FDA Accepts Cemipilmab BLA for Priority Review


The U.S. FDA has accepted for priority review the Biologics License Application for cemiplimab (REGN2810), an investigational anti-PD-1 monoclonal antibody for patients with metastatic CSCC.

The U.S. Food and Drug Administration (FDA) has accepted for priority review the Biologics License Application (BLA) for cemiplimab (REGN2810), an investigational anti-PD-1 monoclonal antibody for patients with metastatic cutaneous squamous cell carcinoma (CSCC).

The target action date for the FDA decision is October 28, 2018.

Metastatic CSCC, also known as locally advanced CSCC, is the most fatal non-melanoma skin cancer, and the second most common type of skin cancer in the U.S. There are currently no FDA-approved treatments available for the indication, and the new regulatory action is intended for patients who are not candidates for surgery.

The drug is currently under clinical development, and is being jointly developed by Regeneron and Sanofi under a global collaboration agreement. As of this writing, its safety and efficacy have not been fully evaluated by any regulatory authority.

The BLA submission is based on data from the Phase 2 EMPOWER-CSCC 1 study, a pivotal, single-arm, open-label clinical trial of cemiplimab for advanced CSCC, as well as Phase 1 data from a pair of advanced CSCC expansion cohorts. Both clinical trials enrolled patients with metastatic CSCC and patients with locally advanced CSCC who were not candidates for surgery.

Topline results from the Phase 2 EMPOWER-CSCC 1 were announced in December 2017, and in the data presented, it was proven that the PD-1 inhibitor induced an overall response rate (ORR) of 46.3% in patients with advanced CSCC. The data reported from the ongoing, single-arm, open-label study were for 82 patients. An estimated two-thirds of the cohort had progressed following systemic chemotherapy or radiation.

“For patients with CSCC who cannot be cured by surgery or radiation, there are no FDA-approved treatment options,” said Israel Lowy, MD, PhD, vice president of Global Clinical Development and Head of Translational Science and Clinical Oncology, Regeneron, in a press release after the data were presented. “This is the largest prospective study ever conducted in this disease, and we are pleased that many patients were able to achieve deep and durable responses with cemiplimab monotherapy. The high and durable response rates seen in this study are particularly notable given that the study enrolled patients regardless of biomarker status.”

Updated results from both the Phase 2 EMPOWER-CSCC 1 study and Phase 1 data from two advanced CSCC expansion cohorts will be presented at the 2018 ASCO Annual Meeting.

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