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The Food and Drug Administration has granted Breakthrough Therapy status to Keytruda (pembrolizumab), an anti-PD-1 therapy, for the treatment of patients with non-small-cell lung cancer (NSCLC) that has progressed on or following platinum-based chemotherapy and that is also epidermal growth factor receptor (EGFR) mutation-negative and anaplastic lymphoma kinase (ALK) rearrangement-negative.

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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.


Keytruda, made by Merck, was recently approved for the treatment of patients with advanced or unresectable melanoma who are no longer responding to other drugs under the FDA's Accelerated Approval program (OT 10/10/14 issue). Keytruda also received breakthrough therapy designation, priority review, and orphan drug status for that indication in melanoma.


The Phase Ib KEYNOTE-001 study of Keytruda in advanced NSCLC is currently ongoing. Updated findings from that trial were reported this fall at the European Society for Medical Oncology Congress.