Cemiplimab Receives FDA Breakthrough Therapy Designation for Advanced Cutaneous Squamous Cell Carcinoma

The FDA has granted Breakthrough Therapy designation status to Regeneron Pharmaceuticals, Inc. and Sanofi's cemiplimab (REGN2810) for the treatment of adults with metastatic cutaneous squamous cell carcinoma (CSCC) and adults with locally advanced and unresectable CSCC. Cemiplimab is an investigational human, monoclonal antibody targeting PD-1.

Regeneron and Sanofi previously reported positive, preliminary results for cemiplimab from two expansion cohorts involving 26 advanced CSCC patients in a Phase 1 study of nearly 400 patients, at the American Society of Clinical Oncology (ASCO) Annual Meeting in June 2017. EMPOWER-CSCC 1, a Phase 2, potentially pivotal, single-arm, open-label clinical trial of cemiplimab is currently enrolling patients with metastatic CSCC and locally advanced and unresectable CSCC. Cemiplimab was discovered using Regeneron's proprietary VelocImmune technology that yields optimized fully-human antibodies, and is being jointly developed by Regeneron and Sanofi under a global collaboration agreement. Pending data results, the companies anticipate submitting a biologics license application for cemiplimab with the FDA in the first quarter of 2018.

Breakthrough Therapy designation serves to expedite the development and review of drugs that target serious or life- threatening conditions. Drugs qualifying for this designation must show credible evidence of a substantial improvement on a clinically significant endpoint over available therapies, or over placebo if there is no available therapy. The designation includes all of the Fast Track program features, as well as more intensive FDA guidance and discussion. The Breakthrough Therapy designation is distinct from both accelerated approval and priority review, which can also be granted to the same drug if relevant criteria are met.

Cemiplimab is currently under clinical development, and its safety and efficacy has not been fully evaluated by any regulatory authority.