FDA Approves Use of Nivolumab in Metastatic Nonsquamous Lung Cancer

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The U.S. Food and Drug Administration (FDA) has approved nivolumab (Opdivo) to treat patients with metastatic nonsquamous non–small cell lung cancer (NSCLC) whose disease progressed during or after platinum-based chemotherapy. Nivolumab is a monoclonal antibody that that blocks the PD-1/PD-L1 [programmed cell death protein 1 and its ligand] cellular pathway.

The FDA approved nivolumab earlier this year to treat patients with advanced squamous NSCLC whose disease progressed during or after platinum-based chemotherapy.

Clinical Trial Results

The safety and effectiveness of nivolumab for this indication were demonstrated in an international, open-label, randomized study of 582 participants with advanced NSCLC whose disease progressed during or after treatment with platinum-based chemotherapy and appropriate biologic therapy.1 Participants were treated with nivolumab or docetaxel.

The primary endpoint was overall survival, and the secondary endpoint was objective response rate. Those treated with nivolumab lived an average of 12.2 months compared with 9.4 months in those treated with docetaxel. Additionally, 19% of those treated with nivolumab experienced complete or partial shrinkage of their tumors, an effect that lasted an average of 17 months, compared with 12% among those taking docetaxel, which lasted an average of 6 months.

While patients who received ni­volumab lived longer than those who received docetaxel across the study, an evaluation of samples from a subgroup of patients’ tumors suggested that the level of PD-L1 expression in NSCLC tumors may help identify patients who are more likely to live longer due to treatment with nivolumab. Therefore, the FDA also approved the PD-L1 IHC 28-8 pharmDx test to detect PD-L1 protein expression levels and help physicians determine which patients may benefit most from nivolumab treatment.

Safety Profile

The most common side effects of nivolumab are fatigue, musculoskeletal pain, decreased appetite, cough and constipation. Nivolumab also has the potential to cause severe immune-mediated side effects.

The FDA granted nivolumab Breakthrough Therapy designation for this indication based on preliminary clinical evidence that suggested nivolumab may offer a substantial improvement over available therapies. It also received Priority Review status.

The approval of nivolumab occurred approximately 3 months ahead of the prescription drug user fee goal date of January 2, 2016, the date when the agency was scheduled to complete its review of the application.  ■


1. Borghaei H, Paz-Ares L, Horn L, et al: Nivolumab versus docetaxel in advanced nonsquamous non–small-cell lung cancer. N Engl J Med. September 27, 2015 (early release online).