FDA Approves Cemiplimab (Libtayo), First Drug for Advanced CSCC

Nick Mulcahy

September 28, 2018

The US Food and Drug Administration (FDA) today approved cemiplimab (Libtayo, Regeneron Pharmaceuticals) for the treatment of patients with metastatic cutaneous squamous cell carcinoma (CSCC), or locally advanced, unresectable CSCC.

The new drug is the first FDA approval specifically for advanced CSCC.

Cemiplimab, an injection for intravenous use, is an immunotherapy that targets the PD-1 pathway and thus may help the body's immune system fight the cancer cells.

"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," Richard Pazdur, MD, director of the FDA's Oncology Center of Excellence and acting director of the Office of Hematology and Oncology Products in the Center for Drug Evaluation and Research, said in a press statement.

Pazdur pointed out that this is the sixth different immune checkpoint inhibitor targeting the PD-1/PD-L1 pathway approved by the FDA for treating various tumors, which now includes CSCC.

"We're continuing to see a shift in oncology toward identifying and developing drugs aimed at a specific molecular target," Pazdur commented.

The safety and efficacy of cemiplimab was evaluated in two open-label clinical trials, with a total of 108 patients (75 with metastatic disease and 33 with locally advanced disease) included in the efficacy evaluation.

Nearly half of the patients (47.2%) treated with cemiplimab had an objective response, which was the study's primary endpoint. The majority of these responses were ongoing at the time of data analysis.

Common side effects of cemiplimab include fatigue, rash, and diarrhea. As with other immunotherapies, the drug can cause severe or life-threatening reactions. These include the risk for immune-mediated adverse reactions including pneumonitis, colitis, hepatitis, and endocrinopathies as well as dermatologic and kidney problems. Patients should also be monitored for infusion-related reactions, the FDA said.  

The agency pointed out that CSCC is the second most common human cancer in the United States, with an estimated annual incidence of approximately 700,000 cases; the majority are cured with surgical resection. Only a small percentage of CSCC patients, the FDA said, will develop advanced disease that no longer responds to local treatments including surgery and radiation and thus are candidates for cemiplimab.

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