The U.S. Food and Drug Administration (FDA) has granted accelerated approval to HERNEXEOS (zongertinib tablets), Boehringer Ingelheim’s new oral targeted therapy for adults with previously treated, HER2-mutant advanced non-small cell lung cancer (NSCLC).
This marks the first FDA-approved oral treatment specifically for patients with unresectable or metastatic non-squamous NSCLC harboring HER2 (ERBB2) activating mutations, identified via an FDA-approved test. The approval is based on objective response rate and duration of response, with continued approval contingent on confirmatory clinical trials.
“Zongertinib offers a meaningful new option for patients with HER2-mutant NSCLC—an underserved group with limited treatment choices,” said Dr. John Heymach of MD Anderson Cancer Center, a lead investigator in the Beamion-LUNG 1 trial.
The FDA’s decision is supported by data from the Phase Ib Beamion-LUNG 1 study, which showed a 75% overall response rate, including 6% complete and 69% partial responses among 71 patients. Additionally, 58% of those responding maintained their response for six months or longer. These results were presented earlier this year at the AACR Annual Meeting and published in The New England Journal of Medicine.
Zongertinib was well tolerated in clinical trials, with a low discontinuation rate of 2.9%. The most common side effects included diarrhea (53%), liver toxicity (27%), rash (27%), fatigue (22%), and nausea (21%).
“Bringing HERNEXEOS to patients within just four years of starting clinical development is a testament to our commitment to advancing precision oncology,” said Shashank Deshpande, Chairman of Boehringer Ingelheim’s Board of Managing Directors. “This approval could redefine the treatment landscape for patients facing HER2-mutant NSCLC.”
HER2 mutations, found in approximately 2–4% of NSCLC cases, are linked to poor prognosis and a higher risk of brain metastases. The availability of HERNEXEOS underscores the growing importance of comprehensive biomarker testing, including next-generation sequencing, in guiding treatment decisions.
Patient advocacy groups welcomed the news. “The approval of zongertinib is a milestone for patients with HER2-mutant lung cancer who have long awaited new, personalized treatment options,” said Marcia Horn, President of the International Cancer Advocacy Network.
HERNEXEOS is now available in the U.S. as an oral tablet for eligible patients who have previously received systemic therapy for advanced HER2-mutant NSCLC.