FDA Approves GSK’s Jideytro for ROS1-Positive Lung Cancer

GSK has received U.S. Food and Drug Administration (FDA) approval for Jideytro (zidesamtinib), a next-generation targeted therapy for adults with locally advanced or metastatic ROS1-positive non-small cell lung cancer (NSCLC) whose disease has progressed after treatment with a previous ROS1 kinase inhibitor.

The approval was granted ahead of the FDA’s original decision deadline of September 18, 2026, following the therapy’s earlier Breakthrough Therapy and Orphan Drug designations. The decision marks an important milestone for GSK, as Jideytro becomes the company’s first approved medicine for lung cancer.

Jideytro entered GSK’s oncology portfolio through its recently completed acquisition of Nuvalent Inc. The drug was specifically designed to address some of the biggest treatment challenges faced by patients with ROS1-positive NSCLC, including resistance to earlier therapies, progression of disease in the brain, and treatment tolerability.

ROS1-positive NSCLC is a rare subtype of lung cancer that affects an estimated 50,000 people worldwide each year. It is more commonly diagnosed in younger adults, often non-smokers in their 40s and 50s, many of whom require long-term treatment because of advances in targeted therapies.

Unlike earlier treatments, Jideytro was engineered with high selectivity for the ROS1 target while maintaining activity against multiple resistance mutations that frequently develop after initial therapy. The medicine was also designed to cross the blood-brain barrier, enabling it to treat cancer that has spread to the brain—a common challenge in patients with ROS1-positive disease.

The FDA approval is supported by results from the Phase I/II ARROS-1 clinical trial, a global single-arm study involving patients with advanced or metastatic ROS1-positive NSCLC who had previously received a ROS1 inhibitor.

Among 117 evaluable patients, Jideytro achieved an objective response rate (ORR) of 44%, meaning nearly half of treated patients experienced measurable tumor shrinkage. The responses also proved durable, with 82% of responders maintaining their response for at least six months and 69% remaining in response after one year.

Importantly, clinical activity was observed in patients with brain metastases as well as those whose cancers had developed ROS1 resistance mutations, two groups that often have limited treatment options.

The safety profile was evaluated in a pooled population of 446 patients. The most commonly reported side effects included swelling (oedema), peripheral neuropathy, constipation, fatigue, and shortness of breath.

Tony Wood, Chief Scientific Officer at GSK, said the rapid approval demonstrates both the strength of the science behind the medicine and the urgent need for new treatment options.

“The rapid progression of Jideytro from development to approval reflects the strength of the underlying science and the need for additional effective and well-tolerated treatments for patients with ROS1-positive lung cancer,” Wood said. “This approval provides an important new option for patients and supports our strategy of acquiring innovative medicines that have the potential to improve standards of care.”

Alexander Drilon, MD, Chief of the Early Drug Development Service at Memorial Sloan Kettering Cancer Center and an investigator in the ARROS-1 study, said treatment resistance and brain metastases continue to pose major clinical challenges.

“Despite advances in treatment, resistance mutations, disease progression in the brain, and treatment-related adverse events remain significant issues,” Drilon said. “The responses observed in ARROS-1, including among heavily pre-treated patients, represent meaningful progress.”

Patient advocacy groups also welcomed the approval.

Janet Freeman-Daily, Co-Founder and President of The ROS1ders, said patients seek treatments that allow them to maintain both survival and quality of life.

“The goal is not just more time, but quality time,” she said. “This approval gives patients and physicians an important new treatment option and represents meaningful progress for the ROS1-positive community.”

The approval also strengthens GSK’s expanding precision oncology portfolio following its acquisition of Nuvalent. In addition to Jideytro, the company is advancing neladalkib (NVL-655) for ALK-altered NSCLC, which is currently under FDA review, and NVL-330, an investigational therapy targeting HER2-altered NSCLC. GSK is also developing risvutatug rezetecan (Ris-Rez), a B7-H3-targeted antibody-drug conjugate that recently reported positive Phase III results in relapsed small-cell lung cancer.

With Jideytro now approved, GSK has entered the lung cancer treatment market with a targeted therapy designed to address resistance, brain metastases, and long-term disease management for patients with ROS1-positive NSCLC.