FDA Grants Priority Review to Repotrectinib for Non-Small Cell Lung Cancer


Repotrectinib is a promising next-generation tyrosine kinase inhibitor under investigation for the treatment of ROS1-positive locally advanced or metastatic non-small cell lung cancer.

The FDA has granted Priority Review to a new drug application (NDA) for repotrectinib (TPX-0005; Turning Point Therapeutics Inc.) for patients with rare ROS1-positive locally advanced or metastatic non-small cell lung cancer (NSCLC), according to a recent Bristol Myers Squibb press release.

Repotrectinib is a next-generation tyrosine kinase inhibitor (TKI) that demonstrated high response rates and clinically meaningful durability of benefit in patients who were treated with or without a TKI (TKI-pretreated and TKI-naïve) or who had ROS1 resistance mutations, in the registrational TRIDENT-1 study.

“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,” said Jonathan Cheng, MD, senior vice president and head of oncology development, Bristol Myers Squibb, in the press release.

Lung cancer is the primary cause of cancer-related death globally, and 84% of cases are NSCLC. Only 1% to 2% of patients with NSCLC have ROS1 fusion (rearrangement)—these patients are ROS1-positive—with most being younger, female, and having little to no smoking history. Repotrectinib is a next-generation, potential best-in-class TKI that targets ROS1- or NTRK-positive locally advanced or metastatic solid tumors, including NSCLC.

TRIDENT-1 is a phase 1/2 open-label, global, multi-center, first-in-human clinical trial that evaluated the safety, tolerability, pharmacokinetics, and anti-tumor activity of repotrectinib in patients with advanced solid tumors. Phase 1 of the trial evaluated safety and pharmacokinetic endpoints.

Phase 2 evaluated the primary endpoint of overall response rate (ORR) and key secondary endpoints of duration of response (DOR), time to response (TTR), progression-free survival (PFS), overall survival (OS) and clinical benefit rate (CBR) in patients treated with or without a TKI and who have ROS1-positive locally advanced or metastatic NSCLC or NTRK-positive advanced solid tumors.

In 2017, the FDA granted repotrectinib orphan drug designation. The FDA also awarded the treatment 3 breakthrough therapy designations for patients with metastatic disease with ROS1 or NTRK gene fusions and who are TKI-naive or TKI-pretreated, and 4 fast-track designations for patients with treated or untreated ROS1-positive advanced NSCLC or NTRK gene fusion. The FDA set a target action date of November 27, 2023.

In August 2022, Bristol-Myers Squibb acquired Turning Point Therapeutics and its pipeline of investigational drugs across precision oncology and advanced solid tumors, including repotrectinib.

Cheng said that “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.”


Bristol Myers Squibb. U.S. Food and Drug Administration Accepts for Priority Review Bristol Myers Squibb’s Application for Repotrectinib for the Treatment of Patients with Locally Advanced or Metastatic ROS1-Positive Non-Small Cell Lung Cancer. News Release. May 30, 2023. Accessed on May 30, 2023. https://news.bms.com/news/details/2023/U.S.-Food-and-Drug-Administration-Accepts-for-Priority-Review-Bristol-Myers-Squibbs-Application-for-Repotrectinib-for-the-Treatment-of-Patients-with-Locally-Advanced-or-Metastatic-ROS1-Positive-Non-Small-Cell-Lung-Cancer/default.aspx

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