On July 22, 2026, the Food and Drug Administration approved zidesamtinib for adults with locally advanced or metastatic ROS1-positive non-small cell lung cancer (NSCLC) who received at least one prior ROS1 tyrosine kinase inhibitor (TKI).

Full prescribing information for zidesamtinib will be posted on Drugs@FDA.

Efficacy was evaluated in ARROS-1 (NCT05118789), a multicenter, single-arm, open-label, multi-cohort trial in patients with previously treated locally advanced or metastatic ROS1-positive NSCLC. The efficacy population included 117 patients; 59 who received one prior ROS1 TKI and 58 who received two or more prior ROS1 TKIs, including lorlatinib, repotrectinib, and/or taletrectinib.

The major efficacy outcome measures were confirmed overall response rate (ORR) and duration of response (DOR) according to RECIST v1.1 as assessed by blinded independent central review (BICR). For the overall efficacy population, ORR was 44% (95% CI: 34, 53) with 82% of responders having a DOR ≥ 6 months and 69% of responders having a DOR ≥ 12 months. In patients who received one prior ROS1 TKI, ORR was 49% (95% CI: 36, 63) and in patients who received two or more prior ROS1 TKIs (n=58), ORR was 38% (95% CI: 26, 52).

The prescribing information includes warnings and precautions for central nervous system adverse reactions, QTc interval prolongation, interstitial lung disease/pneumonitis, skeletal fractures, myalgia with creatine phosphokinase elevation, pancreatic toxicity, and embryo-fetal toxicity.

The recommended zidesamtinib dose is 100 mg taken orally once daily, with or without food, until disease progression or unacceptable toxicity.

This review used the Real-Time Oncology Review (RTOR) pilot program, which streamlined data submission prior to the filing of the entire clinical application, and the Assessment Aid, a voluntary submission from the applicant to facilitate the FDA’s assessment. The FDA approved this application two months ahead of the FDA goal date.

Zidesamtinib received orphan drug designation. A description of FDA expedited programs is in the Guidance for Industry: Expedited Programs for Serious Conditions-Drugs and Biologics.

Healthcare professionals should report all serious adverse events suspected to be associated with the use of any medicine and device to FDA’s MedWatch Reporting System or by calling 1-800-FDA-1088.

For assistance with single-patient INDs for investigational oncology products, healthcare professionals may contact OCE’s Project Facilitate at 240-402-0004 or email OncProjectFacilitate@fda.hhs.gov.

Source: FDA