FDA Approves Cemiplimab as First Immunotherapy to Treat Patients With Advanced BCC


The PD-1 inhibitor cemiplimab was granted approval in patients with advanced basal cell carcinoma previously treated with a hedgehog pathway inhibitor or for whom these therapies are not appropriate.

Cemiplimab (Libtayo) is now approved by the FDA to treat patients with basal cell carcinoma (BCC) following treatment with a hedgehog pathway inhibitor (HHI) or for whom these therapies are not appropriate, according to Regeneron Pharmaceuticals and Sanofi – the drug’s manufacturers.

The fully human monoclonal antibody targeting PD-1 is approved at the recommended dose of 350 mg administered as an intravenous infusion over 30 minutes every 3 weeks until disease progression or unacceptable toxicity.

“Today’s FDA approval of Libtayo will change the treatment paradigm for patients with advanced basal cell carcinoma,’ Karl Lewis, MD, professor in the Division of Medical Oncology at the University of Colorado as well as a trial investigator, said in a press release. “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. With Libtayo, these patients now have a new immunotherapy option that has demonstrated clinically meaningful and durable anti-tumor responses.”

Approval was based on an open-label, multicenter, nonrandomized phase 2 trial of patients with BCC that is locally advanced or metastatic (n = 132). All patients had progressed on or were intolerant to HHI therapy or did not experience a response at 9 months with these agents. The primary end point was objective response rate (ORR) and a key secondary outcome was duration of response (DOR) per independent central review.

ORR in the patients with metastatic disease (n = 28) was 21%, compromised of all partial responses (PRs). In locally advanced tumors (n = 84), the ORR was 29% made up of 5 complete responses and 19 PRs. The median DORs were not reached in either arm and response duration lasting 6 months or greater happened at rates of 100% and 79%, respectively.

In the safety analysis, adverse events (AEs) occurring in at least 15% of patients were fatigue (49%), musculoskeletal pain (33%), diarrhea (25%), rash (22%), pruritis (20%), and upper respiratory tract infections (15%). Serious AEs occurred in 32% of the population, and those that occurred in more than 1 patient each were urinary tract infection, colitis, acute kidney injury, adrenal insufficiency, anemia, infected neoplasm and somnolence. AEs leading to permanent discontinuation happened at a rate of 13%, most commonly attributable to colitis and general physical health deterioration.

BCC is the most common skin cancer is the United States accounting for approximately 2 million new cases per year. Although most cases are caught early and cured, some patients develop advanced tumors that progress to metastatic disease.

“Patients with advanced forms of basal cell carcinoma face a very difficult prognosis,” Peter Adamson, Global Development Head, Oncology and Pediatric Innovation at Sanofi, said in the release. “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 [United States] affected by advanced BCC, another devastating nonmelanoma skin cancer.”

This is the second approval for cemiplimab following its indication for the treatment of patients with metastatic cutaneous squamous cell carcinoma or for patients whose disease is locally advanced and who are not candidates for curative surgery or curative radiation.


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 9, 2021. https://bit.ly/3tIRoB6

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