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WASHINGTON -- The FDA to include certain patients with basal cell carcinoma (BCC), the first immuno-oncology approval for the disease.

The approval applies to advanced BCC previously treated with a hedgehog pathway inhibitor (HHI) or for which an HHI was not appropriate. The FDA granted full approval of cemiplimab for locally advanced BCC and accelerated approval for metastatic BCC.

"FDA approval of Libtayo will change the treatment paradigm for patients with advanced basal cell carcinoma," Karl Lewis, MD, of the University of Colorado Anschutz Medical Campus in Aurora, said in a from Sanofi and Regeneron.

"Advanced basal cell carcinoma is a persistent, painful, and highly disfiguring cancer. While the primary systemic options are hedgehog inhibitors, many patients will eventually progress on or become intolerant to this therapy," he added. "[T]hese patients now have a new immunotherapy option that has demonstrated clinically meaningful and durable antitumor responses in locally advanced BCC."

The FDA initially approved cemiplimab for metastatic cutaneous squamous cell carcinoma (CSCC) and locally advanced CSCC ineligible for curative surgery or radiation.

Supporting data for the BCC indication came from a pivotal phase II trial of unresectable locally advanced or metastatic BCC. The trial included a total of 132 patients, all of whom had progressive disease with an HHI or did not respond or were intolerant to HHI therapy. The primary endpoint was objective response rate (ORR), and duration of response (DOR) was the key secondary outcome.

Efficacy results in 112 evaluable patients showed an ORR of 27% (30 of 112), including six of 28 (21%) patients with metastatic BCC and 24 of 84 (29%) patients with locally advanced disease. Five patients in the locally advanced subgroup had complete responses with cemiplimab.

Median DOR has yet to be reached in the trial, overall or in the metastatic and locally advanced subgroups. The data showed that all six responding patients with metastatic disease had responses lasting at least 6 months, as did 19 of 24 (79%) responding patients in the locally advanced subgroup.

Adverse events (AEs) occurring in at least 15% of patients consisted of fatigue, musculoskeletal pain, diarrhea, rash, pruritus, and upper respiratory tract infection. Serious AEs occurred in a third of patients, including two or more patients with urinary tract infection, colitis, acute kidney injury, adrenal insufficiency, anemia, infected neoplasm, and somnolence. AEs leading to treatment discontinuation occurred in 13% of patients.

Accelerated approval for the metastatic indication is a conditional status, and full approval may require additional data to confirm clinical benefits.

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