Benzinga | Jun 25, 2021 05:40AM EDT

Regeneron Pharmaceuticals and Sanofi Announced Libtayo Approved by the European Commission as the First Immunotherapy Indicated for Patients with Advanced Basal Cell Carcinoma

Regeneron Pharmaceuticals, Inc. (NASDAQ:REGN) and Sanofi (NYSE:SNY) today announced that the European Commission (EC) has approved the PD-1 inhibitor Libtayo(r) (cemiplimab) to treat adults with locally advanced or metastatic basal cell carcinoma (BCC) who have progressed on or are intolerant to a hedgehog pathway inhibitor (HHI).

BCC is the most common type of skin cancer worldwide, representing up to 80% of non-melanoma skin cancers, and incidence is increasing across many European countries. While the large majority of BCCs are caught early and easily cured with surgery and/or radiation, a small proportion of cases can develop into advanced BCC and penetrate deeper into surrounding tissues (locally advanced) or spread to other parts of the body (metastatic), becoming more difficult to treat.

"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, M.D., Ph.D., 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 European Union, following the EC's concurrent approval of Libtayo for the first-line treatment of adult patients with advanced non-small cell lung cancer (NSCLC) whose tumor cells have 50% PD-L1 expression and no EGFR, ALK or ROS1 aberrations. In 2019, Libtayo was approved by the EC as the first treatment for adults with metastatic or locally advanced cutaneous squamous cell carcinoma (CSCC) who are not candidates for curative surgery or curative radiation. Across all of its approved indications, Libtayo had a generally consistent safety profile. Immune-mediated adverse reactions, which may be severe or fatal, can occur in any organ system or tissue during or after treatment with Libtayo.

The EC approval in BCC is based on data from the largest prospective clinical trial (n=119) in patients with advanced BCC previously treated with an HHI to date. Libtayo-treated patients with locally advanced BCC 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. In addition, approximately 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.

Safety was assessed in 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. Immunerelated 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%).

"Since its launch in Europe just two years ago, Libtayo has redefined the standard of care for advanced CSCC and has the potential to do the same in advanced BCC," said Peter C. Adamson, M.D., Global Development Head, Oncology at Sanofi. "Together with Regeneron, we're committed to addressing gaps in the treatment of advanced forms of non-melanoma skin cancer."Regeneron Pharmaceuticals, Inc. (NASDAQ:REGN) and Sanofi today announced that the European Commission (EC) has approved the PD-1 inhibitor Libtayo(r) (cemiplimab) to treat adults with locally advanced or metastatic basal cell carcinoma (BCC) who have progressed on or are intolerant to a hedgehog pathway inhibitor (HHI).

BCC is the most common type of skin cancer worldwide, representing up to 80% of non-melanoma skin cancers, and incidence is increasing across many European countries. While the large majority of BCCs are caught early and easily cured with surgery and/or radiation, a small proportion of cases can develop into advanced BCC and penetrate deeper into surrounding tissues (locally advanced) or spread to other parts of the body (metastatic), becoming more difficult to treat.

"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, M.D., Ph.D., 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 European Union, following the EC's concurrent approval of Libtayo for the first-line treatment of adult patients with advanced non-small cell lung cancer (NSCLC) whose tumor cells have 50% PD-L1 expression and no EGFR, ALK or ROS1 aberrations. In 2019, Libtayo was approved by the EC as the first treatment for adults with metastatic or locally advanced cutaneous squamous cell carcinoma (CSCC) who are not candidates for curative surgery or curative radiation. Across all of its approved indications, Libtayo had a generally consistent safety profile. Immune-mediated adverse reactions, which may be severe or fatal, can occur in any organ system or tissue during or after treatment with Libtayo.

The EC approval in BCC is based on data from the largest prospective clinical trial (n=119) in patients with advanced BCC previously treated with an HHI to date. Libtayo-treated patients with locally advanced BCC 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. In addition, approximately 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.

Safety was assessed in 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. Immunerelated 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%).

"Since its launch in Europe just two years ago, Libtayo has redefined the standard of care for advanced CSCC and has the potential to do the same in advanced BCC," said Peter C. Adamson, M.D., Global Development Head, Oncology at Sanofi. "Together with Regeneron, we're committed to addressing gaps in the treatment of advanced forms of non-melanoma skin cancer."