FDA Approval Summary: Nivolumab in Combination with Ipilimumab for the Treatment of Unresectable Malignant Pleural Mesothelioma.

On October 2, 2020, the U.S. Food and Drug Administration (FDA) approved nivolumab with ipilimumab as first-line treatment for adult patients with unresectable malignant pleural mesothelioma (MPM). The approval was based on results from Study CA209743 (CHECKMATE-743), an open-label trial of patients with MPM randomized to receive nivolumab and ipilimumab for up to two years (n=303) or six cycles of chemotherapy with cisplatin or carboplatin plus pemetrexed (n=302). Overall survival (OS) was improved for patients who received nivolumab and ipilimumab, with a median OS of 18.1 months (95% CI: 16.8, 21.5) compared to 14.1 months (95% CI: 12.5, 16.2) (HR 0.74; 95% CI: 0.61, 0.89; p = 0.002), for patients who received chemotherapy. The magnitude of benefit was larger for patients with non-epithelioid versus epithelioid histology. Additional clinical pharmacology data supports an alternative dosing regimen of nivolumab than evaluated in the trial, which will reduce the number of required treatment visits. This application was reviewed under FDA9s Project Orbis, in collaboration with Australia9s Therapeutic Goods Administration, Switzerland9s Swissmedic, Health Canada, and Brazil9s National Health Surveillance Agency or ANVISA (Agencia Nacional de Vigilância Sanitaria). Nivolumab and ipilimumab is the first drug regimen approved by the FDA for MPM since 2004.