The treatment was granted priority review by the FDA, subsequently followed by an approval under the accelerated approval pathway based on overall response rate and duration of response. However, continued approval for this indication may be contingent upon verification of clinical benefit in confirmatory trials.
The approval was based on data from the ongoing phase 1/2 Checkmate-032 study that is investigating nivolumab in patients who experienced disease progression following platinum-based chemotherapy. The trial enrolled 109 patients into the nivolumab arm and found that 12% (n = 13; 95% CI, 6.5-19.5) responded to treatment based on assessment by a Blinded Independent Central Review, regardless of PD-L1 expression. Of the 13 patients who responded, 12 experienced a partial response and 1 patient had a complete response.
The median duration of response was 17.9 months (95% CI, 7.9-42.1; range, 3-42.1 months). Nivolumab was discontinued in 10% of patients, and serious adverse events occurred in 45% of participants. The most frequent adverse events reported in at least 2% of patients were pneumonia, dyspnea, pneumonitis, pleural effusion, and dehydration.
“While immuno-oncology innovations have dramatically changed how oncologists approach certain cancers, we have had limited progress for patients with small cell lung cancer,” said Leora Horn, MD, MSc, director of the thoracic oncology program at Vanderbilt University Medical Center, in a statement
. “Today’s approval of nivolumab is particularly exciting considering it is the first checkpoint inhibitor approved for these specific patients, and now we can finally treat this devastating disease from a different angle.”