New Targeted Therapy Approved for ROS1-positive Lung Cancer
Zidesamtinib, a next-generation ROS1 inhibitor, was approved for ROS1-positive NSCLC previously treated with ROS1 inhibition.
The U.S. Food and Drug Administration (FDA) has approved zidesamtinib (Jideytro), a next-generation ROS1 inhibitor, for adults with locally advanced or metastatic non-small cell lung cancer (NSCLC) harboring a ROS1 gene fusion whose disease has been previously treated with at least one other ROS1-targeted therapeutic.
Zidesamtinib selectively targets ROS1, a tyrosine kinase receptor that can become abnormally activated through gene fusions and drive cancer growth. By inhibiting ROS1 signaling, it helps prevent ROS1-positive tumors (i.e., tumors harboring a ROS1 gene fusion) from growing and spreading.
A preclinical study published in Molecular Cancer Therapeutics, an American Association of Cancer Research (AACR) journal, showed that the drug was active against a broad range of ROS1 resistance mutations and was able to penetrate the brain—two challenges that have limited the effectiveness of previously approved ROS1-targeted inhibitors. Researchers also showed that zidesamtinib minimized off-target inhibition, a common source of treatment-related side effects.
The approval of zidesamtinib was based on findings from ARROS-1, a multicenter, single-arm, open-label phase I/II study of patients with advanced or metastatic ROS1-positive NSCLC previously treated with ROS1 inhibitors. Among the 117 enrolled patients, 59 had received one prior ROS1 tyrosine kinase inhibitor (TKI) and 58 had received two or more ROS1 TKIs, including lorlatinib (Lorbrena), repotrectinib (Augtyro), and/or taletrectinib (Ibtrozi).
Overall, 44% of patients had responses to zidesamtinib. Among those with responses, 82% had responses that lasted for at least six months and 69% for at least 12 months.
Data from the clinical trial were presented at the AACR Annual Meeting 2026.
Zidesamtinib is recommended to be taken at 100 mg orally once daily, with or without food, until disease progression or unacceptable toxicity.
Lung cancer is the third most common cancer in the United States, with an estimated 229,410 new cases and 124,990 deaths expected in 2026. NSCLC represents approximately 80% of all lung cancers, and ROS1 rearrangements occur in 1% to 2% of patients with the disease.
The FDA rendered its decision on July 22, 2026. Check this resource for updated information on all therapeutics regulated by the FDA.