Keytruda Granted Breakthrough Status for Lung Cancer

The Food and Drug Administration has granted a breakthrough therapy designation to the immunotherapy drug Keytruda (pembrolizumab) for the treatment of patients with non-small cell lung cancer (NSCLC) who are EGFR mutation- or ALK rearrangement-negative and whose disease has progressed on or following platinum-based chemotherapy.

[Breakthrough status explained: FDA’s Breakthrough Therapy Designation Aims to Speed Drug Approvals]

“The emergence of immunotherapies for lung cancer means we are on the cusp of something really great, and the nice thing about immunotherapy is that it might work even in the resistant patient,” said Roy S. Herbst, chief of medical oncology at the Yale Cancer Center and Smilow Cancer Hospital at Yale-New Haven, Connecticut, during his presentation at the 12th Annual International Congress on Targeted Therapies in Cancer.

Data from the ongoing phase 1b KEYNOTE-001 study, and updated findings recently presented at the 2014 European Society for Medical Oncology (ESMO) Congress, formed a basis for the designation. Keytruda was previously granted breakthrough status and ultimately gained accelerated approved for advanced melanoma. It is being studied across more than 30 types of cancers in more than two dozen clinical trials.

In advanced lung cancer, the drug is being studied as a single agent and in combination. A phase 1 study and a separate phase 3 study are ongoing in advanced lung cancer (KEYNOTE-010 and KEYNOTE-024), and an additional phase 3 study is planned to begin in the fourth quarter of 2014 (KEYNOTE-042).

Keytruda is one of several new treatments that boosts the immune system against cancer cells by blocking the PD‐1 signaling pathway inside cells. When Keytruda binds to the PD-1 receptor of certain T lymphocytes, it blocks that signaling pathway and ultimately releases the immune system to attack the cancer cell. Another PD-1 inhibitor, nivolumab, is also being extensively tested in lung cancer, as well as in other solid tumors.

“There is a huge need (in lung cancer),” says Alex Spira, medical director of medical oncology at the Inova Thoracic Oncology Program in Falls Church, Virginia. “Their approval will be a game changer, and we expect one of them to be approved next year.”