Bristol Myers Squibb’s NDA accepted by FDA

Global pharmaceutical company Bristol Myers Squibb (BMS) has announced that its New Drug Application (NDA) has been accepted by the US Food and Drug Administration (FDA) for its ROS1-positive locally advanced or metastatic non-small cell lung cancer (NSCLC) treatment repotrectinib.
Repotrectinib (TPX-0005, BMS-986472) is a next-generation, potentially best-in-class tyrosine kinase inhibitor (TKI), designed to improve the durability of response with favourable properties for human brain penetration to enhance intracranial activity.
The acceptance was based on results from the TRIDENT-1 study, a phase 1/2 open label, multicentre, first-in-human clinical trial. Phase 1 evaluated the safety, tolerability, pharmacokinetics and anti-tumour activity of repotrectinib. Phase 2 had a primary endpoint of overall response rate (ORR) as assessed by Blinded Independent Central Review (BICR) using RECIST v1.1, and secondary endpoints of: duration of response (DOR); time to response (TTR); progression-free survival (PFS); overall survival (OS); and clinical benefit rate (CBR).
Repotrectinib demonstrated high response rates and clinically meaningful durability, with the safety profile being manageable. The study remains ongoing for long-term outcomes and additional endpoints.
Jonathan Cheng MD, senior vice president and head of oncology development at BMS, commented: “Patients with ROS1-positive NSCLC face a rare disease with a significant unmet medical need given the limited durability of benefit and emergence of resistance to approved therapies. The FDA’s acceptance of this application marks an exciting milestone on our journey to bring this next-generation TKI to patients. If approved, this would represent a potential best-in-class option for TKI-naïve patients and a potential first-in-class option for patients with ROS1-positive NSCLC who have been previously treated with TKI, and for whom there are currently no approved targeted therapies available. We are eager to continue working closely with the FDA on the review of this precision medicine, which has shown unprecedented level of durability of responses and robust intracranial responses in patients with ROS1-positive NSCLC.”