Sanofi and Regeneron’s PD-1 inhibitor Libtayo (cemiplimab) has been approved by the European Commission as the first immunotherapy to treat adults with locally advanced or metastatic basal cell carcinoma (BCC).

Specifically, the drug can now be prescribed to treat patients who have progressed on or are intolerant to a hedgehog pathway inhibitor (HHI).

Libtayo's approval in BCC follows data the largest prospective clinical trial (n=119) in patients with advanced BCC previously treated with an HHI to date. 

Patients with locally advanced BCC given the immunotherapy experienced an objective response rate (ORR) of 32% (95% confidence interval [CI]: 22-43) (25% partial response, 7% complete response) by independent central review. 

Libtayo-treated patients with metastatic BCC demonstrated an ORR of 29% (95% CI: 15-46) (26% partial response, 3% complete response) by investigator assessment. 

Also of note, around 90% of patients across both groups had a duration of response (DOR) of 6 months or longer per Kaplan Meier estimates, and the median DOR has not been reached for either group. Median duration of follow-up was 16 months for locally advanced BCC and 9 months for metastatic BCC.

On the safety side, of 816 patients across all four Libtayo monotherapy pivotal trials in its approved indications, adverse events were serious in 30% of patients and led to permanent discontinuation in 8% of patients.

Immune-related adverse reactions occurred in 22% of patients and led to permanent discontinuation in 4% of patients. The most common immune-related adverse reactions were hypothyroidism (8%), hyperthyroidism (3%), pneumonitis (3%), hepatitis (2%), colitis (2%) and immune-related skin adverse reactions (2%).


“Libtayo is the first immunotherapy to show a clinical benefit in patients with advanced BCC after HHI therapy in a pivotal trial, and with this first-in-class approval has the potential to transform treatment for patients in Europe whose cancer has progressed despite HHI treatment,” said Israel Lowy, senior vice president, Translational and Clinical Sciences, Oncology at Regeneron. “We look forward to continuing to investigate this medicine in additional settings, with the goal of helping more patients with difficult-to-treat cancers around the world.”


Libtayo is now approved for three advanced cancers in the EU: BCC, non-small cell lung cancer (NSCLC) and cutaneous squamous cell carcinoma (CSCC).