FDA Gives Breakthrough Designation to Merck Drug

Merck recently announced the US Food and Drug Administration (FDA) has designated lambrolizumab (MK-3475) as a Breakthrough Therapy for the treatment of patients with advanced melanoma. Lambrolizumab is Merck’s investigational antibody therapy targeting Programmed Death receptor (PD-1) that is currently being evaluated for the treatment of patients with advanced melanoma, and other tumor types.

“We are pleased that the FDA has designated lambrolizumab a Breakthrough Therapy for patients with advanced melanoma,” said Gary Gilliland MD, PhD, Senior Vice President and oncology franchise head, Merck Research Laboratories. “The FDA’s decision to place lambrolizumab in a category that may enable expedited development and review is an important milestone for Merck as we advance ongoing programs in multiple cancer indications.”

The designation of an investigational drug as a Breakthrough Therapy is intended to expedite the development and review of a candidate that is planned for use, alone or in combination, to treat a serious or life-threatening disease or condition when preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints. The FDA Safety and Innovation Act (FDASIA) includes a provision that allows sponsors to request that an investigational drug be designated as a Breakthrough Therapy. The implications of Breakthrough Therapy Designation cannot be determined at this time.

In November 2012, early interim results from a single-arm, open-label Phase Ib study of lambrolizumab (MK-3475) administered to 85 patients with advanced (inoperable and metastatic) melanoma were presented at the Society for Melanoma Research of the 9th International Congress of the Society for Melanoma Research (SMR) in Hollywood, CA. (see news release).

Advanced melanoma accounts for more than 80% of skin cancer-related deaths and one to 2% of all cancer deaths in the US. According to the American Cancer Society, in 2012 in the US an estimated 9,180 people died from advanced melanoma.

Researchers have shown that several tumor types are able to hide in plain sight by establishing a “molecular camouflage” that deceives the body’s immune system into thinking they are normal and therefore allow them to grow unchecked. The interaction between the immune checkpoint receptor PD-1 (programmed death) and its ligands represents a potentially important tumor-specific immunomodulatory mechanism. By utilizing the PD-1 pathway, a tumor cell can prevent the activation of T-cells and therefore may block a key step that triggers the immune system.

Lambrolizumab is an investigational antibody therapy designed to disrupt the action of the immune checkpoint protein PD-1 and therefore inhibit the ability of some cancers to evade the body’s immune system. Lambrolizumab is being studied in multiple cancer types including melanoma and non-small cell lung cancer. For further details, please visit http://clinicaltrials.gov.