New Hope for High-Risk Skin Cancer Patients
The U.S. Food and Drug Administration (FDA) has approved Regeneron Pharmaceuticals’ Libtayo (cemiplimab) as the first immunotherapy for adjuvant treatment of cutaneous squamous cell carcinoma (CSCC) in patients who face a high risk of recurrence following surgery and radiation. The approval comes three years after Regeneron paid Sanofi $900 million to obtain full rights to the cancer drug.
This latest authorization expands Libtayo’s reach beyond its initial approval seven years ago, when it became the first drug available for CSCC patients with metastatic disease or locally advanced cases not eligible for curative surgery or radiation.
Addressing a Significant Patient Population
The patient population affected by this condition is considerable, with approximately 1.8 million diagnoses each year in the United States, representing about 20% of the country’s skin cancer cases. Regeneron has also submitted an application in Europe for the same indication, and a decision is expected in the first half of 2026.
“Patients whose CSCC is at a high risk of recurrence following surgery and radiation often have the poorest outcomes,” said Vishal Patel, M.D., professor of dermatology at George Washington University. “Until now, we lacked options to help prevent a devastating recurrence, and immunotherapy was only available for patients with advanced CSCC, who were no longer candidates for curative surgery or curative radiation,” Patel added that the label expansion is “practice-changing” for this group of patients.
Strong Clinical Trial Results
The FDA’s decision was based on results from a phase 3 trial involving 415 patients. In the study, Libtayo reduced the risk of disease recurrence or death by 68% compared with placebo at a median follow-up of 24 weeks. The finding stands out, as a similar trial conducted by Merck with Keytruda was discontinued last year due to futility in treating patients with high-risk, locally advanced CSCC following surgery and radiation.
Libtayo’s Growing Market Presence
Libtayo’s progress has been steady since Regeneron gained full control of the drug. Shortly after the acquisition, the FDA approved a label expansion allowing its use with chemotherapy as a first-line treatment for advanced non-small cell lung cancer.
The therapy achieved blockbuster sales last year, generating $1.22 billion in global revenue. Its momentum continued this year, with worldwide second-quarter sales rising 27% year over year to $377 million.
Future Directions and Challenges
Regeneron is also evaluating Libtayo in combination with its own antibody, fianlimab, as a first-line treatment for melanoma, where it is being compared with Keytruda. During the company’s second-quarter earnings call, Regeneron Chief Scientific Officer George Yancopoulos, M.D., Ph.D., said that if the data confirm strong results in melanoma, it would strengthen confidence in testing the combination in other cancer settings.
Regeneron stated that the FDA approval for Libtayo did not include Catalent’s Indiana facility as a fill-finish site. The facility, which was acquired by Novo Nordisk last year, had previously been cited by the FDA following inspections that uncovered manufacturing lapses, including the improper investigation of contaminants such as cat hair. Regeneron said in August that it is working with regulators to address the issues, which have delayed approvals for other therapies, including Scholar Rock’s muscle weakness treatment.
Libtayo is already approved in the U.S. for advanced skin cancer, basal cell carcinoma, advanced non-small cell lung cancer, and cervical cancer. The recent FDA clearance adds another indication for the drug, as an adjuvant therapy for adult patients with CSCC at high risk of recurrence after surgery and radiation.
In the years ahead, we may see further refinements: biomarkers to select those most likely to benefit, shorter courses, or combination regimens that maintain efficacy while limiting toxicity. For now, the FDA’s decision delivers tangible progress for a group of skin cancer patients previously with few options after surgery and radiation.
