FDA Grants Regular Approval to Cemiplimab for Metastatic Basal Cell Carcinoma


The FDA has granted full approval to cemiplimab-rwlc for the treatment of patients with metastatic basal cell carcinoma who previously received a hedgehog inhibitor (HHI) or for whom a HHI is not appropriate.



The FDA has granted full approval to cemiplimab-rwlc (Libtayo) for the treatment of patients with metastatic basal cell carcinoma who previously received a hedgehog inhibitor (HHI) or for whom a HHI is not appropriate.1,2

The agent received accelerated approval for this indication in February 2021 based on findings from an open-label, multicenter, non-randomized phase 2 trial (Study 1620; NCT03132636).3 The regulatory agency had also granted full approval to cemiplimab in those with locally advanced disease.

This trial enrolled a total of 138 patients with locally advanced or metastatic, nodal or distant, basal cell carcinoma who had progressed on a HHI, had not responded after 9 months of treatment with an HHI, or who proved to be intolerant to previous HHI treatment.4

If patients had autoimmune disease that needed systemic treatment with immunosuppressant agents within 5 years; a history of solid organ transplant; previously received anti–PD-1/PD-L1 therapy or other immune checkpoint inhibitors; infection with human immunodeficiency virus, hepatitis B or hepatitis C; or had an ECOG performance status of 2 or higher, they were excluded.

Study participants were given cemiplimab at 350 mg every 3 weeks for up to 93 weeks. Treatment was continued until progressive disease, intolerable toxicity, or completion.

Objective response rate (ORR) and duration of response (DOR) served as the trial's primary endpoints of the research.

For those who did not have exernally visible lesions, investigators evaluated ORR by RECIST v1.1 criteria. For those with externally visible lesions, they determined ORR by RECIST v1.1 criteria and World Health Organization criteria.

Of the 138 total patients who were evaluated in the efficacy analysis of the trial, 61% had locally advanced disease and 39% had metastatic disease.

In those with locally advanced disease (n = 84), the median age was 70 years (range, 42-89), and most patients were male (67%), White (68%), and had an ECOG performance status of 0 (61%). Additionally, 83% of patients had undergone at least 1 cancer-related surgical procedure and 50% previously received radiotherapy.

In those with metastatic disease (n = 54), the median age was 63.5 years (range, 38-90), and 70% were male. Eighty-seven percent of patients were White and 67% had an ECOG performance status of 0. In this group, 35% had distant metastases only, 9% had nodal disease only, and 54% had distant and nodal disease. Most patients (85%) previously underwent at least 1 cancer-related surgery and 59% received prior radiotherapy.

Data showed that cemiplimab elicited an ORR of 24% (95% CI, 13%-38%) in those with metastatic basal cell carcinoma and 32% (95% CI, 22%-43%) in those with locally advanced basal cell carcinoma. The complete response rates in these groups were 2% and 7%, respectively, and the partial response rates were 22% and 25%, respectively.

Among the 13 responders in the metastatic group, the median DOR was 16.7 months (range, 4.8-25.8+), with 92% of patients experiencing a DOR that lasted for at least 6 months. Among the 27 responders in the locally advanced group, the median DOR was not yet reached (range, 2.1-36.8+) with 85% of patients having responded for at least 6 months.

The safety of cemiplimab was evaluated in all 138 patients who had a median exposure of 45 weeks (range, 2.1-98) to the drug. Thirty-four percent of patients experienced serious toxicities with the agent, the most common of which included diarrhea (3.6%), urinary tract infection (3.6%), pneumonia (2.9%), and hemorrhage (2.2%).

Moreover, 4.3% of patients experienced adverse reactions that proved to be fatal; these patients experienced acute kidney injury (0.7%) and cachexia worsending because of colitis (0.7%).

Adverse effects (AEs) resulted in dose interruptions for 40% of patients. Fourteen percent of patients experienced AEs that resulted in the permanent discontinuation of the agent; these included diarrhea, acute kidney injury, hepatits, and general deterioration in physical health.

The most common AEs observed with cemiplimab, that were expereinced by 15% or more of participants, included fatigue (50%), musculoskeletal pain (36%), diarrhea (33%), rash (30%), upper respiratory tract infection (22%), pruritus (19%), hemorrhage (18%), and hypertension (17%).

The most frequently experienced grade 3 or 4 toxicities were hypertension, diarrhea, fatigue, musculoskeletal pain, hypokalemia, hyponatremia, pneumonia, urinary tract infection, visual impairment and decreased weight.


  1. Drugs@FDA: FDA-approved drugs. FDA. Accessed May 1, 2023.
  2. Supplemental approval/fulfillment of postmarketing requirement. Letter. FDA. Accessed May 1, 2023.
  3. FDA approves Libtayo (cemiplimab-rwlc) as first immunotherapy indicated for patients with advanced basal cell carcinoma. News release. Sanofi. February 9, 2021. Accessed May 1, 2023.
  4. Cemiplimab-rwlc (Libtayo). Prescribing information. Regeneron. Updated April 2023. Accessed May 1, 2023.
Related Videos
Zeynep Eroglu, MD
Zeynep Eroglu, MD
Zeynep Eroglu, MD
Zeynep Eroglu, MD
Daniel Olson, MD
Vishal Patel, MD, FAAD, FACMS, associate professor, Dermatology, George Washington (GW) School of Medicine & Health Sciences
Omid Hamid, MD, professor, medicine, Cedars-Sinai; director, Clinical Research and Immunotherapy, director, Cutaneous Oncology and Melanoma, The Angeles Clinic and Research Institute
John M. Kirkwood, MD, Distinguished Service Professor of Medicine, Sandra and Thomas Usher Professor of Medicine, Dermatology & Translational Science, coleader, Melanoma and Skin Cancer Program, Division of Hematology/Oncology, the University of Pittsburgh
Daniel Olson, MD
Omid Hamid, MD