On February 9, 2021, the U.S. Food and Drug Administration (FDA) approved cemiplimab-rwlc (Libtayo®) for use in patients with locally advanced basal cell carcinoma (laBCC) previously treated with a hedgehog pathway inhibitor (HHI) or for whom an HHI is not appropriate. FDA also granted accelerated approval to cemiplimab-rwlc for patients with metastatic BCC (mBCC) previously treated with an HHI or for whom an HHI is not appropriate. 

FDA Approves Cemiplimab-Rwlc for Locally Advanced and Metastatic Basal Cell Carcinoma

Efficacy was evaluated in an ongoing, open-label, multicenter, nonrandomized trial (Study 1620; NCT03132636) of patients with laBCC or mBCC who had progressed on HHI therapy, had no objective response after nine months on HHI therapy, or were intolerant of prior HHI therapy. Eligibility required that laBCC patients were not candidates for curative surgery or curative radiation therapy per multidisciplinary assessment. All patients received cemiplimab-rwlc 350 mg every three weeks for up to 93 weeks until they experienced disease progression or unacceptable toxicity or completed planned treatment. 

The main efficacy outcome measures were confirmed objective response rate (ORR) and duration of response (DOR) as assessed by independent central review. For patients without externally visible target lesions (mBCC), confirmed ORR was assessed according to response evaluation criteria in solid tumors (RECIST) 1.1. Investigators used a composite response assessment incorporating clinical response criteria with digital medical photography and RECIST 1.1 for those with externally visible target lesions (laBCC and mBCC). 

Among 84 patients with laBCC, confirmed ORR was 29% (95% CI = 19, 40) with a median DOR not reached (2.1—21.4+ months) and 79% of responders maintaining their response for at least six months. Among 28 patients with mBCC, confirmed ORR was 21% (95% CI = 8, 41) with a median DOR not reached (9—23.0+ months) and all responders maintaining their responses for at least six months. 

Severe adverse reactions were immune-mediated adverse events—including pneumonitis, hepatitis, colitis, adrenal insufficiency, hypo- and hyperthyroidism, diabetes mellitus, and nephritis—and infusion reactions. The most common adverse reactions (≥ 20%) were fatigue, musculoskeletal pain, diarrhea, rash, and pruritis.   

The recommended dosage of cemiplimab-rwlc is 350 mg via IV infusion over 30 minutes every three weeks until patients experience disease progression or unacceptable toxicity. 

View full prescribing information for cemiplimab-rwlc

The review used Assessment Aid, a voluntary submission from the applicant to facilitate FDA’s assessment. 

FDA granted the application priority review. A description of FDA expedited programs is in the Guidance for Industry—Expedited Programs for Serious Conditions—Drugs and Biologics

Healthcare professionals should report all serious adverse events suspected to be associated with the use of any medicine and device to FDA’s MedWatch Reporting System or by calling 800-FDA-1088.   

For assistance with single-patient oncology investigational new drug applications, contact OCE’s Project Facilitate at 240-402-0004 or email OncProjectFacilitate@fda.hhs.gov