The FDA granted full approval for the locally advanced BCC indication and accelerated approval for the metastatic BCC indication, according to a press release from Regeneron and Sanofi, the companies jointly developing cemiplimab.
Cemiplimab is a programmed death–1 inhibitor that was first FDA approved in 2018 for locally advanced or metastatic cutaneous squamous cell carcinoma not eligible for curative surgery or radiation.
The new approval “will change the treatment paradigm for patients with advanced basal cell carcinoma,” according to Karl Lewis, MD, a professor at the University of Colorado at Denver, Aurora, and an investigator on the phase 2 trial of cemiplimab.
“While the primary systemic treatment options are hedgehog inhibitors, many patients will eventually progress on or become intolerant to this therapy,” Dr. Lewis said in the press release. “With Libtayo [cemiplimab], these patients now have a new immunotherapy option.”
The approval of cemiplimab in BCC was based on an open-label, phase 2 trial of 132 patients with advanced BCC. Patients could not tolerate, had progressed on, or had not responded to HHIs after 9 months of treatment.
Cemiplimab was given at 350 mg every 3 weeks. The study was not placebo controlled and has not been published, a Regeneron spokesperson said.
There were 112 patients in the efficacy analysis. The overall response rate was 21% (6/28) in metastatic BCC patients, with no complete responders. In locally advanced BCC patients, the objective response rate was 29% (24/84), with five complete responders.
The median duration of response was not reached in either group but was at least 6 months long in all metastatic patients and in 79% (19/84) of the locally advanced BCC patients.
The most common adverse events among the 132 subjects evaluable for safety were fatigue (49%), musculoskeletal pain (33%), diarrhea (25%), rash (22%), pruritus (20%), and upper respiratory tract infection (15%).
Serious adverse events occurred in 32% of patients, including colitis, acute kidney injury, adrenal insufficiency, and anemia. Adverse events led to discontinuation in 13% of patients, most often for colitis and general physical health deterioration.
For more details on cemiplimab, see the full prescribing information.