New Agent for Treating NSCLC Patients Receives Breakthrough Therapy Designation

An investigational cancer immunotherapy known as MPDL3280A (anti-PDL1) is receiving a breakthrough therapy designation from the US Food and Drug Administration (FDA) for treating non-small cell lung cancer (NSCLC). The designation was announced on February 1, 2015 for those patients with PD-L1 (programmed death-ligand 1) positive NSCLC whose disease has progressed during or after platinum-based chemotherapy and an appropriate targeted therapy for those with an EGFR mutation-positive or ALK-positive tumor.

Sandra Horning, MD, who is Chief Medical Officer and head of Global Product Development for the Roche Group, said the company is pleased the FDA has granted breakthrough designation for MPDL3280A in NSCLC. She said the company is committed to personalized healthcare and developing medicines like MPDL3280A with companion tests that may help better identify those patients who may be appropriate candidates.

Several studies of MPDL3280A are currently underway and they are prospectively evaluating PD-L1 expression. Some studies are evaluating the medicine regardless of a tumor’s PD-L1 status. Other studies are evaluating the medicine only in patients whose tumors are characterized as PD-L1 positive. The breakthrough therapy designation for lung cancer patients are based on early results of MPDL3280A in patients whose NSCLC was characterized as PD-L1 positive by an investigational test being developed by Roche.

It is hoped the new breakthrough therapy designation will help improve morbidity and mortality in a significant number of NSCLC patients sooner rather than later. This type of designation is designed to expedite the development and review of medicines intended to treat serious diseases and to help ensure patients have access to them through FDA approval as soon as possible.

The FDA granted the first breakthrough therapy designation for MPDL3280A in metastatic bladder cancer last year. Dr. Horning said ongoing pivotal studies of MPDL3280A for treating lung and bladder cancer are now underway. The company plans to initiate phase III studies in additional tumor types in 2015.

MPDL3280A (also known as anti-PDL1 and RG7446) is an investigational monoclonal antibody. It targets PD-L1 expressed on tumor cells and tumor-infiltrating immune cells, and it prevents it from binding to PD-1 and B7-1 on the surface of T cells. It is theorized that by inhibiting PD-L1, this new agent can activate T cells and restore their ability to effectively detect and attack tumor cells.