FDA Approves First Immunotherapy for Patients With Advanced Basal Cell Carcinoma


Libtayo is the first treatment to show a clinical benefit in patients with advanced basal cell carcinoma after hedgehog pathway inhibitor therapy in a pivotal trial.

Cemiplimab-rwlc (Libtayo, Regeneron) has been granted FDA approval as the first immunotherapy treatment indicated for patients with advanced basal cell carcinoma (BCC) who were previously treated with a hedgehog pathway inhibitor (HHI) or for whom an HHI is not appropriate.

Libtayo is a programmed cell death ligand-1 (PD-1) inhibitor and is the first treatment to show a clinical benefit in patients with advanced BCC after HHI therapy in a pivotal trial. As a fully human monoclonal antibody that binds to PD-1 on T cells, it has been shown to block cancer cells from using the PD-1 pathway to suppress T cell activation.

“Today’s FDA approval of Libtayo will change the treatment paradigm for patients with advanced basal cell carcinoma,” said trial investigator Karl Lewis, MD, 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.”

The open-label, multicenter, non-randomized phase 2 trial included patients with unresectable locally advanced BCC or metastatic BCC and was the largest prospective clinical trial among this patient population. It included 132 total participants, with 112 patients included in the efficacy analysis. Patients in both cohorts had either progressed on HHI therapy, had not had an objective response after 9 months on HHI therapy, or were intolerant of prior HHI therapy.

The primary efficacy endpoint was confirmed objective response rate (ORR) and a key secondary endpoint was duration of response (DOR). According to a press release, the confirmed ORR was 21% among patients with metastatic BCC and 29% for patients with locally advanced BCC. Furthermore, 6% of patients with locally advanced BCC achieved a complete response (CR), whereas no patients in the metastatic BCC cohort achieved CR.

When investigators analyzed DOR, they found that 100% of patients with metastatic BCC achieved a DOR of 6 months or longer, compared with 79% of patients with locally advanced BCC.

The most common adverse events (AEs) reported in at least 15% of patients were fatigue, musculoskeletal pain, diarrhea, rash, pruritus, and upper respiratory tract infection. Serious AEs occurred in 32% of patients and included urinary tract infection, colitis, acute kidney injury, adrenal insufficiency, anemia, infected neoplasm, and somnolence. AEs resulting in permanent discontinuation occurred in 13% of patients.


FDA Approves Libtayo (Cemiplimab-rwlc) As First Immunotherapy Indicated For Patients With Advanced Basal Cell Carcinoma [news release]. Regeneron; February 9, 2021. https://investor.regeneron.com/news-releases/news-release-details/fda-approves-libtayor-cemiplimab-rwlc-first-immunotherapy. Accessed February 10, 2021.

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