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The Food and Drug Administration has accepted a supplemental Biologics License Application for and granted Priority Review status to the anti-PD-1 therapy Keytruda (pembrolizumab) for the treatment of patients with advanced non-small cell lung cancer (NSCLC) whose disease has progressed on or after platinum-containing chemotherapy and an FDA-approved therapy for EGFR or ALK genomic tumor aberrations, if present.

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The FDA's Priority Review designation shortens the time to complete a drug's review and aims to deliver a decision on marketing approval designation for drugs that may offer major advances in treatment or provide a treatment where no adequate therapy exists within six months under the Prescription Drug User Fee Act (PDUFA). The FDA action date for Keytruda for this indication is October 2.


Keytruda, marketed by Merck, is a humanized monoclonal antibody that blocks the interaction between PD-1 and its ligands, PD-L1 and PD-L2, by binding to the PD-1 receptor and blocking the interaction with the receptor ligands. Keytruda releases the PD-1 pathway-mediated inhibition of the immune response, including the anti-tumor immune response. Keytruda received Breakthrough Therapy status for this designation for NSCLC last year (OT 11/25/14 issue).


The drug is already FDA-approved for the treatment of patients with unresectable or metastatic melanoma and disease progression following treatment with ipilimumab and, if BRAF V600-mutation positive, a BRAF inhibitor (OT 10/10/14 issue).


Keytruda's submission for this new application were based on data from the KEYNOTE-001 trial, which were reported in April at the American Association for Cancer Research Annual Meeting (OT 6/10/15 issue).


The FDA also granted Orphan Drug status to GMI-1271 for use in combination with chemotherapy for the treatment of patients with acute myeloid leukemia (AML). GMI-1271 is a novel and proprietary E-selectin antagonist that increases the ability of chemotherapy to kill cancer cells.


The Orphan Drug designation-to encourage development of drugs in the diagnosis, prevention, or treatment of a medical condition affecting fewer than 200,000 people in the U.S.-grants a product market exclusivity for a seven-year period if the sponsor complies with certain FDA specifications, as well as tax credits and prescription drug user fee waivers. The designation does not, though, shorten the duration of the regulatory review and approval process.


Approximately 20,830 individuals in the U.S. will be diagnosed with AML this year, and 10,460 will die from the disease, according to estimates from the American Cancer Society.


An open-label, multicenter Phase I/II study to evaluate the safety, pharmacokinetics, and efficacy of GMI-1271 used in combination with chemotherapy for the treatment of adult patients with AML is currently recruiting patients. Preclinical data supporting use of the drug were presented at the 2013 American Society of Hematology Annual Meeting.