On Friday, September 28, 2018, the U.S. Food and Drug Administration approved cemiplimab (Libtayo®, Regeneron and Sanofi) for the treatment of patients with metastatic cutaneous squamous cell carcinoma (CSCC) as well as patients with locally advanced CSCC for whom surgery or radiation is not applicable.
"Today's FDA decision is great news for patients with advanced CSCC, who previously had no approved treatment options. This is especially true because these patients are no longer candidates for curative surgery or radiation," said The University of Texas MD Anderson Cancer Center’s Michael R. Migden, M.D., a lead investigator on these pivotal studies, in a press release.
This approval, the first for cemiplimab, a checkpoint immunotherapy that targets the PD-1 pathway, is also the first option to be approved for patients with advanced forms of CSCC, the second most common skin cancer in the U.S. and the deadliest non-melanoma skin cancer. The approval was based on results from the phase 2 EMPOWER-CSCC-1 study plus two expansion cohorts from the Phase 1 clinical trial.
Among 108 total patients, 47% responded to the immunotherapy treatment and 4% experienced complete responses characterized by total tumor elimination. In this combined population, 61% of responses lasted at least six months. Response rates were not significantly different between the group of patients with metastatic disease (47% response rate) versus those with locally advanced disease (49%), although those with metastatic disease experienced a higher complete response rate (5% vs 0%).
Israel Lowy, M.D., Ph.D., Regeneron’s VP of Global Clinical Development and Head of Translational Science and Clinical Oncology, noted that “by following the science, we identified early on that advanced CSCC was a promising target for investigation with Libtayo.”
"We are proud to offer patients in the U.S. this first and only treatment for advanced CSCC and remain focused on advancing our clinical research investigating Libtayo as a potential monotherapy and combination therapy in other cancer types,” Lowy, who also serves on the Cancer Research Institute’s Cancer Immunotherapy Consortium (CIC), continued.
“We’re continuing to see a shift in oncology toward identifying and developing drugs aimed at a specific molecular target. With the Libtayo approval, the FDA has approved six immune checkpoint inhibitors targeting the the PD-1/PD-L1 pathway for treating a variety of tumors, from bladder to head and neck cancer, and now advanced CSCC,” added Richard Pazdur, M.D., the director of the FDA’s Oncology Center of Excellence. “This type of cancer can be difficult to treat effectively when it is advanced and it is important that we continue to bring new treatment options to patients.”