FDA Approves Nivolumab for Advanced Non-Squamous NSCLC

Nivolumab is now approved for treating squamous and non-squamous NSCLC

The US Food and Drug Administration (FDA) expanded the approval of the anti-PD-1 immunotherapy agent nivolumab (Opdivo) to include advanced non-squamous non–small-cell lung cancer (NSCLC). It was approved in March 2015 for the treatment of squamous NSCLC.

Nivolumab blocks the activation of PD-1, which in turn produces cell-mediated immune responses against tumor cells. It is also approved for the treatment of melanoma.

The new approval is based on results from the CheckMate 057 trial, which included 582 individuals with advanced NSCLC whose disease progressed during or following treatment with platinum-based chemotherapy. In that study, patients were randomized to either nivolumab or docetaxel; the trial was halted early, in April of this year, because of superiority of the study drug at an interim analysis.

Results of the study were published online ahead of print in the New England Journal of Medicine. The nivolumab patients had an overall survival of 12.2 months, compared with 9.4 months for the docetaxel patients. After 18 months, 39% of nivolumab patients remained alive, compared with 23% for docetaxel. The hazard ratio for death with nivolumab was 0.73 (96% confidence interval [CI], 0.59–0.89; P = .002).

The response rate also favored nivolumab, at 19% compared with 12% with docetaxel (P = .02). The progression-free survival was no different between the groups, at 2.3 months for nivolumab and 4.2 months for docetaxel, but at 1 year the PFS rates were 19% with nivolumab and 8% with docetaxel.

Grade 3 or 4 treatment-related adverse events occurred in 10% of patients in the nivolumab group, compared with 54% of docetaxel patients.

The FDA also approved a test to measure PD-L1 expression levels, which the agency says could help identify patients best suited for nivolumab therapy.

“There is still a lot to learn about the PD-1/PD-L1 pathway and its effects in lung cancer, as well as other tumor types,” said Richard Pazdur, MD, of the FDA, in a press release. “While Opdivo showed an overall survival benefit in certain NSCLC patients, it appears that higher expression of PD-L1 in a patient’s tumor predicts those most likely to benefit.”