Repotrectinib
Repotrectinib, sold under the brand name Augtyro, is an anti-cancer medication used for the treatment of non-small cell lung cancer. It is taken by mouth. Repotrectinib is an inhibitor of proto-oncogene tyrosine-protein kinase ROS1 and of the tropomyosin receptor tyrosine kinases TRKA, TRKB, and TRKC.
The most common adverse reactions include dizziness, dysgeusia, peripheral neuropathy, constipation, dyspnea, ataxia, fatigue, cognitive disorders, and muscular weakness.
Repotrectinib was approved for medical use in the United States in November 2023, and in the European Union in January 2025.
Medical uses
Repotrectinib is indicated for the treatment of adults with locally advanced or metastatic ROS1-positive non-small cell lung cancer.In June 2024, the US Food and Drug Administration expanded the indication to include the treatment of people twelve years of age and older with solid tumors that have a neurotrophic tyrosine receptor kinase gene fusion, are locally advanced or metastatic or where surgical resection is likely to result in severe morbidity, and that have progressed following treatment or have no satisfactory alternative therapy.
Side effects
The most common adverse reactions include dizziness, dysgeusia, peripheral neuropathy, constipation, dyspnea, fatigue, ataxia, cognitive impairment, muscular weakness, and nausea.History
Approval by the US Food and Drug Administration was based on TRIDENT-1, a global, multicenter, single-arm, open-label, multi-cohort clinical trial which included participants with ROS1-positive locally advanced or metastatic non-small cell lung cancer. Efficacy was evaluated in 71 ROS1 tyrosine kinase inhibitor-naïve participants who received up to one prior line of platinum-based chemotherapy and/or immunotherapy and 56 participants who received one prior ROS1 tyrosine kinase inhibitor with no prior platinum-based chemotherapy or immunotherapy.The FDA granted the application for repotrectinib priority review, breakthrough therapy, and fast track designations.