Regeneron Pharmaceuticals, Inc. received approval from the U.S. FDA for the PD-1 inhibitor Libtayo (cemiplimab-rwlc) in combination with platinum-based chemotherapy for the first-line treatment of advanced non-small cell lung cancer (NSCLC) with no EGFR, ALK or ROS1 aberrations. Patients must either have metastatic or locally advanced tumors that are not candidates for surgical resection or definitive chemoradiation. 
 
The approval is based on data from the global Phase 3 trial, EMPOWER-Lung 3, that enrolled 466 patients with locally advanced or metastatic NSCLC, irrespective of PD-L1 expression or tumor histology, and with no ALK, EGFR or ROS1 aberrations. Among those enrolled, 43% had tumors with squamous histology, 67% had tumors with <50% PD-L1 expression, 15% had inoperable locally advanced disease not eligible for definitive chemoradiation, and 7% had pretreated and clinically stable brain metastases.
 
The trial was stopped early based on a recommendation by the Independent Data Monitoring Committee after the Libtayo combination demonstrated a significant improvement in overall survival (OS), the primary endpoint. Efficacy results comparing the Libtayo combination to chemotherapy alone include:
 
22-month median OS versus 13 months, representing a 29% relative reduction in the risk of death. The 12-month probability of survival was 66% for the Libtayo combination versus 56% for chemotherapy.
 
8-month median PFS versus 5 months, representing a 44% reduction in the risk of disease progression. The 12-month probability of PFS for the Libtayo combination was 38% versus 16% for chemotherapy.
 
43% overall response rate versus 23%.
 
The most common adverse reactions occurring in >15% of patients  were alopecia (37% Libtayo combination, 43% placebo), musculoskeletal pain (30% Libtayo combination, 36% placebo), nausea (25% Libtayo combination, 16% placebo), fatigue (23% Libtayo combination, 18% placebo), peripheral neuropathy (23% Libtayo combination, 19% placebo) and decreased appetite (17% Libtayo combination, 12% placebo). Serious adverse reactions occurred in 25% of patients, with treatment discontinuations due to adverse reactions in 5% and fatal adverse reactions in 6%. No new Libtayo safety signals were observed.