On February 9, 2021, the FDA approved a new indication for cemiplimab-rwlc (Libtayo; Regeneron/sanofi-aventis) for the treatment of locally advanced or metastatic basal-cell carcinoma (BCC) in patients who had received or are ineligible to receive a hedgehog inhibitor. The FDA granted cemiplimab a priority review and accelerated its approval for this indication.
Cemiplimab was previously approved for metastatic or locally advanced cutaneous squamous-cell carcinoma.
This FDA approval of cemiplimab for BCC was based on results from Study 1620, a multicenter, nonrandomized, open-label clinical trial of 84 patients with locally advanced BCC and 28 patients with metastatic BCC. The main efficacy measures were objective response rate (ORR) and duration of response (DOR).
The ORR was 29% (95% confidence interval [CI], 19-40) in those with locally advanced BCC, and the median DOR was not reached; 79% of the patients maintained their responses for ≥6 months. Among the patients with metastatic BCC, the confirmed ORR was 21% (95% CI, 8-41), and the median DOR was not reached; all the responders maintained their response for ≥6 months.
The most common (≥20%) adverse reactions were fatigue, musculoskeletal pain, diarrhea, rash, and pruritus. Severe adverse reactions were immune-mediated adverse reactions (ie, pneumonitis, hepatitis, colitis, adrenal insufficiency, hypo- and hyperthyroidism, diabetes, and nephritis) and infusion reactions.
On February 22, 2021, the FDA approved cemiplimab for the first-line treatment of patients with advanced or metastatic non–small-cell lung cancer (NSCLC) who are not candidates for resection or definitive chemoradiation and whose tumors have high PD-L1 expression and no EGFR, ALK, or ROS1 mutations. The FDA granted this indication priority review.
The approval was based on data from Study 1624, a multicenter, randomized, open-label clinical trial of 710 patients with metastatic NSCLC or patients with advanced NSCLC who were not candidates for resection or chemoradiation. The patients were randomized to cemiplimab 350 mg intravenously every 3 weeks for 108 weeks or to platinum-based chemotherapy. The main end points were overall survival (OS) and progression-free survival (PFS).
The median OS was 22.1 months (95% confidence interval [CI], 17.7-not estimable) with cemiplimab versus 14.3 months (95% CI, 11.7-19.2) with chemotherapy. The median PFS was 6.2 months (95% CI, 4.5-8.3) with cemiplimab versus 5.6 months (95% CI, 4.5-6.1) with chemotherapy. The overall response rate was 37% (95% CI, 32%-42%) with cemiplimab and 21% (95% CI, 17%-25%) with chemotherapy.
The most common (≥10%) adverse reactions with cemiplimab were musculoskeletal pain, rash, anemia, fatigue, decreased appetite, pneumonia, and cough.