Type of information: Granting of a Market Authorisation in the US
Product name: Libtayo® (cemiplimab-rwlc REGN2810)
Therapeutic area: Cancer - Oncology
- monoclonal antibody/immune checkpoint inhibitor. REGN2810 is a fully human monoclonal antibody to Programmed Death-1 (PD-1).
- Cemiplimab is being jointly developed by Regeneron and Sanofi under a global collaboration agreement, and was invented by Regeneron using the company's proprietary VelocImmune® technology.
Company: Regeneron Pharmaceuticals (USA - NY) Sanofi (France)
- metastatic cutaneous squamous cell carcinoma (CSCC) or locally advanced CSCC who are not candidates for curative surgery or curative radiation
- • On September 28, 2018, the FDA approved Libtayo® (cemiplimab-rwlc) injection for intravenous use for the treatment of patients with metastatic cutaneous squamous cell carcinoma (CSCC) or locally advanced CSCC who are not candidates for curative surgery or curative radiation. This is the first FDA approval of a drug specifically for advanced CSCC. CSCC is the second most common human cancer in the United States with an estimated annual incidence of approximately 700,000 cases. CSCC usually develops in skin areas that have been regularly exposed to the sun or other forms of ultraviolet radiation. While the majority of patients with CSCC are cured with surgical resection, a small percentage of patients will develop advanced disease that no longer responds to local treatments including surgery and radiation. Advanced CSCC may cause disfigurement at the site of the tumor and local complications such as bleeding or infection, or it may spread (metastasize) to local lymph nodes, distant tissues and organs and become life-threatening.
- The safety and efficacy of Libtayo was studied in two open label clinical trials. A total of 108 patients (75 with metastatic disease and 33 with locally-advanced disease) were included in the efficacy evaluation. The study’s primary endpoint was objective response rate, or the percentage of patients who experienced partial shrinkage or complete disappearance of their tumor(s) after treatment. Results showed that 47.2 percent of all patients treated with Libtayo had their tumors shrink or disappear. The majority of these patients had ongoing responses at the time of data analysis.
Pivotal advanced CSCC clinical program and results
The FDA approval of Libtayo was based on a combined analysis of data from an open-label, multi-center, non-randomized Phase 2 trial known as EMPOWER-CSCC-1 (Study 1540) and two advanced CSCC expansion cohorts from a multi-center, open-label, non-randomized Phase 1 trial (Study 1423). Together, the trials represent the largest prospective data set in advanced CSCC.
- The major efficacy outcome measures for the integrated analysis of EMPOWER-CSCC-1 and the two CSCC expansion cohorts were confirmed objective response rate (ORR), as assessed by independent central review (ICR), and ICR-assessed duration of response (DOR). The efficacy analysis was conducted when all patients had the opportunity for at least six months of follow-up.
Combined efficacy results (n=108) from EMPOWER-CSCC-1 and the two advanced CSCC expansion cohorts from the Phase 1 trial were as follows:
+Denotes ongoing at last assessment
*Median duration of follow-up: metastatic CSCC: 8.1 months; locally advanced CSCC: 10.2 months; combined CSCC: 8.9 months
†Only includes patients with complete healing of prior cutaneous involvement; locally advanced CSCC patients in EMPOWER-CSCC-1 required biopsy to confirm complete response.
For the combined safety analysis (n=163) of EMPOWER-CSCC-1 and the two advanced CSCC expansion cohorts, the most common adverse reactions reported were fatigue (29%), rash (25%) and diarrhea (22%). Libtayo was permanently discontinued due to adverse reactions in 5% of patients; adverse reactions resulting in permanent discontinuation were pneumonitis, autoimmune myocarditis, hepatitis, aseptic meningitis, complex regional pain syndrome, cough and muscular weakness. Serious adverse reactions (SAEs) occurred in 28% of patients. SAEs that occurred in at least 2% of patients were cellulitis, sepsis, pneumonia, pneumonitis and urinary tract infection.
(n = 75)
(n = 108)
(95% confidence interval [CI])
|Complete response rate†
|Partial response rate
|Range in months
|Patients with DOR ? 6 months, n (%)
- • On December 13, 2017, Regeneron Pharmaceuticals and Sanofi announced positive topline results from a pivotal Phase 2 clinical study of cemiplimab in 82 patients with advanced cutaneous squamous cell carcinoma (CSCC). These pivotal data will form the basis of a rolling Biologics License Application (BLA) submission to the FDA, which has been initiated and is expected to be completed in the first quarter of 2018. A submission to the European Medicines Agency (EMA) is also expected to be completed in the first quarter of 2018.
- • On September 8, 2017, Sanofi and Regeneron announced that the FDA has granted Breakthrough Therapy designation status to cemiplimab (REGN2810) for the treatment of adults with metastatic cutaneous squamous cell carcinoma (CSCC) and adults with locally advanced and unresectable CSCC.
Submission of marketing authorization application USA :
Submission of marketing authorization application UE:
Withdrawal of marketing authorization application USA:
Withdrawal of marketing authorization application UE:
US authorization: 2018-09-28
Favourable opinion UE:
Favourable opinion USA:
Orphan status USA:
Orphan status UE:
Pediatric exclusivit _USA:
Pediatric exclusivity UE: