On 22 February 2021, the US Food and Drug Administration (FDA) approved cemiplimab-rwlc (Libtayo, Regeneron Pharmaceuticals, Inc.) for the first-line treatment of patients with locally advanced non-small cell lung cancer (NSCLC) who are not candidates for surgical resection or definitive chemoradiation or metastatic NSCLC whose tumours have high PD-L1 expression (Tumour Proportion Score [TPS] > 50%) as determined by an FDA-approved test, with no EGFR, ALK or ROS1 aberrations.

Efficacy was evaluated in Study 1624 (NCT03088540), a multicentre, randomised, open-label study in 710 patients with locally advanced NSCLC who were not candidates for surgical resection or definitive chemoradiation or with metastatic NSCLC. Patients were randomised (1:1) to receive cemiplimab-rwlc 350 mg intravenously every 3 weeks for up to 108 weeks or a platinum-based chemotherapy. The main efficacy outcome measures were overall survival (OS) and progression-free survival (PFS) per blinded independent central review (BICR).

The study demonstrated statistically significant improvements in OS and PFS for patients receiving cemiplimab-rwlc compared to those treated with platinum-based chemotherapy. Median OS was 22.1 months (95% confidence interval [CI] 17.7, NE) for patients in the cemiplimab-rwlc arm compared with 14.3 months (95% CI 11.7, 19.2) in the chemotherapy arm (HR 0.68; 95% CI 0.53, 0.87, p=0.0022). Median PFS per BICR was 6.2 months (4.5, 8.3) in the cemiplimab-rwlc arm and 5.6 months (4.5, 6.1) in the chemotherapy arm (HR 0.59; 95% CI 0.49, 0.72, p<0.0001). Confirmed overall response rate per BICR was 37% (95% CI 32, 42) and 21% (95% CI 17, 25) in the cemiplimab-rwlc and chemotherapy arms, respectively.

The most common adverse reactions (>10%) with cemiplimab-rlwc as a single agent in Study 1624 were musculoskeletal pain, rash, anaemia, fatigue, decreased appetite, pneumonia and cough.

The recommended cemiplimab-rwlc dose for treatment of NSCLC is 350 mg every 3 weeks, intravenously over 30 minutes.

Full prescribing information for Libtayo is available here.

This review used the Assessment Aid, a voluntary submission from the applicant to facilitate the FDA’s assessment. This application was granted priority review.

Healthcare professionals should report all serious adverse events suspected to be associated with the use of any medicine and device to FDA’s MedWatch Reporting System.

For assistance with single-patient INDs for investigational oncology products, healthcare professionals may contact FDA’s Oncology Center of Excellence Project Facilitate.