FDA Approves Cemiplimab for First-Line Advanced NSCLC With PD-L1 Expression

The PD-1 inhibitor cemiplimab-rwlc, sold as Libtayo, was FDA approved as a monotherapy for patients with first-line advanced non–small cell lung cancer (NSCLC) with PD-L1 expression of at least 50%.

Regeneron Pharmaceuticals, Inc. and Sanofi today announced that the PD-1 inhibitor cemiplimab-rwlc, sold as Libtayo, received FDA approval as a monotherapy for patients with first-line advanced non–small cell lung cancer (NSCLC) with PD-L1 expression of at least 50% as determined by an FDA-approved test.1 Patient eligibility criteria also include having either metastatic or locally advanced NSCLC that cannot be resected or treated with definitive chemoradiation, and tumors cannot have EGFR, ALK, or ROS1 aberrations.

The approval is based on an analysis of 710 participants in the phase 3, open-label EMPOWER-Lung 1 trial (NCT03088540), which randomized patients 1:1 to receive either cemiplimab or chemotherapy. Patients were intended to have high PD-L1 expression, and the cemiplimab cohort had a 32% lower risk of death than the chemotherapy group in the overall population.

A prespecified analysis was performed on a cohort of 563 patients with confirmed PD-L1–high tumors proven by the PD-L1 IHC 22C3 pharmDx kit, and those patients showed a 43% lower risk of death compared with those treated with chemotherapy. The results were published recently in The Lancet.2

“Libtayo has demonstrated an impressive level of efficacy in advanced NSCLC with at least 50% PD-L1 expression in its pivotal trial,” study investigator Ahmet Sezer, MD, professor in the department of medical oncology at Başkent University in Adana, Turkey, said in a statement. “As published in The Lancet, a prespecified analysis in the subset of patients proven to have PD-L1 expression of at least 50%, Libtayo reduced the risk of death by 43% compared to chemotherapy. This was achieved with a greater than 70% crossover rate to Libtayo following disease progression on chemotherapy, as well as the largest population of patients with pretreated and clinically stable brain metastases among advanced NSCLC pivotal trials to date.”

The approval of cemiplimab for this indication comes after a priority review by the FDA, which is granted when a drug may significantly improve patient outcomes in serious diseases. It is the third cemiplimab approval in the U.S. and the second in February 2021 alone.

Earlier in the month, the drug was approved for the treatment of advanced basal cell carcinoma (BCC) previously treated with a hedgehog pathway inhibitor (HHI) or in cases when an HHI is not appropriate.3 It was granted full approval for locally advanced BCC and accelerated approval for patients with metastatic BCC. In 2018, it cemiplimab was approved for the treatment of locally advanced or metastatic cutaneous squamous cell when curative surgery or radiation is not an option.4

“With this third approval for Libtayo, we are proud to deliver on our ambition to bring our PD-1 inhibitor to patients in need with difficult-to-treat cancers, such as advanced non-small cell lung cancer," Peter C. Adamson, MD, global development head of Oncology and Pediatric Innovation at Sanofi, said. "As the leading cause of cancer deaths globally, the need for additional therapeutic options in advanced NSCLC is clear. Libtayo allows physicians to further optimize treatment of these patients whose tumors have high expression of PD-L1. We thank all of the trial investigators, patients and their caregivers who helped make this milestone possible.”

In the trial, 355 patients in the cemiplimab cohort and 342 in the chemotherapy group were included in the safety assessment. In the cemiplimab group, rash and cough occurred more often than in the chemotherapy group and in at least 10% of patients (15% vs 6% and 11% vs 8%, respectively). The most common serious adverse reactions were pneumonia and pneumonitis, which occurred in 5% and 2%, respectively, of cemiplimab patients; and 6% and 0% of chemotherapy patients. Overall, no new safety signals were found in the cemiplimab cohort.

“Libtayo has already changed the treatment paradigm for certain patients with advanced cutaneous squamous cell carcinoma and is poised to do the same for advanced basal cell carcinoma,” said Israel Lowy, MD, PhD, senior vice president of clinical sciences, head of translational science, and head of oncology at Regeneron. “Now, Libtayo has the opportunity to make a meaningful difference for the many U.S. patients battling advanced non-small cell lung cancer. Libtayo is being investigated in a variety of settings, and we hope to share updates later this year on our pivotal trials in cervical cancer and in combination with chemotherapy in advanced non-small cell lung cancer.”


1. FDA approves Libtayo (cemiplimab-rwlc) monotherapy for patients with first-line advanced non-small cell lung cancer with PD-L1 expression of ≥50%. News release. Regeneron Pharmaceuticals, Inc; February 22, 2021. Accessed February 22, 2021. https://investor.regeneron.com/news-releases/news-release-details/fda-approves-libtayor-cemiplimab-rwlc-monotherapy-patients-first

2. Cemiplimab monotherapy for first-line treatment of advanced non-small-cell lung cancer with PD-L1 of at least 50%: a multicentre, open-label, global, phase 3, randomised, controlled trial. Lancet. 2021;397(10274):592-604. doi:10.1016/S0140-6736(21)00228-2

3. FDA approves Libtayo (cemiplimab-rwlc) as first immunotherapy indicated for patients with advanced basal cell carcinoma. News release. Regeneron Pharmaceuticals, Inc; February 9, 2021. Accessed February 22, 2021. https://investor.regeneron.com/news-releases/news-release-details/fda-approves-libtayor-cemiplimab-rwlc-first-immunotherapy

4. FDA approves Libtayo (cemiplimab-rwlc) as first and only treatment for advanced cutaneous squamous cell carcinoma. Regeneron Pharmaceuticals, Inc; September 28, 2018. Accessed February 22, 2021. https://investor.regeneron.com/news-releases/news-release-details/fda-approves-libtayor-cemiplimab-rwlc-first-and-only-treatment