A “Breakthrough Therapy” designation was granted by the Food and Drug Administration (FDA) to Merck’s Keytruda for the treatment of non-small cell lung cancer patients whose disease continued progressing on or following platinum-based chemotherapy.
Pembrolizumab by Merck & Co. (Keytruda) is an anti-programmed cell death protein 1 (anti-PD1) that targets a family of receptors expressed both in immune cells and tumors; these receptors are responsible for tumor cell evasion from immune-mediated responses. It is an anti-PD1 therapy for patients with Epidermal Growth Factor Receptor (EGFR) mutation-negative and Anaplastic Lymphoma Kinase (ALK) rearrangement-negative non-small cell lung cancer, and it was previously granted a breakthrough therapy designation for the treatment of advanced melanoma.
Roger Perlmutter, MD, president of Merck Research Laboratories, said in a press release, “The FDA’s Breakthrough Therapy Designation of Keytruda underscores that new treatment approaches for advanced non-small cell lung cancer continue to be needed (…) 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.”
Pembrolizumab is already administered in patients suffering with unresectable or metastatic melanoma and disease progression following ipilimumab (a human monoclonal antibody that blocks interaction between PD-1 and its ligands PD-L1 and PD-L2 allowing anti-tumor immune responses) and a BRAF inhibitor in those who are BRAF V600 mutation-positive.
An accelerated approval based on tumor response rates and durability of responses has been granted, however it has not yet been defined if the drug potentiates improvement in survival or disease-related symptoms.
Depending on the clinical benefit outcomes from ongoing studies, a continued approval for pembrolizumab will be decided. Currently, the drug is being tested for more than 30 types of cancers, both as a monotherapy and in combination treatment.
A previous clinical trial in advanced melanoma registered adverse events such as pneumonitis (2.9%), colitis (1%), hepatitis (0.5%), hypophysitis (0.5%), nephritis (0.7%), and hyperthyroidism (1.2%).
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).