Merck's Rapid Strides With Pembrolizumab

Today, Merck announced that Keytruda has been granted breakthrough therapy designation for treating patients with epidermal growth factor receptor mutation-negative and anaplastic lymphoma kinase rearrangement-negative non-small cell lung cancer (NSCLC).

With the immuno-oncology realm heating up, pharmaceutical companies are in a tight race trying to bring their products to the market to treat a variety of cancers. The approval of ipilimumab (Yervoy, BMS) in 2011, the first CTLA-4 inhibitor approved for the treatment of advanced melanoma, was followed by a lag phase in immunotherapy.

However, at the annual meeting of the American Society of Clinical Oncology, held in May 2014 in Chicago, immuno-oncology took center stage as the big players in the field, BMS, Merck, Roche, and AstraZeneca presented encouraging data on their PD-1 and PD-L1 inhibitors. While BMS is still the biggest player, devoting an estimated $649 million to immuno-oncology studies with 78 trials, Merck has an estimated $327 million invested on immune drugs in oncology.

And then, early last month, the FDA announced the approval of Merck’s PD-1 inhibitor, pembrolizumab (Keytruda), in patients with advanced melanoma who had developed resistance to other treatments. The first PD-1 inhibitor to be approved, Keytruda is recommended for use in melanoma patients who fail Yervoy and in those who harbor the BRAF V600 mutation and have been treated with Yervoy and a BRAF inhibitor. Despite being the sixth melanoma treatment to be approved since 2011, FDA’s director of the Office of Hematology and Oncology, Richard Pazdur, MD, said, “Many of these treatments have different mechanisms of action and bring new options to patients with melanoma.” Keytruda, which received a priority review and a breakthrough designation, was granted accelerated approval following its evaluation in 173 patients with advanced melanoma.

Today, Merck announced that the drug has been granted breakthrough therapy designation for treating patients with epidermal growth factor receptor mutation-negative and anaplastic lymphoma kinase rearrangement-negative non-small cell lung cancer (NSCLC), who present with disease progression after platinum-based chemotherapy. The FDA reviewed the phase 1b results of the KEYNOTE-001 study that is ongoing, and whose updated findings were presented at the European Society for Medical Oncology 2014 Congress held in Spain last month.

“The FDA’s Breakthrough Therapy Designation of KEYTRUDA underscores that new treatment approaches for advanced non-small cell lung cancer continue to be needed,” said Dr Roger Perlmutter, president, Merck Research Laboratories. “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.”

While phase 2 and phase 3 studies in NSCLC are ongoing for Keytruda, the drug is being evaluated as monotherapy and as combination therapy in 30 different cancer subtypes.