TARRYTOWN, NY & PARIS—Regeneron Pharmaceuticals, Inc. and Sanofi SA have received the news that the FDA has approved the companies’ PD-1 inhibitor Libtayo (cemiplimab-rwlc) as the first immunotherapy treatment indicated for patients with advanced basal cell carcinoma (BCC) previously treated with a hedgehog pathway inhibitor (HHI), or for whom an HHI is not appropriate. Full approval was granted for patients with locally advanced BCC, and accelerated approval was granted for patients with metastatic BCC.
BCC is the most common type of skin cancer in the US, with approximately two million new cases diagnosed each year. While the vast majority of BCCs are caught early and cured with surgery and/or radiation, a small proportion of tumors can become locally advanced, or metastatic.
“Today’s FDA approval of Libtayo will change the treatment paradigm for patients with advanced basal cell carcinoma. Advanced basal cell carcinoma is a persistent, painful, and highly disfiguring cancer. While the primary systemic treatment options are hedgehog inhibitors, many patients will eventually progress on or become intolerant to this therapy,” said Dr. Karl Lewis, professor in the Division of Medical Oncology at the University of Colorado, and a trial investigator. “With Libtayo, these patients now have a new immunotherapy option that has demonstrated clinically meaningful and durable anti-tumor responses.”
Libtayo is said to be the first treatment to show a clinical benefit in patients with advanced BCC after HHI therapy in a pivotal trial. The full approval in locally advanced BCC is based on the primary analysis from the trial. For metastatic BCC, the accelerated approval is based on an interim analysis showing the impact of Libtayo on tumor response rate, and durability of response. Continued approval in metastatic BCC could be contingent on additional data from the trial, verifying clinical benefit.
“With today’s approval, Libtayo is now approved for both advanced cutaneous squamous cell and basal cell carcinomas, building a strong foundation in dermato-oncology,” noted Dr. Israel Lowy, senior vice president of Translational and Clinical Sciences, Oncology, at Regeneron. “Beyond skin cancers, we also continue to investigate the potential of Libtayo in other difficult-to-treat cancers, starting with non-small cell lung cancer [NSCLC], where an FDA decision is expected by the end of February.”
This marks the second US approval for Libtayo. The therapy was approved in 2018 as the first systemic treatment for adults with metastatic cutaneous squamous cell carcinoma (CSCC) or locally advanced CSCC who are not candidates for curative surgery or curative radiation.
“Patients with advanced forms of basal cell carcinoma face a very difficult prognosis. Thanks to the participation and support of researchers, clinicians, and patients around the world, we are proud to bring forward a new immunotherapy treatment option for appropriate patients in the US affected by advanced BCC, another devastating non-melanoma skin cancer,” added Peter Adamson, global development head of Oncology and Pediatric Innovation at Sanofi. “Together with Regeneron, we continue to develop Libtayo in numerous clinical trials and settings, including as monotherapy and in combination with several other therapeutic approaches as part of our commitment to innovation towards meaningful treatment options for patients with significant unmet needs.”
The European Medicines Agency (EMA) is assessing Libtayo in both advanced NSCLC, and locally advanced BCC following treatment with a hedgehog inhibitor. Decisions from the European Commission are expected in mid-2021.
The clinical program for Libtayo is focused on difficult-to-treat cancers. In skin cancer, this includes trials in adjuvant and neoadjuvant CSCC. Libtayo is also being investigated in pivotal trials in NSCLC (in combination with chemotherapy) and cervical cancer, as well as in trials combining Libtayo with either conventional or novel therapeutic approaches for both solid tumors and blood cancers.