Merck’s KEYTRUDA (pembrolizumab), has been granted Breakthrough Therapy Designation by the U.S. Food and Drug Administration (FDA) for the treatment of patients with Epidermal Growth Factor Receptor (EGFR) mutation-negative, and Anaplastic Lymphoma Kinase (ALK) rearrangement-negative non-small cell lung cancer (NSCLC) whose disease has progressed on or following platinum-based chemotherapy.
“The FDA’s Breakthrough Therapy Designation of KEYTRUDA underscores that new treatment approaches for advanced non-small cell lung cancer continue to be needed,” Dr. Roger Perlmutter, president, Merck Research Laboratories said in a press release. “Our data investigating the use of KEYTRUDA in this difficult-to-treat malignancy are very encouraging, and we look forward to working closely with the FDA to expedite our clinical program.”
Earlier this year, KEYTRUDA had already received a Fast Track designation by the FDA for the treatment of advanced stage, unresectable, and unresponsive melanoma.
KEYTRUDA is an anti-PD-1 (programmed death receptor-1) therapy, targeting a family of receptors and ligands expressed both in immune cells and tumors, which are responsible for tumor cell evasion from immune-mediated responses.
PD-1/PD-L1 are two immune checkpoints exponentially targeted in the development of new immunotherapies against several types of cancers, since they are crucial for an efficient anti-tumoral immune response by T cells.
While the majority of anti-cancer drugs aims to directly kill cancer cells, this type of immunotherapeutic drugs enhance the body’s own ability to recognize and destroy cancer cells, resulting in immunological memory that lasts for a longer time and can prevent cancer recurrence.
The awarded Breakthrough Therapy Designation in advanced NSCLC is based on encouraging data from the ongoing Phase 1b KEYNOTE-001 study, whose findings were presented at the European Society of Medical Oncology (ESMO) 2014 Congress. This designation is given to accelerate the development and review of a drug considered to treat a serious or life-threatening disease when preliminary clinical data suggests the drug may provide significant improvement over existing therapies.
Currently, Merck has two ongoing Phase 2 and 3 clinical trials in advanced lung cancer (KEYNOTE-010 and KEYNOTE-024) evaluating the efficacy and safety of KEYTRUDA, and will begin an additional Phase 3 study by the end of 2014 (KEYNOTE-042).