February 9, 2021 - The FDA has approved cemiplimab-rwlc as the first immunotherapy for use in patients with advanced basal cell carcinoma that has previously been treated with a hedgehog pathway inhibitor (HHI) or for whom a HHI is not appropriate.
The FDA has approved cemiplimab-rwlc (Libtayo) as the first immunotherapy for use in patients with advanced basal cell carcinoma (BCC) that has previously been treated with a hedgehog pathway inhibitor (HHI) or for whom a HHI is not appropriate.
The agent was granted full approval for use in patients with locally advanced BCC; it received accelerated approval for use in patients with metastatic BCC.
The regulatory decision was based on data from an open-label, multicenter, non-randomized phase 2 trial, which included a total of 132 patients with unresectable locally advanced BCC or metastatic BCC, either nodal or distant.
A total of 112 patients were included in the efficacy analysis. Of these patients, 28 had metastatic BCC and 84 had locally advanced BCC. Participants in both cohorts had either progressed on HHI therapy, had not achieved an objective response following 9 months of HHI treatment, or were intolerant of previous HHI therapy.
Results from the trial showed that cemiplimab elicited a confirmed objective response rate of 21% (95% CI, 8%-41%) in patients with metastatic BCC and 29% (95% CI, 19%-40%) in those with locally advanced BCC.
“Patients with advanced forms of basal cell carcinoma face a very difficult prognosis,” Peter Adamson, global development head of Oncology and Pediatric Innovation at Sanofi, stated in the press release. “Thanks to the participation and support of researchers, clinicians, and patients around the world, we are proud to bring forward a new immunotherapy treatment option for appropriate patients in the United States affected by advanced BCC, another devastating non-melanoma skin cancer.”
Additional results from the trial showed that no patients with metastatic BCC achieved a CR with the immunotherapy, while 21% (n = 6) achieved a partial response (PR). The median duration of response (DOR) this subgroup had not yet been reached (range, 9-23+) and all patients who achieved a PR experienced a DOR of at least 6 months.
Among the patients with locally advanced BCC, 6% (n = 5) achieved a CR with cemiplimab and 23% (n = 19) achieved a PR. Here, the median DOR had also not yet been reached (range, 2-21+) and 79% (n = 19) of patients had a DOR of at least 6 months.
Among 132 patients analyzed for safety, adverse effects reported in at least 15% of those who received cemiplimab included fatigue, musculoskeletal pain, diarrhea, rash, pruritus, and upper-respiratory tract infection. Thirty-two percent of patients experienced serious toxicities with the immunotherapy; these included urinary tract infection, colitis, acute kidney injury, adrenal insufficiency, anemia, infected neoplasm, and somnolence. Thirteen percent of patients discontinued treatment due to toxicities.
FDA approves Libtayo (cemiplimab-rwlc) as first immunotherapy indicated for patients with advanced basal cell carcinoma. News release. Sanofi. February 9, 2021. Accessed February 9, 2021. http://bit.ly/2LEYHZk.