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The U.S. Food and Drug Administration has granted breakthrough therapy designation to MPDL3280A for the treatment of patients previously treated for metastatic urothelial bladder cancer who are PD-L1 positive. MPDL3280A is an anti-PDL1 monoclonal antibody, which targets PD-L1 expressed on tumor and tumor-infiltrating immune cells to activate T cells to restore their ability to effectively detect and attack tumor cells.

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The breakthrough therapy designation, enacted as part of the FDA's 2012 Safety and Innovation Act, was created to expedite the development and review time of a potential new drug for serious or life-threatening disease where early clinical evidence suggests the drug may demonstrate substantial improvement compared with existing therapies.


This latest action for MPDL3280A is based on data from a Phase I study of 68 patients with previously treated, metastatic bladder cancer. Of the 30 patients who were identified as having PD-L1 positive tumors, 13 patients (43%) responded to the drug, having their tumors shrink after six weeks of follow up, and 2 patients (7%) had no radiographic evidence of tumor after 12 weeks of follow up. Four out of 35 patients in the study who were PD-L1-negative responded to the drug, having some tumor shrinking (11%).


Additional follow-up data were presented at the American Society of Clinical Oncology Annual Meeting (Abstract 5011).


The drug is marketed by Genentech.