FDA Prioritizes Review of Boehringer's Zongertinib NDA for NSCLC

The U.S. Food and Drug Administration (FDA) has decided to prioritize the review of Boehringer Ingelheim’s new drug application for zongertinib, intended to treat individuals with non-small cell lung cancer (NSCLC) that is either unresectable or metastatic. This decision focuses on patients whose tumors display mutations in the human epidermal growth factor receptor 2 (HER2), specifically those who have previously undergone systemic therapy. The FDA has established a Prescription Drug User Fee Act (PDUFA) action date for the third quarter of 2025, indicating the timeline for the decision.

Shashank Deshpande, who is the head of Human Pharma at Boehringer Ingelheim and a member of the board of managing directors, expressed optimism about zongertinib's impact. Deshpande stated that the drug holds the potential to revolutionize treatment for patients with HER2-mutant advanced NSCLC who have limited options after initial treatments. The priority review emphasizes the urgent need for innovative solutions in this area and highlights the potential of zongertinib as a significant advancement in treatment.

The application for zongertinib was supported by the results from the Phase Ib Beamion LUNG-1 clinical trial. One cohort of this trial included 75 participants with mutations in the HER2 tyrosine kinase domain. The findings revealed a 71% objective response rate, with 69% of patients experiencing progression-free survival at six months, and a 73% duration of response rate. The therapy demonstrated a favorable safety profile, with only 5% of participants needing dose reductions and 3% discontinuing treatment due to adverse effects.

Zongertinib is a selective irreversible tyrosine kinase inhibitor that targets HER2 mutations while avoiding the epidermal growth factor receptor, which helps to minimize potential toxicity. The drug is administered orally and is not only being developed for HER2-mutant advanced NSCLC but is also being tested in studies focusing on other solid tumors with HER2 alterations.

In recognition of its potential, the FDA had previously granted zongertinib both breakthrough therapy designation and fast-track status. Additionally, Japan’s Pharmaceuticals and Medical Devices Agency had awarded it orphan drug designation, underscoring the global interest in this innovative therapy.

Beyond the clinical development of zongertinib, Boehringer Ingelheim is continuing its expansion in the oncology sector. In September 2024, the company inaugurated a new €60 million research facility in Vienna, Austria. This move is part of Boehringer Ingelheim’s broader strategy to strengthen its research capabilities and expand its influence in cancer treatment.

Boehringer Ingelheim’s commitment to advancing cancer therapy is evident not only in its development of zongertinib but also in its increased investment in research infrastructure. The anticipated FDA review and potential approval of zongertinib could mark a significant step forward for patients with HER2-mutant NSCLC, offering new hope for those with limited treatment options. The ongoing research and development efforts continue to demonstrate the company’s dedication to delivering innovative solutions for challenging medical conditions.