set for 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 durability of response and address resistance to existing tyrosine kinase inhibitors are critical to helping patients with these aggressive tumors," stated Joseph Fiore, Vice President and Global Program Lead for Augtyro at Bristol Myers Squibb. "We look forward to working closely with the FDA on the review of our application for Augtyro for this tumor-agnostic indication and potentially offering patients with NTRK-positive disease a new, durable treatment option."

The filing acceptance was based on data from the registrational Phase 1/2 TRIDENT-1 trial for adult patients and the CARE study for pediatric patients, both evaluating Augtyro in NTRK-positive solid tumors. In the TRIDENT-1 study, Augtyro demonstrated clinically meaningful response rates, with robust durability of response even in patients with tumors harboring common resistance mutations. Notably, intracranial responses were also observed. Augtyro exhibited a well-tolerated and generally manageable safety profile. The ongoing TRIDENT-1 study continues to assess long-term outcomes and additional endpoints. The results from TRIDENT-1 were corroborated by findings from the CARE study, which included evaluation of Augtyro in pediatric and young adult patients with locally advanced or metastatic solid tumors harboring ALK, ROS1, or NTRK1-3 gene alterations.

Bristol Myers Squibb expresses gratitude to the patients and investigators involved in the TRIDENT-1 and CARE clinical trials, acknowledging their crucial contributions to advancing treatment options for these challenging cancers.

As a subsidiary of Bristol Myers Squibb since August 2022, Turning Point Therapeutics is responsible for the development of repotrectinib, marketed as Augtyro. In November 2023, Augtyro received FDA approval for the treatment of adult patients with locally advanced or metastatic ROS1-positive non-small cell lung cancer (NSCLC).